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

P

Penwest Pharmaceuticals Co.

Status and phase

Completed
Phase 2

Conditions

Friedreich's Ataxia

Treatments

Drug: placebo
Drug: alpha-tocopherolquinone (A0001)

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)
High Dose
Experimental group
Description:
A0001 (0.75 g BID)
Treatment:
Drug: alpha-tocopherolquinone (A0001)
Placebo
Placebo Comparator group
Description:
Placebo
Treatment:
Drug: placebo

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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