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Showing 1–24 of 65 matching trials from the live ClinicalTrials.gov search.
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Conditions
Friedreich Ataxia
Interventions
CTI-1601
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 60 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
8
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 5 more
Conditions
Friedreich's Ataxia
Interventions
Placebo, EPI-743 400 mg, EPI-743 200 mg
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Philadelphia, Pennsylvania
Completed Phase 1Phase 2 Interventional Results available

FRDA Investigator Initiated Study (IIS) With Elamipretide

NCT05168774
Conditions
Friedreich Ataxia
Interventions
Elamipretide
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
10 Years and older
Enrollment
85 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2022
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
Interventions
AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
Biological · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
New York, New York
Conditions
Friedreich Ataxia
Interventions
Rosuvastatin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Friedreich Ataxia, Cardiomyopathy
Interventions
Not listed
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
La Jolla, California • Tampa, Florida • Indianapolis, Indiana + 5 more
Conditions
Neurologic Disorder, Stroke, Spinal Cord Injuries, Cerebral Palsy, Hemiplegia, Hemiparesis, Multiple Sclerosis, Parkinson Disease, Trauma, Brain, Transverse Myelitis, Friedreich Ataxia
Interventions
Seated exercise
Behavioral
Lead sponsor
Burke Medical Research Institute
Other
Eligibility
18 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
White Plains, New York
Active, not recruiting No phase listed Observational Accepts healthy volunteers

FA Clinical Outcome Measures

NCT03090789
Conditions
Friedreich Ataxia, Neuro-Degenerative Disease
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 80 Years
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2030
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more
Conditions
Friedreich's Ataxia
Interventions
VP 20629, Placebo
Drug
Lead sponsor
Shire
Industry
Eligibility
18 Years to 45 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
5
States / cities
Los Angeles, California • Tampa, Florida • Atlanta, Georgia + 2 more
Conditions
Friedreich Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
No Longer Available No phase listed Expanded access

EPI-743 for Mitochondrial Respiratory Chain Diseases

NCT01370447
Conditions
Mitochondrial Diseases
Interventions
EPI-743
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
16
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 12 more
Conditions
Friedreich Ataxia
Interventions
Vatiquinone, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years and older
Enrollment
146 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
Recruiting No phase listed Observational Accepts healthy volunteers

Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)

NCT02316314
Conditions
Friedreich's Ataxia
Interventions
Cardiac magnetic resonance imaging (CMR), Exercise-stress test, Echocardiogram (ECHO), Cardiac-related blood studies
Procedure
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
1
States / cities
New York, New York
Completed No phase listed Observational Accepts healthy volunteers

TCA Cycle in the Dentate in Friedreich's Ataxia

NCT03122925
Conditions
Friedreich Ataxia
Interventions
MR
Device
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years and older · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Morrisville, North Carolina
Conditions
Friedreich Ataxia
Interventions
Nomlabofusp, Placebo
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 17 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Chevy Chase, Maryland
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Friedreich's Ataxia
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 18 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Conditions
Iron-sulfur Cluster Deficiency, Pulmonary Hypertension, Friedreich Ataxia
Interventions
Right Heart Catheterization, Blood draw
Procedure · Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 60 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Friedreich Ataxia
Interventions
Respiratory Strength Training (RST)
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Friedreich Ataxia
Interventions
CTI-1601, Placebo
Biological
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Eatontown, New Jersey
Completed Not applicable Interventional Results available

NAD+ and Exercise in FA

NCT04192136
Conditions
Friedreich Ataxia 1
Interventions
Nicotinamide Riboside, Placebo, Exercise Intervention
Dietary Supplement · Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
10 Years to 40 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Friedreich Ataxia, Rare Diseases
Interventions
Geneticlly confirmed disease causing FXN mutatuion, Healthy Control
Other
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 21 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
3
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania • Memphis, Tennessee