A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.

PTC Identifier:
PTC743-NEU-005-FA
ClinicalTrials.gov Identifier:
NCT05485987
EudraCT Identifier:
2022-003265-38
EU CT ID:
N/A
Study Completed

Trial Purpose

Age
N/A - 6 Years
Sex
Both (Female and Male)
Healthy Volunteers
No

Key Participation Requirements

Inclusion Criteria:

  • Friedreich ataxia diagnosis (homozygous for GAA repeat expansion in intron-1 of FXN gene), confirmed by clinical genetic testing.
  • Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days before the Baseline Visit and for the duration of the study; any possible discontinuation of anticoagulants should be monitored and indicated by a specialist (for example, cardiologist, neurologist, or hematologist), and discontinuation will be noted by the prescribing physician.

Exclusion Criteria:

  • Previous treatment with vatiquinone.
  • Allergy to vatiquinone or sesame oil.
Inclusion and Exclusion Criteria
Inclusion criteria
  • Friedreich ataxia diagnosis (homozygous for GAA repeat expansion in intron-1 of FXN gene), confirmed by clinical genetic testing.
  • Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days before the Baseline Visit and for the duration of the study; any possible discontinuation of anticoagulants should be monitored and indicated by a specialist (for example, cardiologist, neurologist, or hematologist), and discontinuation will be noted by the prescribing physician.
  • Must be able to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John’s wort, grapefruit juice, or any grapefruit product) for at least 30 days prior to enrollment and for the duration of the study.
  • Must be able to take vatiquinone oral solution with food.
Exclusion criteria
  • Previous treatment with vatiquinone.
  • Allergy to vatiquinone or sesame oil.
  • Ejection fraction <50%.
  • Participation in any other interventional clinical trial or receipt of any study drug in any other clinical trial within 60 days prior to the Baseline Visit. Participants may be rescreened after the exclusionary period of 60 days has passed.
  • Concomitant use of interventional CoQ10, vitamin E, or any approved or non-approved medication for FA within 30 days prior to the Baseline Visit. These prohibited medications can be discontinued at the Screening Visit.
  • Note: Other inclusion and exclusion criteria may apply.

Trial Summary

Total Participants

This is the number of individuals who are taking part in the trial.

Total Participants
5

Trial Dates

These are the start and end dates for the trial. Note that future dates are estimates that may be updated before or during the trial.

Trial Dates
Oct 2022 - Aug 2024

Phase

These are the different steps that a treatment goes through during clinical development before it is approved for use.

Phase
Phase 2

Placebo Controlled

In this type of study, some participants receive a “placebo,” which looks like the study treatment, but does not actually contain the active treatment.

Placebo Controlled
No

Product

This is any type of drug, device, or other treatment that is being studied in the trial.

Product
Vatiquinone

Accepts Healthy Volunteers

Healthy volunteers are individuals who participate in the trial and do not have that specific disease or condition.

Accepts Healthy Volunteers
No
Discuss with the trial center for details

Trial Design

Trial Type

This is the general nature of the study.

Trial Type
Interventional

Interventional

Participants in these studies are subject to an intervention by researchers. These trials are used to understand the effects of a particular intervention.

Discuss with the trial center for details

Blinding

Study participants know which treatments they might get if they join the trial, however during a trial they may not be informed which treatment they are actually taking. The act of not disclosing this information to the participant is known as “blinding.”

Blinding
N/A
Discuss with the trial center for details

Intervention Type

This is a medicine, device, or other type of treatment that is being studied or is already approved for use. Interventions can also include behavior changes, like diet and exercise.

Intervention Type
Drug

Allocation

This determines how study participants are assigned their treatment.

Allocation
N/A

Trial Purpose

This is the rationale behind why the clinical trial is being performed.

Trial Purpose
Treatment

Trial Arms

These are the groups of participants in a clinical study. Each different “arm” is assigned a different treatment to use. Different types of trial arms in studies can include:

Trial Arms
Experimental

Experimental arm

Experimental arm: The group assigned to use the study treatment

Primary Outcome

Primary Outcome

This is the most important measurement of the study and is used to help researchers evaluate the effect of a treatment.

Plasma Concentration of Vatiquinone

Timeframe: Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24

Area Under the Curve (AUC) of Vatiquinone

Timeframe: Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24

Number of Participants With Adverse Events

Timeframe: Baseline up to Week 76

Secondary Outcome

Secondary Outcome

This measurement is a secondary measurement and is still important in helping researchers evaluate the effect of a treatment.

Not Available

Contact Information

Location
Status
Location
The Children's Hospital of Philadelphia
Philadelphia, PA, United States, 19104
Status
Active, not recruiting