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A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT05515536 · View on ClinicalTrials.gov ↗
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05515536
The ClinicalTrials.gov registry entry for NCT05515536 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 130 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is PTC Therapeutics, which has 15 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05515536 reports 14 study locations spanning 7 distinct geographic areas — top geographies include Other, Florida, Quebec. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05515536?
The interventions under investigation include: Vatiquinone (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05515536?
This trial is sponsored by PTC Therapeutics, which has 15 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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