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Does Caudal Block Increase the Incidence of Urethrocutaneous Fistula Formation Following Hypospadias Repair in Infants?
This is a prospective randomized multi-center non-inferiority trial conducted through the Pediatric Regional Anesthesia Network study sites to determine if caudal block increases the incidence of urethrocutaneous fistula following distal or mid shaft hypospadias repair compared with penile nerve block.
4 Months to 2 Years old
• infants/ children with midshaft or distal hypospadias undergoing primary single stage repair in one of the Pediatric Regional Anesthesia Network participating centers.
• prior hypospadias surgery,
• proximal or penoscrotal hypospadias,
• abnormal caudal anatomy or spinal dysraphism,
• cyanotic congenital heart disease,
• infection or rash at the block injection site.
Drug: Caudal block with ropivacaine, Drug: penile nerve block with bupivacaine
Hypospadias, Urethrocutaneous Fistula
hypospadias, caudal, urethrocutaneous fistula, penile nerve block
Phase 3 Alogliptin Pediatric Study
This study will evaluate the efficacy of alogliptin 25 milligram (mg) once daily compared to placebo when administered as monotherapy, or when added onto a background of metformin alone, insulin alone, or a combination of metformin and insulin, as measured by the glycosylated hemoglobin (HbA1c) change from Baseline at Week 26 in pediatric participants with type 2 diabetes mellitus (T2DM).
10 Years to 17 Years old
Inclusion Criteria:1. Has a confirmed diagnosis of T2DM using American Diabetes Association (ADA) and World Health Organization (WHO) criteria (laboratory determinations of fasting plasma glucose [FPG] greater than or equal to [>=] 126 mg/dL, random glucose >=200 mg/dL [>=11.10 mmol/L], HbA1c >=6.5 percent (%), or 2-hour oral glucose tolerance test [OGTT] glucose >=200 mg/dL), documented in the participants' medical record. 2. Is thought to be able to swallow the tablet containing the study medication. 3. The participant and/or his/her legal representative (that is, parents or legal guardians) are able and willing to monitor their own blood glucose concentrations with a home glucose monitor and complete participant diaries.
Exclusion Criteria:1. Has a history of hypersensitivity or allergy to alogliptin, other dipeptidyl peptidase-4 (DPP-4) inhibitors, metformin, insulin or related compounds. 2. Has a confirmed diagnosis of type 1 diabetes mellitus or maturity-onset diabetes of the young (MODY). 3. Has a hemoglobin level <11.0 gram per deciliter (g/dL) (<110 gram per liter [g/L]) for males and <10.0 g/dL (<100 g/L) for females. 4. Has a history of any hemoglobinopathy that may affect determination of HbA1c levels. 5. Has a history of bariatric surgery. 6. Has a history of proliferative diabetic retinopathy within the 6 months prior to Screening. 7. Has had more than 1 episode of diabetic ketoacidosis (DKA) at any time after diagnosis of T2DM. 8. Has a history of more than 1 episode of pancreatitis. 9. Has serum creatinine >=1.5 mg/dL for male participants or >=1.4 mg/dL for female participants, or creatinine clearance <60 milliliter per minute (mL/min) based on calculation by central lab using the Schwartz formula for estimated glomerular filtration rate (eGFR) at screening Visit. 10. Has a documented history of infection with human immunodeficiency virus or chronic active viral hepatitis. 11. The participant and/or his/her legal representative (that is, parents or legal guardians) is unable to understand verbal or written English, or any other language for which a certified translation of the approved informed consent/assent is available. Additional Criteria That Must be Met Prior to Randomization: For participants who have had the diagnosis of T2DM for less than 1 year and/or who are taking insulin at Screening, additional criteria will need to be met prior to randomization: 1. Must have an HbA1c level of >=6.5% to <11.0%. 2. Must not have received an antidiabetic agent other than metformin or insulin within the 12 weeks prior to randomization. 3. Must not have received oral or parenteral steroids for more than 3 weeks (cumulatively) within the 6 months prior to randomization or have received a course of oral or parenteral steroids within the 2 months prior to randomization. 4. Has a systolic blood pressure <160 millimeter of mercury (mmHg) and a diastolic pressure <100 mmHg. (Antihypertensive medications will be allowed during the study). 5. Has an alanine aminotransferase (ALT) level <3*upper limit of normal (ULN) or an ALT level <5 *ULN with a confirmed diagnosis of nonalcoholic fatty liver disease (NAFLD). 6. Does not plan to leave the geographic area within 1 calendar year following randomization. For participants who have had the diagnosis of T2DM for less than 1 year and/or who are taking insulin prior to randomization, the following criteria must also be met: 7. Must have a fasting C-peptide concentration>=0.6 nanogram per milliliter (ng/mL) (>=0.20 nanomole per liter [nmol/L]) (drawn at least 1 week after treatment for ketosis or acidosis, if applicable). 8. No presence of autoantibodies as documented by glutamic acid decarboxylase [GAD] 65 and islet antigen [IA]-2 antibodies below the upper limit of the normal reference ranges at randomization. 9. Have a body mass index (BMI) greater than (>) 85th percentile, documented at randomization.
Drug: Alogliptin Benzoate, Drug: Placebo
Diabetes Mellitus, Type 2
Multicenter Prospective Cohort Study on Current Treatments of Legg-Calvé-Perthes Disease (IPSG1)
Legg-Calvé-Perthes disease is a childhood hip disorder which is common enough to be a significant public health problem (affects 1 in 740 boys between ages 0—14), but uncommon enough to have a sufficient number of patients from a single institution to perform a definitive prospective study comparing the results of current treatments. The present study will establish a database of prospectively identified patients with Legg-Calvé-Perthes (LCP) Disease and collect information regarding their presentation, treatment, and outcomes in the course of receiving currently available treatments. This study seeks to compare the outcomes of current treatments in the management of different age groups (ages 1-6, 6—8, 8—11, >11) of patients with Perthes disease at two- and five-year followup and at skeletal maturity. For each age group, two to three common treatment regimens currently used by practicing pediatric orthopaedic surgeons will be compared. The intervention a patient receives is determined through physician preference. Physicians pick an intervention for each age group and treat each patient with the same intervention.
6 Years to 16 Years old
• Diagnosed with Legg-Calvé-Perthes disease
• Between age 1-18
• Patients with possible secondary femoral osteonecrosis if over the age of 11 due to trauma or corticosteroid therapy are also eligible.
• Patients with previous surgical treatment on the affected hip if not in the >11 age group
Procedure: osteotomy, Procedure: multiple epiphyseal drilling
Legg Calve Perthes Disease
femur head necrosis, hip, pediatric orthopedics, MRI, Osteonecrosis, Bone diseases, Legg Calve Perthes Syndrome
Diaphragmatic Hernia Research & Exploration, Advancing Molecular Science (DHREAMS)
The goal of this study is to identify genes that convey susceptibility to congenital diaphragmatic hernia in humans. The identification of such genes, and examination of their structure and function, will enable a delineation of molecular pathogenesis and, ultimately, prevention or treatment of congenital diaphragmatic hernia. There are many different possible modes of inheritance for congenital anomalies, including autosomal dominant, autosomal recessive, and multifactorial. Multi-factorial inheritance is responsible for many common medical disorders, including hypertension, myocardial infarction, diabetes and cancer. This type of inheritance pattern appears to involve environmental factors as well as a combination of genetic variations that together can predispose to or produce congenital anomalies, such as congenital diaphragmatic hernia. Our study is designed to establish a small, well-defined genetic resource consisting of 1) Nuclear families suitable for linkage analysis by parametric,non-parametric (e.g. sib pairs, TDT) and association techniques, 2) Individuals with congenital diaphragmatic hernia who can be directly screened for allelic variation in candidate genes, and 3) Individuals who can serve as controls (are unaffected by congenital diaphragmatic hernia). Neonates and their families will be collected from homogenous and heterogeneous populations. By characterizing diverse populations, it should be possible to increase the likelihood of demonstration of genetic variation in selected candidate genes that can then be used in association and linkage studies in individual subjects with congenital diaphragmatic hernia.
• All individuals affected with a congenital diaphragmatic hernia (CDH), or with a family history of a CDH
• Individuals with no personal history of a CDH or family history of a family member affected with congenital diaphragmatic hernia
Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia (CDH), Genes, Genetic, Genetic testing, exome sequencing, genome sequencing, RNAseq
Enalapril in Treating Heart Damage Patients Who Received Anthracycline Chemotherapy for Childhood Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Chemoprotective drugs, such as enalapril, may protect normal cells from the toxic effects of chemotherapy. It is not known whether enalapril is more effective than a placebo in treating heart damage in patients who received anthracycline chemotherapy for childhood cancer. PURPOSE: Randomized double-blinded phase III trial to compare the effectiveness of enalapril with a placebo in treating heart damage in patients who received anthracycline chemotherapy for childhood cancer.
8 Years and over
DISEASE CHARACTERISTICS: Histologically diagnosed childhood malignancy that had prior anthracycline therapy Echocardiographic evidence of reduced fractional shortening, reduced contractility, or increased afterload, or any combination At least 6 months oncologic disease free PATIENT CHARACTERISTICS: Age: At least 8 at study entry and less than 22 at diagnosis Performance status: Not specified Life expectancy: Not specified Hematopoietic: Not specified Hepatic: Not specified Renal: No history of renal disease No known renal artery stenosis Cardiovascular: No congenital cardiovascular malformations No active congestive heart failure not attributable to sepsis or renal failure No medication for heart condition No history of symptomatic arrhythmia antedating anthracycline therapy No constrictive pericarditis No uncontrolled hypertension Pulmonary: No primary valvular or outflow tract obstruction Other: Not pregnant or lactating Must use adequate contraception No reaction or intolerance to ACE inhibitors PRIOR CONCURRENT THERAPY: Biologic therapy: Not specified Chemotherapy: At least 1 year since prior cumulative anthracycline therapy of at least 200 mg/m2 No prior amsacrine therapy Endocrine therapy: Not specified Radiotherapy: No prior mediastinal, spinal, or total body irradiation that included the heart Surgery: Not specified Other: No concurrent angiotensin converting enzyme (ACE)inhibitor treatment No concurrent treatment with other investigational drug No oncologic therapy within past 6 months
Drug: enalapril maleate, Procedure: quality-of-life assessment
Unspecified Childhood Solid Tumor, Protocol Specific, Cardiac Toxicity
unspecified childhood solid tumor, protocol specific, cardiac toxicity
VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)
The purpose of this program is to provide elexacaftor(ELX, VX-445)/tezacaftor(TEZ)/ivacaftor(IVA) combination therapy to CF patients in critical need who are 12 years of age and older, heterozygous for F508del and a minimal function (MF) mutation in response to unsolicited physician requests.
12 Years and over
Inclusion Criteria:1. Patients who have F/MF genotypes AND who meet at least 1 of the following criteria:
• The percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 for a minimum of 2 months before the date of the request, OR
• Documentation of being active on a lung transplant waiting list or documentation of being evaluated for lung transplantation, but deemed unsuitable because of contraindications
Exclusion Criteria:1. Patients with severe hepatic impairment (Child-Pugh Class C) 2. History of any other comorbidity that, in the opinion of the treating physician, might pose undue risk in administering ELX/TEZ/IVA to the patient 3. Pregnancy Other protocol defined Inclusion/Exclusion criteria may apply
Drug: ELX/TEZ/IVA, Drug: IVA