Neuromuscular DiseasePhase 2NCT01074359
Safety and Efficacy Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation
A Phase 2 study testing A0001 (alpha-tocopherolquinone) in people with Neuromuscular Disease, run by Penwest Pharmaceuticals Co. at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Neuromuscular Disease
- What is being tested?
- A0001 (alpha-tocopherolquinone)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Feb 2010to Nov 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of neuromuscular symptoms due to the A3243G mitochondrial DNA point mutation
- PCR/ATP ratio of <1.9 following the Cardiac MRS at screening
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
University of Newcastle upon Tyne
Newcastle, Framlington Place, NE2 4HH
About this study
Official title: A Phase 2a, Double Blind, Randomized, Placebo-controlled, 28 Day, Two-arm, Parallel Group Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation and Evidence of Impaired Mitochondrial Function
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.
- Conditions
- Neuromuscular Disease
- Being tested
- A0001 (alpha-tocopherolquinone), Placebo
- Keywords
- A3243G Mitochondrial DNA Point Mutation
- Medical subject headings
- Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
A0001 (alpha-tocopherolquinone)Drug
28 days (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
PlaceboDrug
28 days of placebo oral capsules. Treatment taken twice daily with meals.
| Study group | Role | What they receive |
|---|---|---|
| A0001 | Experimental | A0001 (0.75 g BID) Receives: Drug: A0001 (alpha-tocopherolquinone) |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement in the rate of ATP recovery ("Vmax") in cardiac muscle as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS)
Measured: Baseline and Day 28
Secondary outcomes (8)
Improvement in cardiac structure and function as measured by Magnetic Resonance Imaging (MRI)
Measured: Baseline and Day 28
Exercise tolerance as measured by a 6 minute walk test
Measured: Baseline, Day 14 and Day 28
Improvement in the rate of Maximal ATP recovery (Vmax) as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS) MRI of calf muscle during a standardized isolated calf muscle procedure of 2 bouts of plantar flexion exercise
Measured: Baseline and Day 28
Fasting blood lactate, fasting blood glucose, fasting blood insulin , fasting blood HbA1c levels
Measured: Baseline, Day 14 and Day 28
Mitochondrial disease severity (NMDAS)
Measured: Baseline and Day 28
Quality of life (SF-36® Health Survey Questionnaire)
Measured: Baseline and Day 28
Global impression of clinical severity
Measured: Baseline, Day 14 and Day 28
Modified fatigue impact scale
Measured: Baseline, Day 14 and Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2010 (estimated)
- Study startFeb 2010
- Primary completionNov 2010
- Study completionNov 2010
Why it stopped: Terminated for Commercial Reasons. There were no safety issues involved in the decision to terminate the study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Penwest Pharmaceuticals Co.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Patrick F Chinnery, Principal investigator, University of Newcastle Upon-Tyne
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01074359
- Sponsor's protocol ID
- MEL01
- Status last verified
- Apr 2011
- First submitted
- 22 Feb 2010
- Last updated
- 22 Apr 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Double Blind, Randomized, Placebo-controlled, 28 Day, Two-arm, Parallel Group Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation and Evidence of Impaired Mitochondrial Function
- ClinicalTrials.gov ID
- NCT01074359
- Acronym / sponsor ID
- MEL01
- Conditions
- Neuromuscular Disease
- Keywords
- A3243G Mitochondrial DNA Point Mutation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated for Commercial Reasons. There were no safety issues involved in the decision to terminate the study.
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Penwest Pharmaceuticals Co.Industry (pharma, biotech, devices)
- Study start
- Feb 2010
- Primary completion
- Nov 2010 (actual)
- Study completion
- Nov 2010 (actual)
- First posted
- 24 Feb 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A0001 | Experimental | Drug: A0001 (alpha-tocopherolquinone)A0001 (0.75 g BID) |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of neuromuscular symptoms due to the A3243G mitochondrial DNA point mutation
- PCR/ATP ratio of <1.9 following the Cardiac MRS at screening
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| A0001 (alpha-tocopherolquinone)28 days (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals. | Drug | A0001 |
| Placebo28 days of placebo oral capsules. Treatment taken twice daily with meals. | Drug | Placebo |
Primary outcome
Improvement in the rate of ATP recovery ("Vmax") in cardiac muscle as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS)
Time frame: Baseline and Day 28
8 secondary outcomes
Improvement in cardiac structure and function as measured by Magnetic Resonance Imaging (MRI)
Time frame: Baseline and Day 28
Exercise tolerance as measured by a 6 minute walk test
Time frame: Baseline, Day 14 and Day 28
Improvement in the rate of Maximal ATP recovery (Vmax) as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS) MRI of calf muscle during a standardized isolated calf muscle procedure of 2 bouts of plantar flexion exercise
Time frame: Baseline and Day 28
Fasting blood lactate, fasting blood glucose, fasting blood insulin , fasting blood HbA1c levels
Time frame: Baseline, Day 14 and Day 28
Mitochondrial disease severity (NMDAS)
Time frame: Baseline and Day 28
Quality of life (SF-36® Health Survey Questionnaire)
Time frame: Baseline and Day 28
Global impression of clinical severity
Time frame: Baseline, Day 14 and Day 28
Modified fatigue impact scale
Time frame: Baseline, Day 14 and Day 28
Results and safety
The study was stopped early (reason given: Terminated for Commercial Reasons. There were no safety issues involved in the decision to terminate the study.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2011.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Patrick F Chinnery principal investigator
University of Newcastle Upon-Tyne
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing A0001 (alpha-tocopherolquinone) in people with Neuromuscular Disease, run by Penwest Pharmaceuticals Co. at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Apr 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Terminated for Commercial Reasons. There were no safety issues involved in the decision to terminate the study.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement in the rate of ATP recovery ("Vmax") in cardiac muscle as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS) (time frame: Baseline and Day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2011.