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A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy…
Phase 1b Open-label Basket Trial of RAY121 to Inhibit Classical Complement Pathway in Immunological Diseases (RAINBOW Trial)
This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a …
Prospective Investigation of Cirrhotic Cardiomyopathy in Humans
Cirrhotic Cardiomyopathy (CCM) is a recognized complication of cirrhosis, but understudied despite recent retrospective data suggesting it may be common, affec…
A Phase 1/2 Multicenter, Open-Label, Dose-Escalation and Dose-Expansion Trial to Assess the Safety, Tolerability, Pharmacodynamics, and Preliminary Efficacy of AFTX-201 Administered to Adult Participants With BCL2-Associated Athanogene 3 (BAG3) Mutation-Associated Dilated Cardiomyopathy: UPBEAT Trial
This is a Phase 1/2, open-label, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, pharmacodynamics, and preliminary eff…
A Multicenter Pilot Study to Investigate Ocular Involvement and the Potential of Ocular Imaging for Non-invasive Screening for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
This multicenter pilot study will enroll adult participants with a confirmed clinical diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) and heart fai…
An Open-Label, Exploratory, Phase 2 Scintigraphy Study Evaluating 18F mFBG for Imaging Myocardial Sympathetic Innervation in Subjects With Heart Failure and Implantable Cardioverter-Defibrillators
This is a Phase 2 study evaluating the positron-emitting radiopharmaceutical 18F-mFBG as an imaging agent for quantification of myocardial sympathetic innervat…
A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701) in Subjects With Dilated Cardiomyopathy Arising From Pathogenic BAG3 Variants (BAG3-DCM)
This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV admini…
A Phase III, Randomised, Double-blind, Placebo-controlled, Parallel-group, Pivotal Trial to Assess the Efficacy and Safety of Sonlicromanol in Adult Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Variant
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life,…
A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells (nMSCs) in Young Adult (1A) and Pediatric (1B) Patients With Dilated Cardiomyopathy (DCM)
This is a Phase 1 study to determine the safety and efficacy of allogeneic neonatal mesenchymal stromal cells (nMSCs) for the treatment of Dilated Cardiomyopat…
Identifying Subclinical Transthyretin Cardiac Amyloidosis in Asymptomatic Carriers of the V122I TTR Allele
Approximately 1.5 million of the 44 million Blacks in the United States are carriers of the valine-to-isoleucine substitution at position 122 (V122I) in the tr…
WiTNNess: An International Natural History Study of Autosomal Recessive TNNT1 Myopathy
WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim …
Pediatric Cardiomyopathy Mutation Analysis
The goal of this protocol is to obtain information from individuals with cardiomyopathy and from their families in order to elucidate the molecular genetics of…