Clinical trials: FRDA
Freidreich's Ataxia
Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)
This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study.
- Trial name
- NCT00993967
- Testing
- idebenone
- Where
- 11 sites in Austria, Belgium and 3 more
- Volunteers
- 200 people
- Sponsor
- Santhera Pharmaceuticals
Friedreich Ataxia
Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia
Friedreich's ataxia (FA) is a rare, inherited neurodegenerative disease that typically begins in children and young people. It primarily affects the spinal cord, peripheral nerves and cerebellum of the brain.
- Trial name
- NCT07013292
- Testing
- SKYCLARYS (omaveloxolone)
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Friedreich Ataxia
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
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 prescribe for people with Friedrich's Ataxia, also known as FA.
- Trial name
- NCT06628687
- Testing
- Omaveloxolone
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Biogen
Iron-sulfur Cluster Deficiency, Pulmonary Hypertension
Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension
The investigators are performing this research study to determine whether having low iron-sulfur cluster levels can cause a disease known as pulmonary hypertension (PH).
- Conditions
- Iron-sulfur Cluster Deficiency, Pulmonary Hypertension, Friedreich Ataxia
- Trial name
- NCT02594917
- Testing
- Right Heart Catheterization, Blood draw
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of Pittsburgh
Friedreich Ataxia
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA).
- Trial name
- PROFA (NCT05943002)
- Where
- 6 sites in Austria, France and 1 more
- Volunteers
- 200 people
- Sponsor
- German Center for Neurodegenerative Diseases (DZNE)
Neurologic Disorder, Stroke
Home Based Tele-exercise for People With Chronic Neurological Impairments
To assess the impact of a 12-week virtual seated physical intervention on cardiovascular health and wellness in people with chronic neurological impairments (CNI).
- Conditions
- Neurologic Disorder, Stroke, Spinal Cord Injuries, Cerebral Palsy
- Trial name
- Telex (NCT04564495)
- Testing
- Seated exercise
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Burke Medical Research Institute
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Friedreich Ataxia
A Study of Resveratrol as Treatment for Friedreich Ataxia
The purpose of this study is to determine the effect of two doses of resveratrol taken for a 12 week period, on frataxin levels in individuals with Friedreich ataxia.
- Trial name
- NCT01339884
- Testing
- Resveratrol
- Where
- Australia
- Volunteers
- 27 people
- Sponsor
- Murdoch Childrens Research Institute
Friedreich's Ataxia
Cardiac Output and Fatigue in Friedreich's Ataxia
This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will…
- Trial name
- NCT07444333
- Testing
- Aerobic Exercise
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Scott Barbuto
Friedreich Ataxia
Coronary Artery Disease in Patients With Friedreich's Ataxia
Friedreich's ataxia is a debilitating, inherited disease cause by mutations in a protein called frataxin (FXN). FXN is one of several proteins that controls the production of iron-sulfur clusters, molecules that are essential for…
- Trial name
- NCT04649866
- Testing
- plethysmography
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of Pittsburgh
Friedreich Ataxia
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.
- Trial name
- NCT05515536
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia, Motor Disorders
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Prospective, exploratory, multicenter pilot study investigating the structural and functional connectome in patients with Friedreich's Ataxia (FRDA) using high-density electroencephalogram (HD-EEG).
- Trial name
- CONNETTOMA (NCT07095062)
- Testing
- HD-EEG recordings, combined with cognitive and motor assessment
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Friedreich Ataxia
A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)
This is a natural history study prospectively investigating neuroimaging markers of disease progression in children and adults with Friedreich ataxia (FA). There will be three assessment periods (baseline, 12 and 24 months).
- Trial name
- (TRACK-FA) (NCT04349514)
- Testing
- Natural history
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Monash University
Friedreich Ataxia
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants.
- Trial name
- NCT06772870
- Testing
- DT-216P2, Saline
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Design Therapeutics, Inc.
Friedreich Ataxia
Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia.
- Trial name
- NCT05285540
- Testing
- DT-216
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Design Therapeutics, Inc.
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Friedreich Ataxia
Respiratory Training in Friedreich's Ataxia
This is a prospective interventional study of patients with Friedreich's Ataxia that receive respiratory strength training for a period of 12 weeks with two research visits at the beginning and at the end of the study period.
- Trial name
- NCT06539598
- Testing
- Respiratory Strength Training (RST)
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Florida
Friedreich's Ataxia (FA)
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).
- Trial name
- FALCON (NCT07180355)
- Testing
- SGT-212
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Solid Biosciences Inc.
Friedreich's Ataxia
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.
- Trial name
- PROTI (NCT01303406)
- Testing
- Idebenone
- Where
- 6 sites in Austria, Germany and 2 more
- Volunteers
- 29 people
- Sponsor
- Santhera Pharmaceuticals
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