Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia

Penwest Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Friedreich's Ataxia

Treatments: Drug: alpha-tocopherolquinone (A0001); Drug: placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01035671

FRD02

Details and patient eligibility

About

This is a Phase 2a double-blind, placebo-controlled study with two dose levels of A0001 given twice daily for 28 days. Potential subjects will be screened first to determine eligibility, after which they will be randomized to receive either a high dose of A0001, a low dose of A0001 or placebo for 28 days.

Eligible subjects will return within 21 days of screening for the baseline visit and randomization to one of three potential treatments. The subjects will be required to take 3 capsules of study medication in the morning with a morning meal and 3 capsules of study medication at night with an evening meal for 28 days. Additional visits to the clinic are planned for Day 14 and Day 28, at which time a number of clinical and biochemical assessments will be done.

Enrollment: 42 estimated patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Individuals with genetically confirmed Friedreich's Ataxia (GAA or point mutation)
• Impaired Glucose Tolerance, measured by Oral GTT

Exclusion criteria

• Overt Diabetes Mellitus
• Presence of clinically significant cardiovascular disease

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

42 participants in 3 patient groups, including a placebo group

Low Dose

Experimental group

Description:

A0001 (0.5 g BID)

Treatment:

Drug: alpha-tocopherolquinone (A0001)

Drug: alpha-tocopherolquinone (A0001)

High Dose

Experimental group

Description:

A0001 (0.75 g BID)

Treatment:

Drug: alpha-tocopherolquinone (A0001)

Drug: alpha-tocopherolquinone (A0001)

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: placebo