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

Primary Purpose

Friedreich's Ataxia

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
alpha-tocopherolquinone (A0001)
alpha-tocopherolquinone (A0001)
placebo
Sponsored by
Penwest Pharmaceuticals Co.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Friedreich's Ataxia

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept 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

Sites / Locations

  • The Children's Hospital of Philadelphia

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

Low Dose

High Dose

Placebo

Arm Description

A0001 (0.5 g BID)

A0001 (0.75 g BID)

Placebo

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Sensitivity index (SI) calculated from IVGTT
Glucose effectiveness (SG) calculated from IVGTT
AIRg for glucose and insulin during IVGTT
Fasting Glucose, Insulin and Lactate
HbA1C
Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark)
Specific Activity of Complex 1 in whole blood
Timed 25 Foot Walk Test
FARS/neurological exam
9-Hole Peg Test
Vision Low Contrast Letter Acuity Test
Global Impression of Clinical Severity
Modified Fatigue Impact Scale
Activities of Daily Living
SF-36®

Full Information

First Posted
December 17, 2009
Last Updated
April 21, 2011
Sponsor
Penwest Pharmaceuticals Co.
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1. Study Identification

Unique Protocol Identification Number
NCT01035671
Brief Title
Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia
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
Study Type
Interventional

2. Study Status

Record Verification Date
April 2011
Overall Recruitment Status
Completed
Study Start Date
December 2009 (undefined)
Primary Completion Date
February 2011 (Actual)
Study Completion Date
March 2011 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Penwest Pharmaceuticals Co.

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich's Ataxia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
42 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Low Dose
Arm Type
Experimental
Arm Description
A0001 (0.5 g BID)
Arm Title
High Dose
Arm Type
Experimental
Arm Description
A0001 (0.75 g BID)
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
alpha-tocopherolquinone (A0001)
Intervention Description
28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
Intervention Type
Drug
Intervention Name(s)
alpha-tocopherolquinone (A0001)
Intervention Description
28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
28 days of placebo oral capsules. Treatment taken twice daily with meals.
Primary Outcome Measure Information:
Title
Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test
Time Frame
Baseline, Day 14 and Day 28
Secondary Outcome Measure Information:
Title
Sensitivity index (SI) calculated from IVGTT
Time Frame
Baseline, Day 14 and Day 28
Title
Glucose effectiveness (SG) calculated from IVGTT
Time Frame
Baseline, Day 14 and Day 28
Title
AIRg for glucose and insulin during IVGTT
Time Frame
Baseline, Day 14 and Day 28
Title
Fasting Glucose, Insulin and Lactate
Time Frame
Baseline, Day 14 and Day 28
Title
HbA1C
Time Frame
Baseline, Day 14 and Day 28
Title
Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark)
Time Frame
Baseline, Day 14 and Day28
Title
Specific Activity of Complex 1 in whole blood
Time Frame
Baseline, Day 14 and Day 28
Title
Timed 25 Foot Walk Test
Time Frame
Baseline and Day 28
Title
FARS/neurological exam
Time Frame
Baseline and Day 28
Title
9-Hole Peg Test
Time Frame
Baseline, Day 14 and Day 28
Title
Vision Low Contrast Letter Acuity Test
Time Frame
Baseline and Day 28
Title
Global Impression of Clinical Severity
Time Frame
Baseline, Day 14 and Day 28
Title
Modified Fatigue Impact Scale
Time Frame
Baseline and Day 28
Title
Activities of Daily Living
Time Frame
Baseline and Day 28
Title
SF-36®
Time Frame
Baseline and Day 28

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Lynch, MD, PhD
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
22744651
Citation
Lynch DR, Willi SM, Wilson RB, Cotticelli MG, Brigatti KW, Deutsch EC, Kucheruk O, Shrader W, Rioux P, Miller G, Hawi A, Sciascia T. A0001 in Friedreich ataxia: biochemical characterization and effects in a clinical trial. Mov Disord. 2012 Jul;27(8):1026-33. doi: 10.1002/mds.25058. Epub 2012 Jun 28.
Results Reference
derived
PubMed Identifier
22633670
Citation
Friedman LS, Schadt KA, Regner SR, Mark GE, Lin KY, Sciascia T, St John Sutton M, Willi S, Lynch DR. Elevation of serum cardiac troponin I in a cross-sectional cohort of asymptomatic subjects with Friedreich ataxia. Int J Cardiol. 2013 Aug 20;167(4):1622-4. doi: 10.1016/j.ijcard.2012.04.159. Epub 2012 May 26.
Results Reference
derived

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

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