Clinical Trials for Friedreich Ataxia

A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

NCT ID: NCT06953583

This clinical trial is studying a medication called BIIB141, also known as omaveloxolone or SKYCLARYS®, to see how it affects children and teens aged 2 to 15 years with Friedreich's Ataxia (FA). Wh...

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

NCT ID: NCT06016946

The Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study is a research project aimed at understanding the progression of Friedreich ataxia, a genetic condition that affects mo...

Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia

NCT ID: NCT07013292

This clinical trial is studying the effectiveness of a medication called Omaveloxolone in treating dysphagia, which is difficulty swallowing, in patients with Friedreich's ataxia (FA). FA is a rare...

Respiratory Training in Friedreich's Ataxia

NCT ID: NCT06539598

This clinical trial is studying the effects of respiratory strength training in patients with Friedreich's Ataxia, a genetic condition that can affect movement and coordination. Over a period of 12...

A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

NCT ID: NCT06054893

This clinical trial is studying a medication called omaveloxolone to see how it works and if it is safe for children with Friedreich's Ataxia, a genetic condition that affects movement and coordina...

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

NCT ID: NCT01793168

The Coordination of Rare Diseases at Sanford (CoRDS) is a clinical trial aimed at creating a large, centralized registry for individuals affected by rare diseases. This registry helps connect patie...

A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

NCT ID: NCT06874010

This clinical trial is studying a new medication called DT-216P2 to see how safe it is and how well it works in people with Friedreich's Ataxia (FA), a rare genetic condition that affects movement ...

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

NCT ID: NCT05302271

This clinical trial is looking at a new gene therapy called AAVrh.10hFXN to see if it can help treat heart problems caused by Friedreich's ataxia (FA), a genetic condition that affects movement and...

Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia

NCT ID: NCT05874388

This clinical trial is looking at how Friedreich's Ataxia (FA) affects thinking and cognitive skills in patients. FA is a genetic disease that usually starts in childhood or adolescence and leads t...

Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia

NCT ID: NCT06692296

This clinical trial is investigating whether a special rehabilitation tool called the "Prokin 252" stabilometric platform can help improve balance in people with Friedreich's Ataxia, a condition th...

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

NCT ID: NCT06623890

This clinical trial is focused on understanding the long-term safety of a medication called BIIB141, also known as omaveloxolone or SKYCLARYS®, for individuals with Friedreich's Ataxia (FA) who are...

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients with Neuromuscular Disorders

NCT ID: NCT06605612

Clinical Course Of Disease In Participants With FA-CM

NCT ID: NCT06865482

This clinical trial, called CLARITY-FA, is looking at how Friedreich Ataxia (FA) affects the heart, particularly in people who have a condition known as cardiomyopathy. Cardiomyopathy means that th...

A Study to Assess Nomlabofusp in Adolescents and Children with Friedreich's Ataxia

NCT ID: NCT06681766

This clinical trial is studying a new treatment called nomlabofusp (also known as CTI-1601) for children and teenagers with Friedreich's ataxia (FRDA), a genetic condition that affects movement and...

A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants

NCT ID: NCT06772870

This clinical trial is studying a new treatment called DT-216P2 to see how safe it is and how the body processes it in healthy adults. The researchers want to understand how different doses of this...

A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies

NCT ID: NCT06628687

The SKYCLARYS® (Omaveloxolone) Pregnancy and Lactation Surveillance Program is a clinical trial designed to understand the effects of the medication omaveloxolone on women with Friedreich's Ataxia ...

Frataxin mRNA in Biofluids

NCT ID: NCT06496451

This clinical trial is studying a new way to measure a specific type of genetic material called frataxin messenger RNA (mRNA) in blood and cerebrospinal fluid (CSF) from patients with Friedreich At...

An Open Label Extension Study of CTI-1601 in Subjects With Friedreich's Ataxia

NCT ID: NCT06447025

Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia

NCT ID: NCT05943002

The PROFA study is a research project looking into the experiences of people living with Friedreich Ataxia (FA), a rare genetic condition that affects movement and coordination. The goal of the stu...