Eligibility criteria for study enrollment:
• Signed Informed Consent Form (ICF) by parent(s)/guardian(s) of subjects and informed assent form (IAF) for subjects ≥7 years, or as per local or country specific guidelines or regulations.
• Male or female subjects 2 to 17 years old at Visit 1.
• Documented medical history or physician-confirmed diagnosis of IgE-mediated CMA with systemic symptoms related to ingestion of milk or dairy products.
• Subjects currently following a strict cow's milk-free diet, with no consumption of dairy or baked milk products.
• Cow's milk-specific IgE level at screening ≥10 kU/L
• Positive Skin Prick Test (SPT) to cow's milk with a largest wheal diameter ≥6 mm.
• Positive DBPCFC at screening with an eliciting dose ≤300 mg cow's milk proteins (approximately ≤9.4 mL of cow's milk).
• Negative urine pregnancy test for female subjects of childbearing potential. Female subjects of childbearing potential must agree and commit to use effective medical methods of contraception for the entire duration of their participation in the study. Sexual abstinence will be accepted as an effective method of contraception for girls below 15 years of age.
• Ability to perform spirometry procedures in accordance with the American Thoracic Society guidelines (2005) for subjects ≥6 years old. Ability to perform peak expiratory flow (PEF) measurements for subjects ≥5 years old. Subjects <8 years of age who have documented inability to adequately perform spirometry can perform only the PEF evaluation. Subjects <5 years of age may be enrolled if they had no clinical features of moderate or severe persistent asthma severity (as defined by the 2007 National Heart, Lung, and Blood Institute [NHLBI] Guidelines) within 1 year before Visit 1.
• Subjects and/or parents/guardians willing to comply with all study requirements during participation in the study.
• History of severe anaphylaxis to cow's milk resulting in hypotension, hypoxia or neurological compromise (collapse, loss of consciousness or incontinence) or requiring mechanical ventilation.
• Pregnancy or lactation.
• Spirometry forced expiratory volume in 1 second (FEV1) <80% of the predicted value at Visit 1 for subjects ≥6 years and able to perform the spirometry, or PEF <80% of predicted value at Visit 1 for subjects performing only the PEF measurements.
• Any clinical features of moderate or severe persistent asthma severity (as defined by the 2007 NHLBI guidelines) and high daily doses of inhaled corticosteroids.
• Known allergy to the Viaskin patch materials or excipients, or to any of the components of the food challenge formulas other than the cow's milk proteins.
• Allergy or known history of reaction to Tegaderm® medical dressing with no possibility to use an alternative adhesive dressing authorized by the sponsor in replacement.
• Subjects having objective symptoms to the placebo formula leading to stopping the challenge during the screening DBPCFC.
• Severe reaction during the screening DBPCFC defined as need for intubation, and/or hypotension persisting after epinephrine administration, and/or the need for >2 doses of epinephrine.
• Symptomatic allergy to pollens with symptoms during the pollen season that might interfere with the symptoms observed during the DBPCFC, if the DBPCFC is performed during the pollen season. Screening of such subjects should be made out of the pollen season.
• Inability to discontinue short-acting antihistamines for 3 days or long-acting antihistamines for 5 to 7 days (depending on the half-life) before the DBPCFC.
• Use of systemic long-acting corticosteroids within 12 weeks before Visit 1 and/or use of systemic short-acting corticosteroids within 4 weeks before Visit 1 or use of systemic long-acting or short-acting corticosteroids during screening (unless used to treat symptoms triggered by the DBPCFC or triggered by accidental allergen consumption; in the latter case DBPCFC must then be scheduled after a minimum of 7 wash-out days).
• Subjects with asthma conditions meeting 1 or several criteria below:
• Uncontrolled persistent asthma (as defined by the 2007 NHLBI guidelines) or subject being treated with a combination therapy of medium or high daily dose of inhaled corticosteroid with a long acting inhaled β2-agonist. Intermittent asthmatic subjects who require intermittent use of inhaled corticosteroids for rescue are permitted.
• At least 2 systemic corticosteroid courses for asthma within 1 year before Visit 1 or 1 oral corticosteroid course for asthma within 3 months before Visit 1, or during screening (unless used to treat symptoms triggered by the DBPCFC).
• Prior intubation/mechanical ventilation due to asthma within 2 years before Visit 1, or during screening.
• Upper respiratory infection or gastroenteritis within 7 days of DBPCFC (DBPCFC must then be rescheduled at least 7 days after resolution of these conditions).
• Any history of milk immunotherapy (eg, oral immunotherapy, sublingual immunotherapy or specific oral tolerance induction).
• Prior history of any other food allergen immunotherapy (eg, oral immunotherapy, sublingual immunotherapy or specific oral tolerance induction) within 5 years before Visit 1.
• Subjects currently under aeroallergen immunotherapy and unwilling or unable to discontinue at the time of Visit 1. Aeroallergen Immunotherapy must be discontinued at the time of Visit 1.
• Use of any anti-IgE drug (eg, omalizumab), any immunomodulatory therapy, or any biological agent therapy (eg, anti-tumor necrosis factor drugs) within 1 year before Visit 1, or during screening.
• Generalized dermatologic diseases (eg, severe atopic dermatitis, uncontrolled generalized eczema, icthyosis vulgaris) with no intact zones to apply the Viaskin patch, or urticarial and mast cells disorders such as chronic idiopathic urticaria.
• Subject and/or subject's parents/guardians with obvious excessive anxiety and unlikely to cope with the conditions of a food challenge.
• Past or current disease, including but not limited to active eosinophilic gastrointestinal disorders, autoimmune disorders, immunodeficiency, malignancy, uncontrolled disease (hypertension, diabetes, psychiatric disorder, cardiac disease), or other disorders (eg, liver, gastrointestinal, kidney, cardiovascular, pulmonary disease or blood disorder) which in the opinion of the Investigator or the sponsor may affect the subject's participation in the study or place the subject at increased risk.
• Subjects and/or parents/guardians unable to use the epinephrine auto-injector properly in spite of being adequately trained.
• Contraindicated condition for the use of epinephrine.
• Use of any investigational drug or device, or participation in another interventional clinical study within 3 months before Visit 1.
• Subjects receiving beta-blockers or Angiotensin converting-enzyme (ACE) inhibitors.
• Subjects unable to follow the protocol requirements.

For more information regarding Bristol-Myers Squibb Clinical Trial participation, please visit www.BMSStudyConnect.com
• Meets the Systemic Lupus International Collaborating Clinics (SLICC) classification criteria for SLE and diagnosed ≥ 24 weeks before the screening visit
• One of the following: antinuclear antibody (ANA) ≥ 1:80 or positive anti-double-stranded DNA (dsDNA) or positive anti-Smith (Sm)
• Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score ≥ 6 points and clinical SLEDAI-2K score ≥ 4 points
• Subjects with drug-induced SLE, certain other autoimmune diseases, and active, severe lupus nephritis
• SLE overlap syndromes such as scleroderma and mixed connective tissue disease
• Clinically significant abnormalities on chest x-ray or ECG
• History of any significant drug allergy Other protocol defined inclusion/exclusion criteria could apply

1. Male or female ≥ 18 years of age. 2. Myasthenia Gravis Foundation of America (MGFA) Class II-IVa and likely not in need of a respirator for the duration of the study as judged by the Investigator. 3. QMG score ≥12 at Screening and Baseline. Other, more specific inclusion criteria are defined in the protocol. 1. Use of rituximab, belimumab, eculizumab or any monoclonal antibody for immunomodulation within 6 months prior to first dosing. 2. Immunoglobulins given by SC, IV (IVIG), or intramuscular route, or plasmapheresis/plasma exchange (PE) within 4 weeks before Screening. 3. Thymectomy performed < 12 months prior to screening. 4. Total IgG level <6 g/L (at screening). 5. Absolute neutrophil count <1500 cells/mm3(at screening). Other, more specific exclusion criteria are defined in the protocol

1. Between 15 and 49 years of age, inclusive, at Visit 1. 2. Anti-nuclear antibody (ANA) titer of 1:80, or greater, as determined by immunofluorescence assay (IFA). 3. Participants must have at least one (but not three or more) additional clinical or laboratory criterion from the 2012 Systemic Lupus International Collaborating Clinics (SLICC) classification criteria. 4. Written informed consent (and assent when applicable) obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study. 1. The subject meets the 2012 SLICC classification criteria for SLE at Visit 1 (i.e., ANA plus 3 other criteria, or ANA plus biopsy-proven lupus nephritis). 2. The subject has been diagnosed with another autoimmune disorder, other than autoimmune thyroid conditions. 3. The subject has fibromyalgia, based on clinical history and exam. 4. The subject has previously been or is currently being treated with oral antimalarial agents including hydroxychloroquine, chloroquine, or quinacrine. 5. The subject is currently or has been treated with immunosuppressive, immune modifying, or cytotoxic medications as listed in Section 7.2. 6. Use of any investigational agent within the preceding 12 months. 7. History of primary immunodeficiency. 8. Active bacterial, viral, fungal, or opportunistic infection. 9. Evidence of infection with human immunodeficiency virus (HIV), Hepatitis B, or Hepatitis C. 10. Concomitant malignancy or history of malignancy with the exception of adequately treated basal or squamous cell carcinoma of the skin, or carcinoma in situ of the cervix. 11. The subject has significant findings on ophthalmological examination that, in the opinion of the examining Ophthalmologist, prevent safe use of hydroxychloroquine. 12. The subject has other contraindications to treatment with hydroxychloroquine including pre-existing ocular disease, hepatic impairment, psoriasis, porphyria, or allergy to the drug or class. 13. Co-morbidities requiring systemic corticosteroid therapy greater than 10 mg of prednisone per day, or equivalent, or a change in corticosteroid dose within the 3 months prior to Visit 1. 14. Starting, stopping, or changing the dose of over the counter or prescription non-steroidal anti-inflammatory drugs (NSAIDs) in the three months prior to Visit 1. 15. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study. 16. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data. 17. Inability to comply with the study visit schedule and procedures.

• Physician-diagnosed peanut allergy;
• A peanut Skin Prick Test (SPT) with a wheal largest diameter ≥8 mm;
• A specific-peanut Immunoglobulin E (IgE) ≥14 kU/L;
• Subjects following a strict peanut-free diet.
• Generalized dermatologic disease
• Spirometry forced expiratory volume in 1 second (FEV1) <80% of the predicted value, or peak expiratory flow (PEF) <80% of predicted value;
• Receiving β-blocking agents, angiotensin-converting enzyme inhibitors, angiotensin-receptor blockers, calcium channel blockers or tricyclic antidepressant therapy; anti-tumor necrosis factor drugs or anti-IgE drugs (such as omalizumab) or any biologic immunomodulatory therapy;
• Prior or concomitant history of any immunotherapy to any food allergy (for example EPIT, OIT, SLIT, or specific oral tolerance induction).

Key
• Prior participation in an Aimmune AR101 clinical study or any future clinical study that identifies ARC008 as a follow-on study option in the protocol
• Written informed consent and/or assent from subjects/guardians as appropriate
• Use of effective birth control by sexually active female subjects of childbearing potential Key
• Did not complete a minimum of 3 months of AR101 maintenance therapy if the subject was assigned to AR101 in the parent study
• Early discontinuation from the parent study
• In subjects treated with AR101 in the parent study requiring a food challenge, failure to successfully consume at least 300 mg single dose of peanut protein at parent study's exit food challenge

1. Orthotopic heart transplantation 2. Age < 21 years at time of transplant 3. Stable immunosuppression at the time of randomization with no contraindication to everolimus, tacrolimus, or mycophenolate mofetil 4. Planned follow-up at a study site for the 30 month duration of the study. 5. Subject or legal adult representative capable of providing informed consent (in general, assent will be sought for children aged 12 years or older). 1. Multi-organ transplant (e.g. heart-lung or heart-liver). 2. Known hypersensitivity to everolimus, sirolimus, tacrolimus or mycophenolate mofetil (MMF), or to components of the drug products. 3. Patients on maintenance corticosteroid therapy exceeding a dose equivalent of prednisone 0.1 mg/kg/day at randomization. 4. High-risk for rejection defined as active rejection, recurrent (≥ 2 episodes of grade 2R rejection) cellular rejection, recurrent rejection (≥ 2 episodes of any grade) with hemodynamic compromise, steroid-resistant rejection or unresolved antibody-mediated rejection during the first 6 months post-heart transplant 5. Graft dysfunction (LVEF <40% or wedge pressure >22 mmHg or cardiac index <2.2 L/min/m2) 6. Stage 4 or 5 CKD (eGFR <30 ml/min/1.73 m2) or moderate proteinuria (urine protein to urine creatinine ratio >0.5 mg/mg). 7. Active infection requiring hospitalization or treatment dose medical therapy. 8. Patients with ongoing wound healing problems, clinically significant wound infection requiring continued therapy or other severe surgical complication in the opinion of the Site Principal Investigator. 9. Fasting Serum Cholesterol ≥300 mg/dL OR greater than or equal to 7.75 mmol/L, AND fasting triglycerides ≥2.5x the upper limit of normal (ULN). Note: In case one or both of these thresholds are exceeded, the patient can only be included after initiation of appropriate lipid lowering medication, and reduction of serum cholesterol and triglyceride levels to below exclusion ranges is confirmed. 10. Uncontrolled diabetes mellitus. 11. Diagnosis of post-transplant lymphoproliferative disorder (PTLD) during the first 6 months post-heart transplant. 12. History of non-adherence to medical regimens. 13. Patients who are treated with drugs that are strong inducers or inhibitors of cytochrome P450 3A4 (CYP3A4) and cannot discontinue the treatment 14. Patients who are pregnant or breast-feeding or intend to get pregnant during the study period.

• Written informed consent from participant's parent/guardian.
• Age 12-48 months of either sex, any race, any ethnicity.
• A peanut allergy diagnosis with a convincing clinical history of peanut allergy and a serum peanut-specific IgE [UniCAP] > 0.35 kUA/L AND a positive skin prick test to peanut (>3 mm than the negative control) OR are sensitized to peanut (based on a serum IgE [UniCAP] to peanut of > 5 kUA/L) AND a positive skin prick test to peanut (> 3 mm than the negative control) and no known history of ingestion of peanut.
• A positive DBPCFC to 1000 mg of peanut at enrollment.
• History of severe anaphylaxis to peanut, defined as hypoxia, hypotension, or neurologic compromise (cyanosis or SpO2 < 92% at any stage, hypotension, confusion, collapse or loss of consciousness).
• Participation in any interventional study for the treatment of food allergy in the past 6 months.
• Known oat, wheat, or glycerin allergy.
• Eosinophilic or other inflammatory (e.g. celiac) gastrointestinal disease.
• Severe asthma (2007 NHLBI Criteria Steps 5 or 6
•Appendix 2).
• Inability to discontinue antihistamines for skin testing and DBPCFCs.
• Use of omalizumab or other non-traditional forms of allergen immunotherapy (e.g., oral or sublingual) or immunomodulator therapy (not including corticosteroids) or biologic therapy within the past year.
• Use of beta-blockers (oral), angiotensin-converting enzyme (ACE) inhibitors, angiotensin-receptor blockers (ARB) or calcium channel blockers.
• Significant medical condition (e.g., liver, kidney, gastrointestinal, cardiovascular, hematologic, or pulmonary disease) which would make the subject unsuitable for induction of food reactions.

• Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IVa] at Screening
• Positive serology for acetylcholine receptor (AChR) autoantibodies
• QMG score ≥ 12 at Screening and Randomization
• No change in corticosteroid dose for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
• No change in immunosuppressive therapy, including dose, for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
• Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12 week Treatment Period
• History of meningococcal disease
• Current or recent systemic infection within 2 weeks prior to Randomization or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization

Inclusion Criteria 1. Subjects ≥ 6 years of age at time of Informed Consent Form (ICF) or Assent Form signing. 2. Confirmed diagnosis of CF, determined by having clinical features consistent with the CF phenotype, plus one of the following: 1. Positive sweat chloride test (value ≥ 60 mEq/L), 2. Genotype with 2 mutations consistent with CF (i.e., a mutation in each of the cystic fibrosis transmembrane conductance regulator [CFTR] genes). 3. Positive sputum culture or a throat swab culture for MRSA at Screening. 4. In addition to the Screening sample, have at least 2 prior sputum or throat swab cultures positive for MRSA, of which at least 1 sample is more than 6 months prior to Screening. At least 50% of all MRSA cultures (sputum or throat swab culture) collected from the time of the first positive culture (in the previous 1 year) must have tested positive for MRSA. (Note: Screening sample may count towards 50% positive count) 5. Forced expiratory volume in 1 second (FEV1) ≥ 30% and ≤ 90% of predicted that is normal for age, gender, race, and height, using the Global Lung Function Initiative (GLI) equation. 6. At least 1 episode of acute pulmonary infection treated with non-maintenance antibiotics within 12 months prior to the Baseline visit. (Initiation of treatment with intermittent inhaled anti-Pseudomonal therapy will not qualify as treatment with non-maintenance antibiotics). 7. If female of childbearing potential, an acceptable method of contraception must be used during the study and must be combined with a negative pregnancy test obtained during Screening; sexually active male subjects of reproductive potential who are non-sterile (i.e., male who has not been sterilized by vasectomy for at least 6 months, and were not diagnosed with infertility through demonstration of azoospermia in a semen sample and/or absence of vas deferens through ultrasound) must be willing to use a barrier method of contraception, or their female partner must use an acceptable method of contraception, during the study. For purposes of this study, the Sponsor defines "acceptable methods of contraception" as: 1. Oral birth control pills administered for at least 1 monthly cycle prior to administration of the study drug. 2. A synthetic progestin implanted rod (eg, Implanon®) for at least 1 monthly cycle prior to the study drug administration but not beyond the 4th successive year following insertion. 3. Intrauterine devices (IUDs), inserted by a qualified clinician for at least 1 monthly cycle prior to study drug administration. 4. Medroxyprogesterone acetate (eg, Depo-Provera®) administered for a minimum of 1 monthly cycle prior to administration of the study drug and continuing through 1 month following study completion. 5. Hysterectomy or surgical sterilization. 6. Abstinence. 7. Double barrier method (diaphragm with spermicidal gel or condoms with contraceptive foam). NOTE: For subjects prescribed Orkambi: Orkambi may substantially decrease hormonal contraceptive exposure, reducing the effectiveness and increasing the incidence of menstruation-associated adverse reactions. Hormonal contraceptives, including oral, injectable, transdermal, and implantable, should not be relied upon as an effective method of contraception when co-administered with Orkambi. 8. Able and willing to comply with the protocol, including availability for all scheduled study visits and able to perform all techniques necessary to use the AeroVanc inhaler and measure lung function. 9. Agree not to smoke during any part of the clinical trial (Screening visit through end of study). 10. Subjects with a P. aeruginosa co-infection must either be stable on a regular suppression regimen of inhaled antibiotics or must be, in the opinion of the Investigator, stable despite the lack of such treatment. Exclusion Criteria 1. Use of anti-MRSA treatments prescribed as maintenance therapy (intravenous [IV] or inhaled treatment within 28 days; oral treatment within 14 days) prior to the Baseline visit. 2. Use of non-maintenance antibiotic for pulmonary infection or extrapulmonary MRSA infection (IV or inhaled antibiotic within 28 days; oral antibiotic within 14 days) prior to the Baseline visit. 3. History of previous allergies or sensitivity to vancomycin, or other component(s) of the study drug or placebo except for a history of red-man syndrome. 4. Inability to tolerate inhaled products. 5. First time sputum culture or throat swab culture yielding B. cepacia, or nontuberculous Mycobacteria in the previous 6 months to Screening. 6. History of lung or other solid organ transplantation or currently on the list to receive lung or other solid organ transplantation. 7. Resistance to vancomycin at Screening (vancomycin resistant Staphylococcus aureus [VRSA], or vancomycin intermediate resistant Staphylococcus aureus [VISA], with minimum inhibitory concentration [MIC] ≥ 8 μg/mL). 8. Oral corticosteroids in doses exceeding 10 mg prednisone per day or 20 mg prednisone every other day, or equipotent doses of other corticosteroids. 9. Changes in antimicrobial, bronchodilator, anti-inflammatory or corticosteroid medications within 14 days, or changes in CFTR modulators within 28 days, prior to the Baseline visit. 10. Abnormal laboratory findings or other findings or medical history at Screening that, in the Investigator's opinion, would compromise the safety of the subject or the quality of the study data. 11. Inability to tolerate inhalation of a short acting beta2 agonist 12. SpO2 <90% at Screening. 13. Changes in physiotherapy technique or physiotherapy scheduled within 1 week of the Baseline visit. 14. Administration of any investigational drug or device within 4 weeks prior to the Screening visit and during the study 15. Female with positive pregnancy test result during Screening, pregnant (or intends to become pregnant), lactating or intends to breastfeed during the study. 16. Renal insufficiency, defined as creatinine clearance < 50 mL/min using the Cockcroft-Gault equation for adults or Schwartz equation for children at the Screening visit. 17. Abnormal liver function, defined as ≥ 4x upper limit of normal (ULN), of serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT), or known cirrhosis at Screening. 18. Diagnosed with clinically significant hearing loss. 19. History of positive result for human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV). 20. Planned hospitalizations for prophylaxis antibiotic treatment within 28 days prior to Baseline visit or during the double-blind period (Period 1).

• Subjects who completed the PEPITES study.
• Generalized dermatologic disease (for example, active atopic dermatitis, uncontrolled generalized active eczema, ichthyosis vulgaris) extending widely on the skin and especially on the back or arms with no intact zones to apply the Viaskin patches.
• Diagnosis of asthma that evolved to severe, unstable or uncontrolled asthma.

1. Provide written informed consent agreeing to all study procedures, before any study- specific procedures are done. 2. Male and female subjects 18 to 65 years of age, inclusive. 3. Subjects currently being evaluated for new or recurrent LN with a SOC kidney biopsy planned OR being evaluated for IgA nephropathy and with a SOC kidney biopsy planned. 4. Patients with LN must meet American College of Rheumatology (ACR) or Systemic Lupus Collaborating Clinics (SLICC) criteria for Systemic Lupus Erythematosus (SLE). 5. Subjects with a life expectancy >6 months. 1. Participation in another investigational study during same time period. 2. Contraindication to receiving a GBCA. 3. More than 2 previous lifetime exposures to a GBCA. 4. Any contraindication to MRI, including metal implants, claustrophobia or morbid obesity. 5. Acute or chronic severe renal insufficiency (glomerular filtration rate [GFR] <40 mL per minute per 1.73 m2). 6. Subject requiring dialysis. 7. Presence of pre-existing renal disease unrelated to SLE or IgA nephropathy, respectively. 8. Acute renal insufficiency of any severity caused by the hepato-renal syndrome. 9. Previous or pre-existing nephrogenic systemic fibrosis. 10. History of clinically significant anti-phospholipid syndrome. 11. Chronic liver function impairment, indicated by liver function tests (aspartate aminotransferase [AST], alanine aminotransferase [ALT]) >2-fold upper limit of normal. 12. Platelet count <50,000/μL. 13. Hemoglobin <8.0 g/dL. 14. History of or presence of central nervous system (CNS) disease such as active lupus cerebritis or multiple sclerosis that might compromise blood brain barrier function. 15. Infection that is clinically relevant, particularly hepatitis B virus (HBV), hepatitis C virus (HCV) and/or human immunodeficiency virus (HIV). 16. Pregnant or nursing females, or females not using effective contraception. 17. Inability or unwillingness to return to the research site clinic for study visits at baseline and at 6 months.

• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients with recurrent or refractory solid tumors, including non-Hodgkin lymphomas, histiocytoses (e.g. langerhans cell histiocytosis [LCH], juvenile xanthogranuloma [JXG], histiocytic sarcoma), and central nervous system (CNS) tumors are eligible; patients must have had histologic verification of malignancy at original diagnosis or relapse except in patients with intrinsic brain stem tumors, optic pathway gliomas, or patients with pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG); in cases where patient enrolls prior to histologic confirmation of recurrent disease, patient is ineligible and should be withdrawn from study if histology fails to confirm recurrence; please note: Patients with Hodgkin lymphoma and plexiform neurofibroma are not eligible
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must have an formalin-fixed paraffin-embedded (FFPE) tumor sample available for MATCH study testing from a biopsy or surgery that was performed at any point after initial tumor recurrence/progression, or be planned to have a procedure to obtain such a sample that is considered to be of potential benefit by the treating clinicians; a tumor sample from a clinically performed diagnostic (pre-treatment) biopsy will be acceptable for enrollment onto Pediatric MATCH only for children with high-grade gliomas of the brainstem (diffuse intrinsic pontine gliomas) or thalamus
• Please note: Samples that have been decalcified using standardly utilized acid-based decalcification methods are not generally suitable for MATCH study testing; the nucleic acids will have been degraded in the decalcification process
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age); note: neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: NOTE: patient does not need to meet all subprotocol criteria at time of enrollment onto the APEC1621SC screening protocol, but will need to meet all criteria prior to enrollment on any assigned treatment subprotocol. Patients must be enrolled onto a subprotocol within 12 weeks (84 days) of treatment assignment
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age); Note: neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of treatment with subprotocol specified therapy, the patients must have radiographically measurable disease; patients with neuroblastoma who do not have measurable disease but have iobenguane (MIBG) positive (+) evaluable are eligible; measurable disease in patients with CNS involvement is defined as tumor that is measurable in two perpendicular diameters on magnetic resonance imaging (MRI) and visible on more than one slice
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of enrollment onto a subprotocol, the following general criteria for initiation of therapy will be required:
• Patients must have fully recovered from the acute toxic effects of all prior anticancer therapy and must meet the following minimum duration from prior anticancer directed therapy prior to enrollment to the subprotocol; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anticancer agents known to be myelosuppressive: for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anticancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil counts [ANC]): >= 7 days after the last dose of agent; for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell infusions (with or without total-body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, NK cells, dendritic cells, etc.)
• X-ray therapy (XRT)/External Beam Irradiation including Protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation; note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: For patients with solid tumors without known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70ml/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age: 1 to < 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
• Age: 2 to < 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
• Age: 6 to < 10 years; maximum serum creatinine (mg/dL): male 1; female 1
• Age: 10 to < 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
• Age: 13 to < 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
• Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Serum glutamate pyruvate transaminase (SGPT) (alanine transferase [ALT]) =< 135 U/L (for the purpose of this study, the ULN for SGPT is 45 U/L)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients must be able to swallow intact capsules/tablets, unless otherwise specified in the subprotocol to which they are assigned
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Agent specific limitations on prior therapy will be included with specific treatment subprotocols
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies, or because there is currently no available information regarding human fetal or teratogenic toxicities; pregnancy tests must be obtained in females who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Concomitant medications
• Corticosteroids: at the time of consent and enrollment to regimen specific subprotocols, patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment to the subprotocol will not be eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Investigational drugs: patients must meet criteria for prior therapy at the time of consent and enrollment to a subprotocol; other investigational agents may not be administered to patients while they are receiving study drug as part of a subprotocol
• Anticancer agents: patients must meet criteria for prior therapy at the time of consent and enrollment to a subprotocol; other investigational agents may not be administered to patients while they are receiving study drug as part of a subprotocol
• Anti-GVHD agents post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have an uncontrolled infection are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have had a prior solid organ transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Additional agent specific criteria will be included with specific treatment subprotocols

Key
• Male or female from 1-3 years of age;
• Physician-diagnosed peanut allergy;
• Peanut-specific IgE level > 0.7 kU/L;
• Positive peanut SPT with a largest wheal diameter ≥ 6 mm;
• Positive DBPCFC at ≤ 300 mg peanut protein; Key
• Uncontrolled asthma;
• History of severe anaphylaxis to peanut;
• Prior immunotherapy to any food or other immunotherapy;
• Generalized severe dermatologic disease;

Inclusion Criteria Each prospective subject must meet ALL of the following inclusion criteria in order to be eligible for this study: 1. Signed written informed consent granted prior to initiation of any study-specific procedures. 2. 18 years of age and older. 3. For the dose escalation cohorts, relapsed or refractory subjects with a diagnosis of B-cell NHL, CLL/SLL and WM who have received at least two prior systemic therapies . Subjects must have failed or are intolerant to standard therapies and cannot be a candidate for standard salvage regimens. Subjects with low grade lymphoma must be progressing and requiring treatment.. 4. For the expansion cohorts, the following criteria must be met:
• Cohort A: Relapsed/Refractory (R/R) CLL/SLL subjects with at least 2 prior systemic therapies and previously treated with a covalent BTKi who must have a documented BTK mutation on C481 residue
• Cohort B: R/R CLL/SLL subjects who have failed or were intolerant to a BTKi with documentation of the absence of BTK mutation on C481 residue. In this study, intolerance to standard therapy is defined as having experienced a grade 3 or higher adverse event that was caused by the standard therapy and resulted in treatment discontinuation.
• Cohort C: Richter's transformation subjects who have failed at least one prior therapy
• Cohort D: Follicular Lymphoma (FL) subjects who have failed at least 2 prior systemic therapies and are histology grade 1, 2, or 3A
• Cohort E: Mantle Cell Lymphoma (MCL) subjects who have failed at least 2 prior systemic therapies
• Cohort F: Marginal Zone Lymphoma (MZL) subjects who have failed at least 2 prior systemic therapies
• Cohort G: High-grade B-cell lymphoma subjects who have failed at least 2 prior systemic therapies and have known MYC and BCL2 and/or BCL6 translocations
• Cohort H: Waldenström macroglobulinemia (WM) subjects who have failed at least 2 prior systemic therapies 5. Disease status requirement: 1. For CLL subjects, symptomatic disease that mandates treatment (Hallek et al. 2018). 2. For B-cell NHL subjects, measurable disease by imaging scan. 3. For WM, serum immunoglobulin M (IgM) with a minimum IgM level of ≥ 2 times the upper limit of normal (ULN). 6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2. 7. Good organ function 1. Creatinine clearance of ≥ 60 mL/min as estimated by the Cockcroft-Gault equation or by 24-hour urine collection. 2. Total bilirubin ≤ 1.5 x institutional ULN (total bilirubin of ≤ 3 x institutional ULN in subjects with documented Gilbert's syndrome). 3. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × institutional ULN. 4. Platelet count ≥ 50,000/µL 5. Absolute neutrophil count (ANC) ≥ 1000/µL. 6. Hemoglobin (Hgb) ≥ 8.0 g/dL, stable for ≥ 1 week. 8. For men and women of child-bearing potential, willing to use adequate contraception (e.g., latex condom, cervical cap, diaphragm, abstinence, etc.) for the entire duration of the study. 9. Female subjects of child-bearing potential must have a negative serum pregnancy test within 14 days of the first day of drug dosing. 10. Ability to swallow oral medications without difficulty. Exclusion Criteria Potential subjects who meet ANY of the following exclusion criteria are not eligible for enrollment into this study: 11. Had immunotherapy, radiotherapy, radioimmunotherapy, biological therapy, chemotherapy, or treatment with an investigational product within 5 half-lives or four weeks (whichever is shorter) prior to treatment initiation, or oral therapy within 5 half-lives or one week (whichever is shorter) prior to treatment initiation. 12. Transformation of FL to a more aggressive subtype of lymphoma or grade 3b FL 13. Subjects currently being treated with the following drugs: 1. CYP 2C9 substrates with a narrow therapeutic index (such as warfarin, phenytoin) 2. CYP 2C8 substrates with a narrow therapeutic index (such as paclitaxel) 3. CYP 2C19 substrates with a narrow therapeutic index (such as S-mephenytoin) 4. CYP 2D6 substrates with a narrow therapeutic index (such as thioridazine, pimozide) 5. P-gp substrates with a narrow therapeutic index (such as digoxin) Note: A washout period of at least 5 times the half-life after the last dose of any of the above treatments is required for a subject to be eligible for study enrollment. 14. Prior allogeneic bone marrow transplant. 15. Active central nervous system (CNS) involvement. 16. Pregnant or breast-feeding women. 17. Has significant, ongoing co-morbid conditions which would preclude safe delivery of the study drug. 18. Uncontrolled illness including but not limited to ongoing or active infection, symptomatic congestive heart failure (New York Heart Association [NYHA] Class III or IV heart failure), unstable angina pectoris, cardiac arrhythmia, cardiac infarction in the past six months, and psychiatric illness that would limit compliance with study requirements. 19. QTc prolongation (defined as a QTc > 450 msecs) or other significant electrocardiogram (ECG) abnormalities including 2nd degree atrioventricular (AV) block type II, 3rd degree AV block, or bradycardia (ventricular rate less than 50 beats/min). If the screening ECG has a QTc > 450 msecs, the ECG can be submitted for a centralized, cardiologic evaluation. 20. Active human immunodeficiency virus (HIV) infection, Hepatitis B, or Hepatitis C infection. 21. Other medical or psychiatric illness or organ dysfunction which, in the opinion of the Investigator, would either compromise the subject's safety or interfere with the evaluation of the safety of the study agent. 22. History of prior cancer within < 1 year, except for basal cell or squamous cell carcinoma of the skin, cervical cancer in situ or other in situ carcinomas.

1. Infants with SCID, either typical or leaky or Omenn syndrome. 1. Typical SCID is defined as either of the following
• Absence or very low number of T cells (CD3+ T cells <300/microliter AND no or very low T cell function (<10% of lower limit of normal) as measured by response to phytohemagglutinin OR
• Presence of maternally derived T cells 2. Leaky SCID is defined as the following • Absence of maternally derived T cells • AND either one or both of the following (i, ii): i) <50% of lower limit of normal T cell function as measured by response to PHA OR <30% of lower limit of normal T cell function as measured by response to CD3 ii) Absent or <10% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens (must document post vaccination or exposure for this criterion to apply) • AND at least two of the following (i through iii): i) CD3 T cells < 1500/microliter ii) >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (at < 4 years of age) AND/OR are oligoclonal T iii) Low TRECs and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower level of normal. 3. Omenn syndrome • Generalized skin rash
• Maternal lymphocytes tested for and not detected.
• >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (<2 years of age)
• Absent or low (up to 30% lower limit of normal (LLN)) T cell proliferation to antigens (Candida, tetanus) to which the patient has been exposed IF: Proliferation to antigen was not performed, but at least 4 of the following 8 supportive criteria, at least one of which must be among those marked with an asterisk (*) below are present, the patient is eligible as Omenn Syndrome. 1. Hepatomegaly 2. Splenomegaly 3. Lymphadenopathy 4. Elevated IgE 5. Elevated absolute eosinophil count 6. *Oligoclonal T cells measured by CDR3 length or flow cytometry (upload report) 7. *Proliferation to PHA is reduced to < 50% of lower limit of normal (LLN) or SI < 30 8. *Low TRECs and/or percentage of CD4+/RA+ CD31+ or CD4+/RA+ CD62L+ cells below the lower level of normal 2. Documented mutation in one of the following SCID-related genes a. Cytokine receptor defects (IL2RG, JAK3) b. T cell receptor rearrangement defects (RAG1, RAG2) 3. No available genotypically matched related donor (sibling) 4. Availability of a suitable donor and graft source 1. Haploidentical related mobilized peripheral blood cells 2. 9/10 or 10/10 allele matched (HLA-A, -B, -C, -DRB1, -DQB1) volunteer unrelated donor mobilized peripheral blood cells 5. Age 0 to 2 years at enrollment Note: to ensure appropriate hepatic metabolism, age at time of busulfan start: For IL2RG/JAK3: 8 weeks For RAG1/RAG2: 12 weeks 6. Adequate organ function defined as: 1. Cardiac: Left ventricular ejection fraction (LVEF) at rest ≥ 40% or, shortening fraction (SF) ≥ 26% by echocardiogram. 2. Hepatic: Total bilirubin < 3.0 x the upper limit of normal (ULN) for age (patients who have been diagnosed with Gilbert's Disease are allowed to exceed this limit) and AST and ALT < 5.0 x ULN for age. 3. Renal: GFR estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is < 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be > 50 mL/min/1.73 m2. 4. Pulmonary No need for supplemental oxygen and O2 saturation > 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care). 1. Presence of any serious life-threatening or opportunistic infection at time of enrollment and prior to the initiation of the preparative regimen. Serious infections as defined below that occur after enrollment must be reported immediately to the Study Coordinating Center, and enrollment will be put on hold until the infection resolves. Ideally enrolled subjects will not have had any infection. If patients have experienced infections, these must have resolved by the following definitions: a. Bacterial i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site must be negative and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). ii. Tissue-based clinical infection (e.g. cellulitis): Complete resolution of clinical signs (e.g. erythema, tenderness, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). iii. Pneumonia, organism not identified by bronchoalveolar lavage: Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). If possible, radiographic resolution should also be demonstrated. b. Fungal i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site is negative and patient has completed appropriate course of antifungal therapy (typically at least 14 days). The patient may be continued on antifungal prophylaxis following completion of the treatment course. c. Pneumocystis i. Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of therapy (typically at least 21 days). If possible, radiographic resolution should also be demonstrated. The patient may be continued on prophylaxis following completion of the treatment course. d. Viral i. Viral PCRs from previously documented sites (blood, nasopharynx, CSF) must be re-tested and are negative. ii. If re-sampling a site is not clinically feasible (i.e. BAL fluid): Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.). If possible, radiographic resolution should also be demonstrated. 2. Patients with HIV or HTLV I/II infection will be excluded.

• Diagnosed with cutaneous lupus erythematosus and/or systemic lupus erythematosus by clinical, laboratory, and histopathological findings
• Ability to speak and read English or Spanish at a 6th grade reading level (a translator will be available with additional consent forms in Spanish)
• Ability to give written informed consent
• Less than 18 years of age, since the characteristics of the disease in these subjects could be very different
• Due to a medication, in which its discontinuation results in the resolution of cutaneous lupus, since the characteristics of the disease in these subjects could be very different
• Medical conditions who do not warrant a skin biopsy
• Unable to give written, informed consent or undergo a skin biopsy and/or venipuncture for any other reason

Strata A, B, and C (Part 1
•Retrospective Study)-
• Individuals with Severe Combined Immune Deficiency (SCID) diagnosis who: --were treated at a location participating in this consortium from 1968 until present, and --are not enrolled in RDCRN PIDTC-6901 (ClinicalTrials.gov ID: NCT01186913).
• Subjects who received HCT/GT/ERT prior to the present date are eligible for the retrospective study. The enrollment criteria for subjects who died prior to definitive therapy are the same as for Strata A, B and C. Stratum A, Typical SCID:
• Individuals who meet the following inclusion criteria and who received HCT are eligible for enrollment into Stratum A of the study:
• Absence or very low number of T cells (CD3 T cells < 300/microliter), and no or very low T cell function (< 10% of lower limit of normal) as measured by response to phytohemagglutinin (PHA) or cells of maternal origin present.
• If maternal cells are present but the patient does not meet criteria for very low T cell function as defined, the assigned reviewers for the potential subject, and if necessary, the full PID‐SCID RP will review the laboratory report to determine if criteria of maternal engraftment are met for Protocol 6902.
• Laboratory report of testing for maternal engraftment is required, for evaluation by the PID‐SCID RP. Stratum B, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis: Individuals who meet the following criteria are eligible for enrollment into Stratum B of the study: Leaky SCID-
• Maternal lymphocytes tested for and not detected and,
• Either one or both of the following (a,b): a) < 50% of lower limit of normal T cell function (as measured by response to PHA OR < 50% of lower limit of normal T cell function as measured by response to CD3/CD28 antibody, b) Absent or < 30% lower limit of normal proliferative responses to candida and tetanus toxoid antigens postvaccination or exposure,
• AND at least one of the following (a through e): 1. Reduced number of CD3 T cells, 2. > 80% of CD3+ or CD4 T cells are CD45RO+,
• AND/OR >80% of CD3+ or CD4+ T cells are,CD62L negative,
• AND/OR >50% of CD3+ or CD4+ T cells express HLA‐DR (at < 4 years of age),
• AND/OR are oligoclonal T cells. c) Hypomorphic mutation in IL2RG in a male, or homozygous hypomorphic mutation or compound heterozygosity with at least one hypomorphic mutation in an autosomal SCID‐causing gene. d) Low TRECs and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower limit of normal. e) Functional testing in vitro supporting impaired, but not absent, activity of the mutant protein,
• AND does not meet criteria for Omenn Syndrome,
• AND does not have known selective loss of lymphocytes, Ataxia‐ Telangiectasia, or congenital heart defect associated with lymphopenia, unless a SCID genotype is also present. Omenn Syndrome (OS):
• Generalized skin rash,
• Maternal engraftment tested for and not detected,
• Absent or low (up to 30% of normal) T cell proliferation to antigens to which the patient has been exposed.
• If the proliferation to antigen was not performed, but at least 4 of the following 10 supportive criteria, at least one of which must be among those marked with an asterisk (*) are present, the patient is eligible: hepatomegaly; splenomegaly; lymphadenopathy; elevated IgE; elevated absolute eosinophil count; *oligoclonal T cells measured by CDR3 length or flow cytometry >80% of CD4+ T cells are CD45RO+ ;*proliferation to PHA is reduced <50% of lower limit of normal or SI <30; *proliferative response in mixed leukocyte reaction is reduced to increment cpm < 20% or SI <20; hypomorphic mutation to SCID causing gene; low TRECs and/or percentage of CD 4+/ RA+/CD31+; or CD4+/RA+/CD62L+ cells below the lower limit of normal. Reticular Dysgenesis (RD):
• Absence or very low number of T cells (CD3 T cells <300/microliter),
• No or very low (<10% of lower limit of normal) T cell function (as measured by response to phytohemagglutinin (PHA),
• Severe congenital neutropenia (absolute neutrophil count <200/microliter),
• AND at least one of the following:
• Sensorineural deafness and/or absence of granulopoiesis at bone marrow examination and/or a deleterious AK2 mutation,
• absence of granulopoiesis on bone marrow examination; a pathogenic mutation in the adenylate kinase 2 (AK2) gene identified. Stratum C, SCID with Non-HCT Treatments: -Individuals who meet the following criteria and were treated with PEG-ADA or gene therapy with autologous modified cells are eligible for enrollment into Stratum C (SCID with non-HCT treatments) of the study-
•Any SCID patient previously treated with a thymus transplant (includes intention to treat with HCT, as well as PEG‐ADA ERT or gene therapy). Strata A, B, and C (Part 2
•Cross-Sectional Study): Patient inclusion criteria for the cross sectional study: Eligibility for Strata A, B and C are the same as for the retrospective study except that all the patients in the cross-sectional study are currently surviving and are at least 2 years post the most recent class of therapy. Parts 1 and 2
•Retrospective and Cross-Sectional Studies -
• Lack of appropriate testing to rule out HIV infection after 1997 (p24 antigen or more sensitive) or other cause of secondary immunodeficiency,
• Presence of DiGeorge syndrome,
• Most patients with other PIDs such as nucleoside phosphorylase deficiency, ZAP70 deficiency, CD40 ligand deficiency, NEMO deficiency, XLP, cartilage hair hypoplasia or ataxia telangiectasia will not meet the inclusion criteria for Stratum A, B, or C above; however, a patient with one of the above may meet the inclusion criteria for Stratum B and if so will be included-
• MHC Class I and MHC Class II antigen deficiency are excluded,
• Metabolic conditions that imitate SCID or related disorders such as folate transporter deficiency, severe zinc deficiency, transcobalamin deficiency.

• Must have MGFA MG grades 2, 3, or 4 generalized MG, according to the MGFA classification system
• Elevated AChR or MuSK Ab
• Patient's signs and symptoms should not be better explained by another disease process
• IVIg maintenance dose of 0.2 to 2 gm/kg/4 weeks or equivalent dose administered every 2-4 weeks
• Stable IVIg for at least 3 cycles
• Able to complete the study and return for follow-up visits
• Able to give written informed consent before participating in the study
• History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue
• Other major chronic or debilitating illnesses within six months prior to study entry
• Female patients who are premenopausal and are (a) pregnant, (b) breastfeeding, or (c) not using an effective method of double barrier birth control
• Altered levels of consciousness, dementia, or abnormal mental status
• Thymectomy in the previous three months
• History of renal insufficiency or liver disease
• Skin disease that would interfere with assessment of injection site reaction
• History of severe reactions to IVIg or SCIg
• Participation in a research study within the last 3 months
• Treatment with rituximab or other biologics within 12 months of study entry
• Unable to provide informed consent

• Participant Inclusion Criteria (Part 1
•Longitudinal Analysis)
• CGD Patients Undergoing Transplant 1995 to Present with Birth Year In or After 1988 1. CGD Patients will be Defined by both Defective Neutrophil NADPH Oxidase Function and by Clinical History Consistent with CGD Patients must have both of: A functional assay demonstrating abnormal NADPH oxidase function (see A below); AND Clinical history consistent with CGD (see B below). ************************************************************************* Patients must have both "A" and "B": A. Function: Assays of NADPH Oxidase Function I. Dihydrorhodamine (DHR) Assay:
• Blood sample was obtained at a time when patient was clinically stable and not critically ill, with control samples performed simultaneously indicating a qualified assay; and
• Assay unequivocally demonstrates CGD with an stimulation index (SI) SI < 35 or equivalent. Assay report, including mean fluorescence intensity (MFI) from unstimulated and stimulated samples and gating strategy, must be de-identified and provided. OR II. Nitroblue Tetrazolium Oxidation Test (NBT): o Diagnostic of CGD (reported as reduced granulocyte oxidative response). Report must be de-identified and provided. AND B. Clinical History: One or More of the Following:
• Severe and/or recurrent infection (liver, perirectal or lung abscess; pneumonia; adenitis; or osteomyelitis) due to, for example, Staphylococcus aureus, Burkholderia sp, Serratia marcescens, non-albicans Candida sp, Aspergillus sp or other mold; or Nocardia sp or other deep tissue infection characteristic of CGD
• Sterile granulomatous disease in respiratory, gastrointestinal or urogenital tracts; or Crohn's disease-like colitis
• A family history consistent with either X-linked or autosomal recessive CGD In cases where either functional assay (A) or history (B) is equivocal, one or more of the following may be used to confirm a diagnosis of CGD: C. Absent or significantly reduced in expression or abnormal size of any of the 5 phox components (gp91 phox, p47 phox, p22 phox, p67phox, and p40phox) of NADPH oxidase, by either:
• Western blot
• Northern blot OR D. Mutation in a gene encoding one of the 5 phox components (gp91 phox, p47 phox, p22 phox, p67 phox, and p40 phox) of NADPH oxidase that is predictive of a decreased or absent oxidative burst. (Nonsense, frameshift, or previously described missense mutation associated with CGD). Molecular Diagnosis is Desirable In addition, molecular diagnosis (gene sequencing and expression analysis) of CGD is desirable and should be performed when possible. 2. Further Characterization of Oxidase Level, Longitudinal Study, Prospective Cohort Patients who are to undergo transplantation during the study period must be further characterized as oxidase-null or oxidase positive by level of oxidase production by either:
• DHR assay stimulation Index: where SI ≤ 2.5 will be classified as oxidase-null CGD. Those with SI > 2.5 will be classified as oxidase positive CGD. A single validated test that is accepted by the PID-CGD Review Panel is adequate, but testing on two occasions for validation is desirable. OR
• Ferricytochrome C reduction assay of granulocytes with O2 < 2.3 nmoles /106 cells/h classified as oxidase-null CGD. A single validated test that is accepted by the PID-CGD Review Panel is adequate, but testing on two occasions for validation is desirable. OR o Genetic sequencing reporting a mutation that is unequivocally associated to absent oxidase production. (e.g. null mutations) will be classified as oxidase-null CGD (See discussion in Appendix I for how family history, genotype and CGD mutation information will be applied to assigning patients lacking any quantitative oxidase activity measurements to residual oxidase-null or residual oxidase-positive groups). 3. Longitudinal Study, Retrospective Cohort Patients who have already been transplanted will be included regardless of whether further characterization by oxidase level (or genotype/mutation data) is possible or not.
• Non-Transplanted CGD Patients with Birth Year In or After 1988 A non-transplant (conventional therapy) group of CGD subjects will be enrolled in the longitudinal study. The non-transplant subjects will be selected from the potentially eligible (retrospective) patient cohort with diagnosis of CGD treated with conventional non-transplant therapy. Participating sites will enter their entire retrospective cohort of CGD patients having birth year in or after 1988 into the registration cohort for this protocol. Baseline for both non-transplant subjects and HCT subjects for the purpose of comparing survival will be the year of birth. However, for non-transplant subjects, many of the detailed analyses such as infection and autoimmune complication rates will be assessed in the year preceding the date of last contact.
• Participant Inclusion Criteria (Part 2
•Cross-Sectional Analysis) To participate in the Cross-Sectional Analysis, patients must have previously been enrolled into the Longitudinal Analysis of Protocol 6903. All transplanted subjects in the Cross-Sectional Analysis are surviving and shall have at least 3 years of follow-up post-transplant to be included. Non-transplanted CGD subjects will become eligible for consideration for the Cross-Sectional Analysis if they were eligible and enrolled in the retrospective cohort of the Longitudinal Analysis, and if/when they are > 3 years post-diagnosis of CGD. Provision of written informed consent will be required for inclusion in the Cross-Sectional Analysis.
• Participant Exclusion Criteria (Longitudinal and Cross- Sectional Analyses)
• Presence of other primary immunodeficiency syndromes that do not meet the clinical and laboratory criteria for CGD.
• Rac2 Deficiency
• Myeloperoxidase Deficiency (MPO Deficiency)
• Glutathione deficiency
• Leukocyte adhesion deficiency syndrome
• Non-transplant subjects:
• The above exclusions pertain.
• In addition, non-transplant subjects will be excluded if the only assessment of oxidase function available is the nitroblue tetrazolium (NBT) test (a non-quantitative test).

1. Age between 18 and 60 years, inclusive. 2. Diagnosis of relapsing forms of MS using revised McDonald Criteria1. 3. Expanded Disability Status Scale (EDSS) 0
•5.5 (note: functional system changes in cerebral (or mental) functions and in bowel and bladder functions not used in determining EDSS for protocol eligibility). 4. Has had a minimum of 12 monthly doses of continuous natalizumab therapy (300 mg/d). 5. Understands English, and gives informed consent. 1. Natalizumab failure based on clinician's discretion. 2. Any prior exposure to alemtuzumab. 3. Progressive MS. 4. A diagnosis of Progressive multifocal leukoencephalopathy (PML). 5. Known hypersensitivity to alemtuzumab. 6. Initiation of new immunosuppressant treatment after the subject becomes protocol-eligible (except for corticosteroids) or enrollment in a concurrent trial with immuno-active pharmacotherapies. 7. Uncontrolled diabetes mellitus defined as HbA1c > 8% and/or requiring intensive management. 8. History of cytopenia consistent with the diagnosis of myelodysplastic syndrome. 9. Clinically significant autoimmune disease other than MS that may affect the CNS, including neuromyelitis optica (NMO), systemic lupus erythematosus (SLE), or Behcet disease. 10. Active hepatitis B or C infection or evidence of cirrhosis. 11. HIV positivity. 12. Uncontrolled viral, fungal, or bacterial infection. 13. Positive pregnancy test or inability or unwillingness to use effective means of birth control. Effective birth control is defined as: 1. Refraining from all acts of vaginal intercourse (abstinence), 2. Consistent use of birth control pills, 3. Tubal sterilization or male partner who has undergone vasectomy 4. Placement of intrauterine device 5. Use, with every act of intercourse, of a diaphragm with contraceptive jelly and/or condoms with contraceptive foam. 14. Presence of metallic objects implanted in the body that would preclude the ability of the subject to safely have MRI exams. 15. Psychiatric illness, mental deficiency, or cognitive dysfunction making compliance with treatment or informed consent impossible.

• Age < 1 year at the time of surgery
• Undergoing heart surgery with CPB as part of standard clinical care
• Availability and willingness of the parent/legally authorized representative to provide written informed consent
• < 37 weeks adjusted gestational age at time of surgery
• Any oral or intravenous steroid treatment within two days of surgery
• Any patient receiving any of the following medications within 2 days of surgery: Amphoteracin B, aminoglutethimide, anticholesterases, warfarin, P450 3A4 inducers including (but not limited to) carbamazepine, phenobarbital, phenytoin, rifampin, bosentan and nafcillin or P450 3A4 inhibitors including (but not limited to) clarithromycin, voriconazole, itraconazole, ketoconazole, ciprofloxacin, diltiazem, fluconazole, erythromycin and verapamil.
• Infection contraindicating steroid use
• Preoperative mechanical circulatory support or active resuscitation at the time of randomization
• Emergent surgery precluding steroid administration 8-12 hours before surgery

1. Diagnosis of Multiple Sclerosis , ADEM, anti-MOG antibody associated CNS demyelination 2. Age 12 to 18 inclusive at time of enrollment 3. Ability of parent or legal guardian to provide informed consent if participant is under 18. 4. Ability of patients age 12 and older to give assent 5. Completion of the signed HIPPA authorization form by a parent or legal guardian or by participants (18 years of age). 1. Known history of traumatic brain injury that required medical care 2. Non-English speaking (based on standardized neuropsychological testing and questionnaires) 3. Claustrophobic, pregnant, the presence of metallic braces, implants or medical devices that are unsafe at 3T or 7T and/or interfere with the MRI/MRS signals

• Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment
• Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment
• Pediatric and adult patients of any age
• Patients who are receiving only licensed CBUs
• Cord blood transplant recipients at international transplant centers
• Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
• Patients whose selected unlicensed CBU(s) will be more than minimally manipulated

Stratum A: Typical SCID (formerly referred to as Classic SCID)- -Subjects who meet the following inclusion criteria and the intention is to treat with allogeneic hematopoietic cell transplant (HCT) are eligible for enrollment into Stratum A (Typical SCID) of the study:
• Absence or very low number of T cells (CD3 T cells <300/microliter) AND
• No or very low T cell function (<10% of lower limit of normal) as measured by response to phytohemagglutinin (PHA) OR
• T cells of maternal origin present. Stratum B: Leaky SCID, Omenn Syndrome, Reticular Dysgenesis- -Subjects who meet the following criteria and the intention is to treat with HCT are eligible for enrollment into Stratum B: Leaky SCID:
• Maternal lymphocytes tested for and not detected AND
• Either one or both of the following (a,b) :
• a.) <50% of lower limit of normal T cell function as measured by response to PHA, OR response to anti-CD3/CD28 antibody
• b.) Absent or <30% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens
• AND at least two of the following (a through e):
• a.) Reduced number of CD3 T cells
• age ≤2 years: <1500/microliter
• age >2 years and ≤4 years: <800/microliter
• age >4 years: <600/microliter
• b.) ≥80% of CD3+ or CD4+ T cells that are CD45RO+
• AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative
• AND/OR >50% of CD3+ or CD4+T cells express HLA-DR (at <4 years of age)
• AND/OR are oligoclonal T cells
• c.) Hypomorphic mutation in IL2RG in a male, or homozygous hypomorphic mutation or compound heterozygosity with ≥1 hypomorphic mutation in an autosomal SCID-causing gene
• d.) Low T Cell Receptor Excision Circles (TRECs) and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower limit of normal.
• e.) Functional testing in vitro supporting impaired, but not absent, activity of the mutant protein, AND
• Does not meet criteria for Omenn Syndrome. Omenn Syndrome:
• Generalized skin rash
• Maternal lymphocytes tested for and not detected; --Note: If maternal engraftment was not assessed and ruled out, the subject is not eligible as Omenn Syndrome.
• ≥80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR
• 80% of CD3+ or CD4+T cells are CD62L negative AND/OR
• 50% of CD3+ or CD4+ T cells express HLA-DR (at <2 years of age);
• Absent or low (< 30% lower limit of normal) T cell proliferation response to antigens (Candida, tetanus) to which the subject has been exposed NOTE: If proliferation to antigen was not performed, but at least 4 of the following 9 supportive criteria, at least one of which must be among those marked with an asterisk (*) below are present, the subject is eligible as Omenn Syndrome:
• Hepatomegaly
• Splenomegaly
• Lymphadenopathy
• Elevated IgE
• Elevated absolute eosinophil count
• *Oligoclonal T cells measured by CDR3 length or flow cytometry
• *Proliferation to PHA is reduced <50% of lower limit of normal or SI <30
• *Hypomorphic mutation in a SCID causing gene
• Low TRECS and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower limit of normal. Reticular Dysgenesis:
• Absence or very low number of T cells (CD3 <300/µL
• No or very low (<10% lower limit of normal) T cell response to PHA
• Severe neutropenia (absolute neutrophil count < 200 /µL) AND
• ≥2 of the following (a,b,c):
• a.) Sensori-neural deafness
• b.) Deficiency of marrow granulopoiesis on bone marrow examination
• c.) A pathogenic mutation in the adenylate kinase 2 (AK2) gene identified. Stratum C: Subjects who meet the following criteria and the intention is to treat with therapy other than allogeneic HCT, primarily PEG-ADA ERT or gene therapy with autologous modified (gene transduced) cells, are eligible for enrollment into Stratum C:
• ADA Deficient SCID with intention to treat with PEG-ADA ERT
• ADA Deficient SCID with intention to treat with gene therapy
• X-linked SCID with intention to treat with gene therapy
• Any SCID patient previously treated with a thymus transplant (includes intention to treat with HCT, as well as PEG-ADA ERT or gene therapy)
• Any SCID patient who received therapy for SCID deemed "non-standard" or "investigational", including in utero procedures. -Subjects who meet any of the following exclusion criteria are disqualified from enrollment in Strata A, B, or C of the study:
• Presence of an Human Immunodeficiency Virus (HIV) infection (by PCR) or other cause of secondary immunodeficiency
• Presence of DiGeorge syndrome
• MHC Class I and MHC Class II antigen deficiency, and
• Metabolic conditions that imitate SCID or related disorders such as folate transporter deficiency, severe zinc deficiency or transcobalamin deficiency.

General
• For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of <1 percent (%) per year during the treatment period and for at least 24 weeks after the last dose of study treatment or until their B-cells have repleted, whichever is longer Inclusion Criteria Specific to RMS Participants:
• Diagnosis of RMS in accordance with the 2010 revised McDonald criteria
• Expanded Disability Status Scale (EDSS) score of 0 to 5.5 points, inclusive, at Screening
• Disease duration from the onset of multiple sclerosis symptoms less than (<) 15 years in participants with an EDSS score greater than (>) 5.0 at Screening
• Either treatment-naive or receiving treatment with disease-modifying therapies, including prior use of interferon (IFN)-beta-1a (Avonex®, Rebif®), IFN-beta-1b (Betaseron®/Betaferon), or glatiramer acetate (Copaxone®).
• At least one clinically documented relapse in the past year and/or at least one T1-weighted Gadolinium (Gd)-enhancing lesion in the past year and/or at least one new T2 lesion in the past year at the time of enrollment Inclusion Criteria Specific to RMS Cohort Arm 4 Participants:
• Must meet inclusion criteria for the RMS cohort
• Separate signed Informed Consent Form for the RMS Delayed Time to Start Control Arm (Arm 4)
• Must be willing to remain on the same dose and regimen of current standard of care, or no treatment if treatment-naïve, for 12 weeks after study enrollment The treating and/or study physician must agree that the participant is eligible to remain on the same dose and regimen of their current standard of care at Screening, or to receive no treatment if the participant is treatment-naïve, for 12 weeks after study enrollment Inclusion Criteria Specific to PPMS Participants:
• Diagnosis of PPMS in accordance with the 2010 revised McDonald criteria
• EDSS score of 3.0
•6.5 points, inclusive, at Screening
• Disease duration from the onset of multiple sclerosis symptoms <10 years in participants with an EDSS at Screening less than or equal to ( • Documented history of either elevated immunoglobulin G (IgG) Index or one or more IgG oligoclonal bands (OCBs) detected by isoelectric focusing
• Diagnosis of secondary progressive multiple sclerosis without relapses for at least 1 year
• History or known presence of recurrent or chronic infection (human immunodeficiency virus [HIV], syphilis, tuberculosis)
• History of cancer, including solid tumors and hematological malignancies (except basal cell, in situ squamous cell carcinomas of the skin, and in situ carcinoma of the cervix of the uterus that have been excised and resolved with documented clean margins on pathology)
• Known presence or history of other neurologic disorders
• Contraindications or intolerance to oral or IV corticosteroids, including IV methylprednisolone, according to the country label
• Contraindication for LP
• Previous treatment with B cell-targeted therapies (such as rituximab, ocrelizumab, atacicept, belimumab, or ofatumumab)
• Previous treatment with natalizumab/Tysabri®, alemtuzumab, anti-CD4 agents, cladribine, teriflunomide, cyclophosphamide, mitoxantrone, azathioprine, mycophenolate mofetil, cyclosporine, methotrexate, total body irradiation, or bone marrow transplantation
• Treatment with fingolimod/Gilenya®, dimethyl fumarate/Tecfidera®, or similar treatment within 6 months prior to enrollment
• Systemic corticosteroid therapy within 4 weeks prior to Baseline
• Previous or concurrent treatment with any investigational agent or treatment with any experimental procedure for multiple sclerosis (such as treatment for chronic cerebrospinal venous insufficiency)
• Certain laboratory abnormalities or findings at Screening
• Pregnant or lactating, or intending to become pregnant during the study Exclusion Criteria Specific to RMS Participants: Diagnosis of PPMS or secondary progressive multiple sclerosis without relapses

1. Patient must have a clinical diagnosis of morphea confirmed by the primary investigator and by histopathological examination. 2. Ages 0-90 years old 3. Children must weigh more than 20 lbs. in order to satisfy Children's Medical Center policy for the maximum amount of blood drawn in a 24 hour period. 4. Patient or legal guardian must be able to speak and read at a 6th grade reading level. 5. Both male and female patients will be eligible 6. All races and ethnic backgrounds will be included 7. Relationships to proband: All patients with morphea will be included. A patient's family history will be reviewed and if there is a family history of morphea or systemic sclerosis then we will give the study patient the investigator's contact information and ask the family member to call the study team to answer any questions and enroll them in the study if they choose to do so. 8. Ability to give informed consent: Patients must be able to give informed consent or they will give assent with parent or guardian consent as a minor to be a part of the morphea registry.
•Patients who have been coded as morphea (701.0), but do not have morphea/localized scleroderma (examples: steroid atrophy, acquired keratoderma, keloids, nephrogenic fibrosing dermopathy, systemic sclerosis, lichen sclerosis)

Key
•Patients with multiple sclerosis (MS) who are newly initiating treatment with dimethyl fumarate (DMF) under routine clinical care are eligible to participate in the study. Key
• Patients with previous exposure to dimethyl fumarate (DMF), Fumaderm (fumaric acid esters), or compounded fumarates.
• Patients participating in other clinical studies. NOTE: Other protocol-defined inclusion/exclusion criteria may apply.