NCT01035671

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
42

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Dec 2009

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2009

Completed
16 days until next milestone

First Submitted

Initial submission to the registry

December 17, 2009

Completed
4 days until next milestone

First Posted

Study publicly available on registry

December 21, 2009

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2011

Completed
28 days until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2011

Completed
Last Updated

April 22, 2011

Status Verified

April 1, 2011

Enrollment Period

1.2 years

First QC Date

December 17, 2009

Last Update Submit

April 21, 2011

Conditions

Friedreich's Ataxia

Outcome Measures

Primary Outcomes (1)

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

    Baseline, Day 14 and Day 28

Secondary Outcomes (15)

  • Sensitivity index (SI) calculated from IVGTT

    Baseline, Day 14 and Day 28

  • Glucose effectiveness (SG) calculated from IVGTT

    Baseline, Day 14 and Day 28

  • AIRg for glucose and insulin during IVGTT

    Baseline, Day 14 and Day 28

  • Fasting Glucose, Insulin and Lactate

    Baseline, Day 14 and Day 28

  • HbA1C

    Baseline, Day 14 and Day 28

  • +10 more secondary outcomes

Study Arms (3)

Low Dose

EXPERIMENTAL

A0001 (0.5 g BID)

Drug: alpha-tocopherolquinone (A0001)

High Dose

EXPERIMENTAL

A0001 (0.75 g BID)

Drug: alpha-tocopherolquinone (A0001)

Placebo

PLACEBO COMPARATOR

Placebo

Drug: placebo

Interventions

alpha-tocopherolquinone (A0001)DRUG

28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.

Low Dose
placeboDRUG

28 days of placebo oral capsules. Treatment taken twice daily with meals.

Placebo

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

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

You may not qualify if:

  • Overt Diabetes Mellitus
  • Presence of clinically significant cardiovascular disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (2)

  • 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.

    PMID: 22744651DERIVED

  • 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.

    PMID: 22633670DERIVED

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

tocopherylquinone

Condition Hierarchy (Ancestors)

Spinocerebellar DegenerationsCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • David Lynch, MD, PhD

    Children's Hospital of Philadelphia

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

December 17, 2009

First Posted

December 21, 2009

Study Start

December 1, 2009

Primary Completion

February 1, 2011

Study Completion

March 1, 2011

Last Updated

April 22, 2011

Record last verified: 2011-04

Locations

US(1)