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

PTC Therapeutics

Status and phase: Completed; Phase 2

Conditions: Friedreich Ataxia

Treatments: Drug: Vatiquinone

Study type: Interventional

Funder types: Industry

Identifiers

NCT05485987

PTC743-NEU-005-FA

Details and patient eligibility

About

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.

Enrollment: 5 patients

Sex: All

Ages: Under 6 years old

Volunteers: No Healthy Volunteers

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 design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

Vatiquinone

Experimental group

Description:

Participants will receive an oral solution (100 milligrams \[mg\]/milliliter \[mL\]) of vatiquinone (15 mg/kilogram \[kg\] if body weight \<13 kg and 200 mg if body weight ≥13 kg) 3 times a day (TID) for 72 weeks.

Treatment:

Drug: Vatiquinone