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Completed Phase 2 Interventional

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

ClinicalTrials.gov ID: NCT01035671

Public ClinicalTrials.gov record NCT01035671. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 11, 2026, 7:22 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, 28 Day, Three-arm, Parallel Group Study of A0001 in the Treatment of Subjects With Friedreich's Ataxia

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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 identification

NCT ID
NCT01035671
Recruitment status
Completed
Study type
Interventional
Phase
Phase 2
Lead sponsor
Penwest Pharmaceuticals Co.
Industry
Enrollment
42 participants

Conditions and interventions

Eligibility (public fields only)

Age range
18 Years to 60 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Nov 30, 2009
Primary completion
Jan 31, 2011
Completion
Feb 28, 2011
Last update posted
Apr 21, 2011

2009 – 2011

United States locations

U.S. sites
1
U.S. states
1
U.S. cities
1
Facility City State ZIP Site status
The Children's Hospital of Philadelphia Philadelphia Pennsylvania 19104

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01035671, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Apr 21, 2011 · Synced Sep 11, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01035671 live on ClinicalTrials.gov.

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