Clinical trials: Friedreich Ataxia 1
Friedreich Ataxia 1
NAD+ and Exercise in FA
Randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on Peak VO2 and Si in Friedreich's Ataxia (FA).
- Trial name
- ExRx in FA (NCT04192136)
- Testing
- Nicotinamide Riboside, Exercise Intervention
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
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
In this study, researchers will learn more about BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at…
- Trial name
- BOLD (NCT06054893)
- Testing
- Omaveloxolone
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Biogen
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2
Instrumented Data Exchange for Ataxia Study
This research study is testing body-worn sensors to measure movement during simple tests of coordination, in order to evaluate the progression and severity of ataxia.
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6
- Trial name
- IDEA (NCT04268147)
- Where
- 5 sites in United States
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- University of Chicago
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Friedreich Ataxia
A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
- Trial name
- NCT06681766
- Testing
- Nomlabofusp
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Larimar Therapeutics, Inc.
Friedreich Ataxia
Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia
This study will determine the highest amount of idebenone that can be taken without harmful side effects in children, teenagers, and adults with Friedreich's ataxia, a progressive degenerative disease that affects several body…
- Trial name
- NCT00078481
- Testing
- Idebenone
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)