Friedreich ataxia: open trials in United States

16 open trials have a site in United States. Registry data; only the trial team decides who can take part.

NCT07180355
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Trial Treatment · Phase 1 · Solid Biosciences Inc.
The Ohio State University, Columbus · The University of California, Los Angeles (UCLA), Los Angeles · The Children's Hospital of Philadelphia (CHOP), Philadelphia
Registry updated 2026-11-06
NCT01793168
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Research · Sanford Health
Sanford Health, Sioux Falls
Registry updated 2026-10-28
NCT06447025
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
Trial Treatment · Phase 2 · Larimar Therapeutics, Inc.
Uncommon Cures, Chevy Chase · Ohio State University Wexner Medical Center, Columbus · Clinilabs Drug Development, Corp., Eatontown · Fixel Institute for Neurological Disease, University of Florida Health, Gainesville · University of Iowa Hospitals & Clinics, Iowa City · University of California, Los Angeles, Los Angeles · Children's Hospital of Philadelphia, Philadelphia · ICON, Salt Lake City · University of California, San Franciso Medical Center, San Francisco · Morsani Center for Advanced Health Care, University of South Florida Health, Tampa
Registry updated 2026-10-27
NCT06865482
Clinical Course Of Disease In Participants With FA-CM
Research · Lexeo Therapeutics
Boston Children's Hospital, Boston · Cincinnati Children's Hospital, Cincinnati · Indiana University - Riley Children's Health, Indianapolis · University of California San Diego, La Jolla · Oregon Health Science University, Portland · Mayo Clinic, Rochester · Washington University School of Medicine in St. Louis, St Louis · University of South Florida, Tampa
Registry updated 2026-10-19
NCT07870590
GV101 for Friedreich's Ataxia (FA) Treatment
Trial Treatment · Phase 2 · Graviton Bioscience Corporation
CLINiLABS, Eatontown
Registry updated 2026-10-15
NCT07778836
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
Trial Treatment · Phase 3 · Larimar Therapeutics, Inc.
Northwestern Medical Group, Department of Neurology, Chicago · University of Texas Southwestern Medical Center, Dallas · CLINiLABS, Eatontown · Morsani Center for Advanced Health Care, University of South Florida Health, Tampa
Registry updated 2026-10-09
NCT02497534
Biomarkers in Friedreich's Ataxia
Research · University of Florida
University of Florida, Gainesville
Registry updated 2026-10-06
NCT05302271
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
Trial Treatment · Phase 1 · Weill Medical College of Cornell University
Weill Cornell Medicine, New York
Registry updated 2026-09-28
NCT07444333
Cardiac Output and Fatigue in Friedreich's Ataxia
Trial Treatment · Scott Barbuto
Columbia University Irving Medical Center, New York
Registry updated 2026-09-22
NCT07721025
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Trial Treatment · Phase 2 · Lexeo Therapeutics
University of South Florida, Tampa
Registry updated 2026-09-02
NCT06016946
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Research · Friedreich's Ataxia Research Alliance
Emory University Hospital - Neurology, Atlanta · Ohio State University - Neurology, Columbus · University of Colorado, Denver · University of Florida - Neurology, Gainesville · University of Iowa Stead Family Children's Hospital, Iowa City · UCLA Ataxia Center, Los Angeles · St. Jude Children's Research Hospital, Memphis · Children's Hospital of Philadelphia, Philadelphia · USF Ataxia Research Center, Tampa
Registry updated 2026-09-01
NCT07681713
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
Trial Treatment · Phase 3 · PTC Therapeutics
University of Iowa, Iowa City · University of California, Los Angeles, Los Angeles · Children's Hospital of Philadelphia, Philadelphia · University of South Florida, Tampa
Registry updated 2026-08-14
NCT02316314
Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)
Research · Weill Medical College of Cornell University
Weill Cornell Medicine, New York
Registry updated 2026-07-29
NCT06628687
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
Research · Biogen
Evidera, Morrisville
Registry updated 2026-07-10
NCT07635030
GABA and GSH in FRDA
Research · Children's Hospital of Philadelphia
The Children's Hospital Of Philadelphia, Philadelphia
Registry updated 2026-06-09
NCT06623890
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
Research · Biogen
University of Colorado, Aurora · University of Florida, Gainesville · UCLA Neurology, Los Angeles · Children's Hospital of Philadelphia, Philadelphia
Registry updated 2026-04-28

All Friedreich ataxia trials and the map

This page is not medical advice. Talk to your doctor before contacting a trial.

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