Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT05515536 · ClinicalTrials.gov registry record · Phase 3
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
A Phase 3 study of Friedreich Ataxia, sponsored by PTC Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 130
- Enrollment target
- 14
- Study locations
NCT05515536: Active Phase 3 study of Friedreich Ataxia, sponsored by PTC Therapeutics.
NCT05515536 is a Phase 3 study of Friedreich Ataxia that is active but no longer recruiting, run by PTC Therapeutics. The registered enrollment target is 130 participants, below the 440-participant average among 14 other Friedreich Ataxia trials with a reported enrollment target (70% lower). The trial reports 14 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05515536, a Phase 3 study of Friedreich Ataxia, is active but no longer recruiting, sponsored by PTC Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 130 participants
- Enrollment target
- 14
- Study locations
Study Summary
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.
Conditions Studied
Interventions
- DRUG Vatiquinone
Study Locations (14)
Other
- University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology - São Paulo
- Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute) - Paris
- Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE) - Tübingen
- Ospedale Pediatrico Bambino Gesu' IRCCS - Roma
- CBR Neurogenetic Research Clinic, University of Auckland - Auckland
- Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares - Barcelona
Florida
- University of Florida - Gainesville
- University of South Florida - Tampa
Quebec
- Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) - Montreal
- CHU Sainte-Justine - Montreal
California
- UCLA - Los Angeles
Iowa
- University of Iowa - Iowa City
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Victoria
- Murdoch Children's Research Institute - Parkville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 130 participants |
| Start Date | 2022-12-08 |
| Est. Completion | 2027-12-31 |
| Phase | Phase 3 |
What the registry record for NCT05515536 still lists
NCT05515536 is an interventional study that assigns participants to a tested intervention. The registered 130 participants enrollment target is mid-sized for trials with a published cap, below the 440-participant average among 14 other Friedreich Ataxia trials with a reported enrollment target (70% lower).
The record links to 1 condition, with Friedreich Ataxia appearing as the primary indexed condition, and to 1 intervention - of which Vatiquinone is the first listed.
NCT05515536 lists 14 locations in 7 states (Other, Florida, Quebec).
Frequently Asked Questions
What is clinical trial NCT05515536 about?
NCT05515536 is a clinical study titled "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.
What is the current status of trial NCT05515536?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 130 participants. The study started on 2022-12-08. Estimated completion is 2027-12-31.
What conditions does trial NCT05515536 study?
This clinical trial studies the following conditions: Friedreich Ataxia.
What interventions are being tested in trial NCT05515536?
The interventions under investigation include: Vatiquinone (DRUG).
Who is sponsoring clinical trial NCT05515536?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05515536 being conducted?
This trial has 14 study locations across California, Florida, Iowa, Pennsylvania, Victoria. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Friedreich Ataxia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05515536's enrollment target sits among peer trials
130 6th of 14 higher than 9 of 14 other Friedreich Ataxia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Friedreich Ataxia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old (BRAVE)
RECRUITING · Phase 3
-
Clinical Course Of Disease In Participants With FA-CM
RECRUITING
-
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
RECRUITING
-
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
RECRUITING · Phase 1
-
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
RECRUITING · Phase 1
-
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
RECRUITING
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01492036 · 130 participants
Long-Term Follow-Up of Recipient of Gene Transfer Research
- NCT01631617 · 130 participants · Phase 2
Effects of Treatments on Atopic Dermatitis
- NCT02368041 · 130 participants
Utility of Thenar Near Infrared Spectroscopy (NIRS) in Pediatric Patients With Heart Failure
- NCT02604277 · 130 participants · NA
Suicidal Behavior in Patients Diagnosed With Bipolar Disorder
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI