APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy...
Transthyretin Amyloidosis (ATTR) With CardiomyopathyThe purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
Symptomatic Transthyretin Amyloid CardiomyopathyThis prospective study is designed to evaluate the efficacy, safety, and tolerability of ALXN2060 (also known as AG10), as well as to establish its pharmacokinetic and pharmacodynamic profile in Japanese participants with symptomatic ATTR-CM administered on a background of stable heart failure therapy.
A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin...
Transthyretin Amyloid CardiomyopathyThis is a national, multi-center, single-arm study, open-label to patients with symptomatic Transthyretin amyloid cardiomyopathy (ATTR-CM) who are tafamidis naïve. This study is to obtain safety, descriptive efficacy, Pharmacokinetics (PK) and Pharmacodynamics (PD) data for tafamidis orally once daily. Subject eligibility for participation in the study will receive tafamidis once daily or 12 months following the assessment as the screening and baseline, month 1, 3, 6, 9 and 12 visits (or Early Study Discontinuation).
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy...
Transthyretin Amyloidosis (ATTR) With CardiomyopathyThis study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as...
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC). For more information, please visit https://www.cardio-ttransform.com.
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis...
AmyloidosisHereditary1 moreThe purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran subcutaneous (SC) injection once every 3 months (q3M) or the reference comparator patisiran intravenous (IV) injection once every 3 weeks (q3w) during the 18 month Treatment Period. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy endpoints during the 18 Month Treatment Period. Following the 18 Month Treatment Period, all participants will be randomized to receive vutrisiran SC injection once every 6 months (q6M) or q3M in the Randomized Treatment Extension (RTE) Period.
S1702 Isatuximab in Treating Patients With Relapsed or Refractory Primary Amyloidosis
AmorphousEosinophilic13 moreThis phase II trial studies how well isatuximab works in treating patients with primary amyloidosis that has come back or does not respond to treatment. Monoclonal antibodies, such as isatuximab, may interfere with the ability of cancer cells to grow and spread.
A Study of Daratumumab Monotherapy in Previously Untreated Patients With Stage 3B Light Chain (AL)...
Light Chain (AL) AmyloidosisStage 3BThis is an open-label, multicenter, Phase 2 study in subjects with newly diagnosed stage 3B light chain (AL) amyloidosis.
COVID-19 VAX Booster Dosing in Patients With Hematologic Malignancies
Multiple MyelomaAL Amyloidosis1 moreTo determine whether protective antibody levels increase after booster dosing with the Moderna COVID-19 vaccine in patients diagnosed with Hematologic Malignancies who have low antibody levels after a prior first vaccination with any of the SARS-CoV2 vaccines that were authorized for use in the USA. Researchers will also assess whether the booster dosing with the Moderna COVID-19 vaccine is safe in patients with multiple myeloma, amyloidosis, or other blood cancers.
A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
AL AmyloidosisAL 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 the recommended dose of CAEL-101 to facilitate progression of further clinical trials and evaluate safety and tolerability of CAEL-101 in combination with the standard of care (SoC) cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy and daratumumab .