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9 recruiting studies matching your filters

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). …

Friedreich AtaxiaCardiomyopathiesCardiac Hypertrophy+1 more

Estimated

~$10 – $6,250

Weill Medical College of Cornell UniversityNCT05302271

Phase 2

An Open-Label Extension Study to Assess the Long-Term Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects With Friedreich's Ataxia

An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult …

Friedreich Ataxia

Estimated

~$10 – $8,750

Larimar Therapeutics, Inc.NCT06447025

Phase 2

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…

Friedreich AtaxiaCardiomyopathy, Secondary

Estimated

~$10 – $8,750

Lexeo TherapeuticsNCT07721025

Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)

Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)

Friedreich AtaxiaCardiomyopathy

Estimated

~$10 – $1,050

Lexeo TherapeuticsNCT06865482

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression an…

Friedreich Ataxia

Estimated

~$10 – $1,125

Friedreich's Ataxia Research AllianceNCT06016946

Phase 3

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia

To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia

Friedreich AtaxiaFriedreich's Ataxia

Estimated

~$10 – $8,750

Larimar Therapeutics, Inc.NCT07778836

A Post-marketing, Observational, Descriptive Study to Assess the Risk Associated With Pregnancy, the Maternal Complications, and Adverse Effects on the Developing Fetus, Neonate, and Infant Among Individuals Exposed to Omaveloxolone During Pregnancy and/or Lactation

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to presc…

Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, …

Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more

An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to pres…