Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia - Full Text View

Sponsor:	Penwest Pharmaceuticals Co.
Information provided by:	Penwest Pharmaceuticals Co.
ClinicalTrials.gov Identifier:	NCT01035671

Purpose

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.

Condition	Intervention	Phase
Friedreich's Ataxia	Drug: alpha-tocopherolquinone (A0001) Drug: placebo	Phase II

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
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

Resource links provided by NLM:

Genetics Home Reference related topics: ataxia neuropathy spectrum childhood myocerebrohepatopathy spectrum deoxyguanosine kinase deficiency Friedreich ataxia infantile-onset spinocerebellar ataxia Marinesco-Sjögren syndrome mitochondrial neurogastrointestinal encephalopathy disease myoclonic epilepsy myopathy sensory ataxia spinocerebellar ataxia type 1 spinocerebellar ataxia type 2 spinocerebellar ataxia type 3 spinocerebellar ataxia type 6

MedlinePlus related topics: Diabetes Medicines Friedreich's Ataxia

U.S. FDA Resources

Further study details as provided by Penwest Pharmaceuticals Co.:

Primary Outcome Measures:

Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Sensitivity index (SI) calculated from IVGTT [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]
Glucose effectiveness (SG) calculated from IVGTT [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]
AIRg for glucose and insulin during IVGTT [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]
Fasting Glucose, Insulin and Lactate [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]
HbA1C [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]
Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark) [ Time Frame: Baseline, Day 14 and Day28 ] [ Designated as safety issue: No ]
Specific Activity of Complex 1 in whole blood [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]
Timed 25 Foot Walk Test [ Time Frame: Baseline and Day 28 ] [ Designated as safety issue: No ]
FARS/neurological exam [ Time Frame: Baseline and Day 28 ] [ Designated as safety issue: No ]
9-Hole Peg Test [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]
Vision Low Contrast Letter Acuity Test [ Time Frame: Baseline and Day 28 ] [ Designated as safety issue: No ]
Global Impression of Clinical Severity [ Time Frame: Baseline, Day 14 and Day 28 ] [ Designated as safety issue: No ]
Modified Fatigue Impact Scale [ Time Frame: Baseline and Day 28 ] [ Designated as safety issue: No ]
Activities of Daily Living [ Time Frame: Baseline and Day 28 ] [ Designated as safety issue: No ]
SF-36® [ Time Frame: Baseline and Day 28 ] [ Designated as safety issue: No ]

Estimated Enrollment:	42
Study Start Date:	December 2009
Study Completion Date:	March 2011
Primary Completion Date:	February 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Low Dose A0001 (0.5 g BID)	Drug: alpha-tocopherolquinone (A0001) 28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
Experimental: High Dose A0001 (0.75 g BID)	Drug: alpha-tocopherolquinone (A0001) 28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
Placebo Comparator: Placebo Placebo	Drug: placebo 28 days of placebo oral capsules. Treatment taken twice daily with meals.

Eligibility

Ages Eligible for Study:	18 Years to 60 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01035671

Locations

United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104

Sponsors and Collaborators

Penwest Pharmaceuticals Co.

Investigators

Principal Investigator:

David Lynch, MD, PhD

Children's Hospital of Philadelphia

More Information

No publications provided

Responsible Party:	Thomas Sciascia, MD/Chief Medical Officer and VP Clinical Operations and Regulatory Affairs, Penwest Pharmaceuticals Co.
ClinicalTrials.gov Identifier:	NCT01035671 History of Changes
Other Study ID Numbers:	FRD02
Study First Received:	December 17, 2009
Last Updated:	April 21, 2011
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases

Metabolic Diseases
Tocopherylquinone
Vitamin E
Anticoagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions
Antihypertensive Agents
Cardiovascular Agents
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Vitamins
Micronutrients

ClinicalTrials.gov processed this record on February 08, 2012