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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 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.

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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

Friedreich Ataxia

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

FieldValue
Enrollment Target 130 participants
Start Date 2022-12-08
Est. Completion 2027-12-31
Phase Phase 3

Sponsor

PTC Therapeutics

52 total trials

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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, categorized as Phase 3. The registered enrollment target is 130 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 440-participant average among 14 other Friedreich Ataxia trials with a reported enrollment target (70% lower). The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.

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 reports 14 study locations spanning 7 distinct geographic areas - top geographies include 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.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.