Safety and Efficacy Study of A0001 in Patients With the A... | NCT01074359

Studies
Safety and Efficacy Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation

NCT01074359

Sponsor

Penwest Pharmaceuticals Co.

Status

Terminated

Last Update Posted

Apr 22, 2011Estimated

Enrollment

30Estimated

Phase

Phase 2

Conditions

Neuromuscular Disease

Interventions

A0001 (alpha-tocopherolquinone)
Placebo

Countries

United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2011

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Terminated for Commercial Reasons. There were no safety issues involved in the decision to terminate the study.

Expanded Access Info

Start Date

Feb 2010

Primary Completion Date

Nov 2010Actual

Completion Date

Nov 2010Actual

First Submitted Date

Feb 22, 2010

First Submission Date that Met QC Criteria

Feb 23, 2010

First Posted Date

Feb 24, 2010Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 21, 2011

Last Update Posted Date

Apr 22, 2011Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

Not provided

Lead Sponsor

Penwest Pharmaceuticals Co.INDUSTRY

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

This is a phase 2a, double-blind, placebo-controlled, single-center study. Twenty-one patients who qualify for the study will be randomly assigned to either active drug or placebo. The study will take place at Newcastle University. Patients will have a 66% chance of getting active drug. Patients will be required to take study treatment orally twice a day for 28 days. A baseline visit will occur within 21 days of screening visit. All patients will be followed for 1 week after completion of study or early withdrawal from the study.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

30Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
A0001	Experimental	A0001 (0.75 g BID)	Drug: A0001 (alpha-tocopherolquinone)
Placebo	Placebo Comparator	Placebo	Drug: Placebo

Interventions

Name	Type	Description	Arm Group Labels	Other Names
A0001 (alpha-tocopherolquinone)	Drug	28 days (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.	A0001

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Improvement in the rate of ATP recovery ("Vmax") in cardiac muscle as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS)		Baseline and Day 28

Secondary Outcomes

Measure	Description	Time Frame
Improvement in cardiac structure and function as measured by Magnetic Resonance Imaging (MRI)		Baseline and Day 28
Exercise tolerance as measured by a 6 minute walk test		Baseline, Day 14 and Day 28

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Diagnosis of neuromuscular symptoms due to the A3243G mitochondrial DNA point mutation
PCR/ATP ratio of <1.9 following the Cardiac MRS at screening

Exclusion Criteria:

Any major illness not due to the A3243G mitochondrial DNA point mutation in the past three months or any significant ongoing chronic medical illness, especially significant central nervous neurological disease limiting capacity to carry out the study
Use of any investigational product within the past 30 days

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

70 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Patrick F Chinnery	University of Newcastle Upon-Tyne	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Newcastle upon Tyne		Newcastle	Framlington Place	NE2 4HH	United Kingdom

Condition Browse Module

MeSH Terms

ID	Term
D009468	Neuromuscular Diseases

Ancestor Terms

ID	Term
D009422	Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C002421	tocopherylquinone

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided