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

Penwest Pharmaceuticals Co.42 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Friedreich's Ataxia

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 60 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test