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4 Study Matches

A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis

AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract. The primary purpose of this study is to determine if CAEL-101 improves the overall survival in Patients with cardiac AL Amyloidosis.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Larry Anderson
102991
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04504825
STU-2020-1099
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Inclusion Criteria:

• Each patient must meet the following criteria to be enrolled in this study. 1. Be able to and provide written informed consent and be willing and able to comply with all study procedures 2. Adult, 18 years and older 3. AL amyloidosis Mayo stage IIIb based on the 2013 European Modification of the 2004 Standard Mayo Clinic Staging in patients with advanced cardiac involvement at the time of Screening 4. Measurable hematologic disease at Screening as defined by at least one of the following: 1. Involved/Uninvolved Free Light Chain Difference (dFLC) > 4 mg/dL or 2. Involved Free Light Chain (iFLC) > 4 mg/dL with abnormal ratio or 3. Serum Protein Electrophoresis (SPEP) m-spike > 0.5 g/dL 5. Histopathological diagnosis of amyloidosis AND confirmation of AL derived amyloid deposits by at least one of the following: 1. Immunohistochemistry or 2. Mass spectrometry or 3. Characteristic electron microscopy appearance 6. Cardiac involvement as defined by: 1. Documented clinical signs and symptoms supportive of a diagnosis of heart failure in the setting of a confirmed diagnosis of AL amyloidosis in the absence of an alternative explanation for heart failure AND 2. At least one of the following: i. Endomyocardial biopsy demonstrating AL cardiac amyloidosis or ii. Echocardiogram demonstrating a mean left ventricular wall thickness (calculated as [IVSd+LPWd]/2) of > 12 mm at diastole in the absence of other causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening or iii. Cardiac MRI with gadolinium contrast agent diagnostic or cardiac amyloidosis 7. Planned first-line treatment for plasma cell disorder is a CyBorD-based regimen administered as Standard of Care (SoC) 8. Adequate bone marrow reserve and hepatic function as demonstrated by: 1. Absolute neutrophil count ≥ 1.0 x 109/L 2. Platelet count ≥ 75 x 109/L 3. Hemoglobin ≥ 9 g/dL 4. Total direct bilirubin ≤ 2 times the upper limit of normal (x ULN) unless due to Gilbert's syndrome. 5. Aspartate aminotransferase (AST) ≤ 3 x ULN 6. Alanine aminotransferase (ALT) ≤ 3 x ULN 7. Alkaline phosphatase (ALP) ≤ 5 x ULN (except for patients with hepatomegaly and isozymes specific to liver, rather than bone) 9. Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective physician approved contraception from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of her PCD therapy, whichever is longer 10. Men must be surgically sterile or must agree to use effective physician approved contraception and refrain from donating sperm from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of his PCD therapy, whichever is longer.
Exclusion Criteria:

• Patients who meet any of the following criteria will not be permitted entry to the study. 1. Have any other form of amyloidosis other than AL amyloidosis 2. Received prior therapy for AL amyloidosis or multiple myeloma. A maximum exposure of 160 mg dexamethasone (or equivalent corticosteroid) since diagnosis of AL amyloidosis and prior to randomization is allowed. 3. Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma or POEMS syndrome 4. Have supine systolic blood pressure < 90 mmHg or symptomatic orthostatic hypotension, defined as a decrease in systolic blood pressure upon standing of > 30 mmHg despite medical management (e.g., midodrine, fludrocortisones) in the absence of volume depletion 5. Taking prednisone or its equivalent > 10 mg/day 6. Taking doxycycline 7. Receiving dialysis 8. Planned stem cell transplant during the first 6 months of protocol therapy. Stem cell collection during the protocol therapy is permitted. 9. Have had myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias within 6 months prior to screening or percutaneous cardiac intervention with recent stent or coronary artery bypass grafting within 4 months prior to screening. Exacerbation of chronic condition or new acute condition will require discussion and approval by the Medical Monitor. 10. Left Ventricular Ejection Fraction (LVEF) is < 40% by echocardiogram at Screening 11. Have severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area < 1.0 cm2) or severe congenital heart disease 12. Have history of sustained ventricular tachycardia or aborted ventricular fibrillation or a history of atrioventricular nodal or sinoatrial nodal dysfunction for which a pacemaker/implantable cardioverter-defibrillator (ICD) is indicated but not placed. (Participants who do have a pacemaker or ICD are allowed in the study.) 13. QT corrected by Fridericia (QTcF) is > 550 msec. Participants who have a pacemaker may be included regardless of calculated QTc interval. 14. There is evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following: 1. First degree Atrioventricular (AV)-block 2. Second degree AV-block Type 1 (Mobitz Type 1/Wenckebach type) 3. Right or left bundle branch block 4. Atrial fibrillation with a controlled ventricular rate. (An uncontrolled ventricular rate [i.e., > 110 beats per minute] determined by an average of three beats in lead II or representative beats in lead II is not allowed) 15. Have had major surgery within 4 weeks of randomization or is planning major surgery during the study. Patients with surgical procedures conducted under local anesthesia may participate 16. There is active malignancy (including lymphoma) with the exception of any of the following: 1. Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer 2. Adequately treated stage I cancer from which the patient is currently in remission and has been in remission for > 2 years 3. Low-risk prostate cancer with Gleason score < 7 and prostate-specific antigen < 10 mg/mL 4. Other localized and/or low risk malignancies may be permitted with Medical Monitor approval. 17. Have received an investigational drug/device in another clinical investigational study within 60 days before Screening 18. Hypersensitivity to the study drug 19. Have received a live vaccine within 4 weeks prior to first dose of CyBorD 20. Women who are breast feeding 21. Have any other medical, social or psychological factors that could affect the patient's safety or ability to consent personally or comply with study procedures.
Drug: CAEL-101, Other: Placebo, Drug: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD) regimen
Multiple Myeloma, AL Amyloidosis
Plasma Cell Dyscrasia, cyclophosphamide, bortezomib and dexamethasone (CyBorD), AL Amyloidosis, Amyloid, Light chain Amyloidosis, treatment-naïve, Mayo Stage IIIb
UT Southwestern
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CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

To evaluate the efficacy of AKCEA-TTR-LRx compared to placebo for 120 weeks in patients with ATTR-CM receiving available standard of care (SoC). For more information, please visit https://www.cardio-ttransform.com/.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Justin Grodin
74652
All
18 Years to 90 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04136171
STU-2019-1609
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Inclusion Criteria:

• Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method
• Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participant's non-pregnant female partner must be using a highly effective contraceptive method
• Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2- propanodicarboxylic acid [DPD-Tc], 99m Tc-pyrophosphate [PYP-Tc], or 99m Tc-hydroxymethylene-diphosphonate [HMDP-Tc]) with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, centrally confirmed
• End-diastolic interventricular septum thickness of > 12 mm on Screening echocardiogram
• New York Heart Association (NYHA) class I-III
Exclusion Criteria:

• Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
• Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease
• Monoclonal gammopathy of undetermined significance (MGUS) and/or immunoglobulin free light chain ratio < 0.26 and > 1.65, unless fat, bone marrow, or heart biopsy confirming the absence of light chain by mass spectrometry or immunoelectron microscopy
• Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization
• Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or other oligonucleotide or RNA therapeutic (including siRNA)
• Current treatment with diflunisal, doxycycline, and/or calcium-channel blocker. Participants receiving any of these agents must respect a wash-out period of 14 days before randomization.
Drug: AKCEA-TTR-LRx, Drug: Placebo
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
TTR, Amyloidosis, Cardiomyopathy
UT Southwestern
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  Contact the study team
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A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis

AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract. The primary purpose of this study is to determine if CAEL-101 improves the overall survival in Patients with cardiac AL Amyloidosis.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Larry Anderson
102991
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04512235
STU-2020-1138
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Each patient must meet the following criteria to be enrolled in this study. 1. Be able to and provide written informed consent and be willing and able to comply with all study procedures 2. Adult, 18 years and older 3. AL amyloidosis Mayo stage IIIa based on the 2013 European Modification of the 2004 Standard Mayo Clinic Staging in patients with advanced cardiac involvement at the time of Screening 4. Measurable hematologic disease at Screening as defined by at least one of the following: 1. Involved/Uninvolved Free Light Chain Difference (dFLC) > 4 mg/dL or 2. Involved Free Light Chain (iFLC) > 4 mg/dL with abnormal ratio or 3. Serum Protein Electrophoresis (SPEP) m-spike > 0.5 g/dL 5. Histopathological diagnosis of amyloidosis AND confirmation of AL derived amyloid deposits by at least one of the following: 1. Immunohistochemistry or 2. Mass spectrometry or 3. Characteristic electron microscopy appearance 6. Cardiac involvement as defined by: 1. Documented clinical signs and symptoms supportive of a diagnosis of heart failure in the setting of a confirmed diagnosis of AL amyloidosis in the absence of an alternative explanation for heart failure AND 2. At least one of the following: i. Endomyocardial biopsy demonstrating AL cardiac amyloidosis or ii. Echocardiogram demonstrating a mean left ventricular wall thickness (calculated as [IVSd+LPWd]/2) of > 12 mm at diastole in the absence of other causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening or iii. Cardiac MRI with gadolinium contrast agent diagnostic or cardiac amyloidosis 7. Planned first-line treatment for plasma cell dyscrasia is a CyBorD-based regimen administered as Standard of Care (SoC) 8. Adequate bone marrow reserve and hepatic function as demonstrated by: 1. Absolute neutrophil count ≥ 1.0 x 109/L 2. Platelet count ≥ 75 x 109/L 3. Hemoglobin ≥ 9 g/dL 4. Total direct bilirubin ≤ 2 times the upper limit of normal (x ULN) unless due to Gilbert's syndrome. 5. Aspartate aminotransferase (AST) ≤ 3 x ULN 6. Alanine aminotransferase (ALT) ≤ 3 x ULN 7. Alkaline phosphatase (ALP) ≤ 5 x ULN (except for patients with hepatomegaly and isozymes specific to liver, rather than bone) 9. Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective physician approved contraception from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of her PCD therapy, whichever is longer 10. Men must be surgically sterile or must agree to use effective physician approved contraception and refrain from donating sperm from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of his PCD therapy, whichever is longer
Exclusion Criteria:

• Patients who meet any of the following criteria will not be permitted entry to the study. 1. Have any other form of amyloidosis other than AL amyloidosis 2. Received prior therapy for AL amyloidosis or multiple myeloma. A maximum exposure of 160 mg dexamethasone (or equivalent corticosteroid) since diagnosis of AL amyloidosis and prior to randomization is allowed. 3. Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma or POEMS syndrome 4. Have supine systolic blood pressure < 90 mmHg or symptomatic orthostatic hypotension, defined as a decrease in systolic blood pressure upon standing of > 30 mmHg despite medical management (e.g., midodrine, fludrocortisones) in the absence of volume depletion 5. Taking prednisone or its equivalent > 10 mg/day 6. Taking doxycycline 7. Receiving dialysis 8. Planned stem cell transplant during the first 6 months of protocol therapy. Stem cell collection during the protocol therapy is permitted. 9. Have had myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias within 6 months prior to screening or percutaneous cardiac intervention with recent stent or coronary artery bypass grafting within 4 months prior to screening. Exacerbation of chronic condition or new acute condition will require discussion and approval by the Medical Monitor. 10. Left Ventricular Ejection Fraction (LVEF) is < 40% by echocardiogram at Screening 11. Have severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area < 1.0 cm2) or severe congenital heart disease 12. Have history of sustained ventricular tachycardia or aborted ventricular fibrillation or a history of atrioventricular nodal or sinoatrial nodal dysfunction for which a pacemaker/implantable cardioverter-defibrillator (ICD) is indicated but not placed. (Participants who do have a pacemaker or ICD are allowed in the study.) 13. QT corrected by Fridericia (QTcF) is > 550 msec. Participants who have a pacemaker may be included regardless of calculated QTc interval. 14. There is evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following: 1. First degree Atrioventricular (AV)-block 2. Second degree AV-block Type 1 (Mobitz Type 1/Wenckebach type) 3. Right or left bundle branch block 4. Atrial fibrillation with a controlled ventricular rate. (An uncontrolled ventricular rate [i.e., > 110 beats per minute] determined by an average of three beats in lead II or representative beats in lead II is not allowed) 15. Have had major surgery within 4 weeks of randomization or is planning major surgery during the study. Patients with surgical procedures conducted under local anesthesia may participate 16. There is active malignancy (including lymphoma) with the exception of any of the following: 1. Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer 2. Adequately treated stage I cancer from which the patient is currently in remission and has been in remission for > 2 years 3. Low-risk prostate cancer with Gleason score < 7 and prostate-specific antigen < 10 mg/mL 4. Other localized and/or low risk malignancies may be permitted with Medical Monitor approval. 17. Have received an investigational drug/device in another clinical investigational study within 60 days before Screening 18. Hypersensitivity to the study drug 19. Have received a live vaccine within 4 weeks prior to first dose of CyBorD 20. Women who are breast feeding 21. Have any other medical, social or psychological factors that could affect the patient's safety or ability to consent personally or comply with study procedures.
Drug: CAEL-101, Other: Placebo, Drug: cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
Multiple Myeloma, AL Amyloidosis
Plasma Cell Dyscrasia, cyclophosphamide, bortezomib and dexamethasone (CyBorD), AL Amyloidosis, Amyloid, Light chain Amyloidosis, treatment-naïve, Mayo Stage IIIa
UT Southwestern
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Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy (ATTRibute-CM)

Phase 3 efficacy and safety study to evaluate acoramidis (AG10) 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
Call 214-648-5005
studyfinder@utsouthwestern.edu
Justin Grodin
74652
All
18 Years to 90 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03860935
STU-2018-0350
Show full eligibility criteria
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Inclusion Criteria:

• Have an established diagnosis of ATTR-CM with either wild-type TTR or variant TTR genotype
• Have a history of heart failure evidenced by at least one prior hospitalization for heart failure or clinical evidence of heart failure without prior heart failure hospitalization manifested by signs or symptoms of volume overload or elevated intracardiac pressures or heart failure symptoms that required or require ongoing treatment with a diuretic.
• New York Heart Association (NYHA) Class I-III symptoms due to ATTR cardiomyopathy.
• On stable doses of cardiovascular medical therapy
• Completed ≥150 m on the 6MWT on 2 tests that are within 15% of total distance walked prior to randomization
• Biomarkers of myocardial wall stress, NT-proBNP level ≥300 pg/mL at screening
• Have left ventricular wall (interventricular septum or left ventricular posterior wall) thickness ≥12 mm
Exclusion Criteria:

• Had acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening
• Has hemodynamic instability
• Likely to undergo heart transplantation within a year of screening
• Confirmed diagnosis of primary (light chain) amyloidosis
• Biomarkers of myocardial wall stress, NT-proBNP level ≥8500 pg/mL at screening
• Measure of kidney function, eGFR by MDRD formula <15 mL/min/1.73 m2
• Current treatment with marketed drug products and other investigational agents for the treatment of ATTR-CM
• Current treatment with calcium channel blockers with conduction system effects (e.g. verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed. The use of digitalis will only be allowed if required for management of atrial fibrillation with rapid ventricular response
Drug: acoramidis, Drug: Placebo Oral Tablet
Heart Diseases, Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Cardiovascular
Amyloidosis, ATTR-CM, Transthyretin, Amyloid, TTR
UT Southwestern
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