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ClinicalTrials.gov public records Last synced May 21, 2026, 7:40 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 64 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Friedreich Ataxia
Interventions
DT-216, DT-216 matching Placebo
Drug
Lead sponsor
Design Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich's Ataxia
Interventions
Idebenone, Placebo
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 17 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 10, 2023 · Synced May 21, 2026, 7:40 PM EDT
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
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich Ataxia
Interventions
Buccal Swabs and Blood Draws
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
20 participants
Timeline
2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 17, 2020 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Gainesville, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced May 21, 2026, 7:40 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
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
Source: ClinicalTrials.gov public record
Updated Aug 18, 2025 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Spinocerebellar Ataxia - All Sub-types, Friedreich's Ataxia
Interventions
Ataxia rating scale
Other
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Nov 15, 2021 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 2 Interventional Results available
Conditions
Friedreich's Ataxia
Interventions
(+)-Epicatechin
Drug
Lead sponsor
Ralitza Gavrilova
Other
Eligibility
10 Years to 50 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich Ataxia
Interventions
Natural history
Other
Lead sponsor
Monash University
Other
Eligibility
5 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • Minneapolis, Minnesota • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 26, 2024 · Synced May 21, 2026, 7:40 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Jun 1, 2021 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 11, 2006 · Synced May 21, 2026, 7:40 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone, Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
255 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 15, 2026 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 1Phase 2 Interventional Results available
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
Source: ClinicalTrials.gov public record
Updated Dec 11, 2025 · Synced May 21, 2026, 7:40 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Jun 29, 2021 · Synced May 21, 2026, 7:40 PM EDT
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
7
States / cities
La Jolla, California • Tampa, Florida • Indianapolis, Indiana + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich's Ataxia
Interventions
varenicline, placebo
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Tampa, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 23, 2012 · Synced May 21, 2026, 7:40 PM EDT
Completed No phase listed Observational Results available
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 18 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
3
States / cities
Los Angeles, California • Gainesville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 15, 2025 · Synced May 21, 2026, 7:40 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Feb 4, 2025 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich Ataxia
Interventions
CTI-1601, Placebo
Biological · Other
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich's Ataxia (FA)
Interventions
SGT-212
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
18 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 6 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 21, 2025 · Synced May 21, 2026, 7:40 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Jan 22, 2024 · Synced May 21, 2026, 7:40 PM EDT
Conditions
Friedreich's Ataxia
Interventions
Interferon γ-1b, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
10 Years to 25 Years
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced May 21, 2026, 7:40 PM EDT