Duchenne Muscular DystrophyPhase 3NCT00308113
CoQ10 and Prednisone in Non-Ambulatory DMD
A Phase 3 study testing Prednisone and Coenzyme Q10 in people with Duchenne Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 1 site in United States.
Trial summary
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. Read more about this study
- Who is it for?
- Ages 10 to 18 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Prednisone and Coenzyme Q10
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2007to Nov 2010
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 10 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 10-18 years
- Non-ambulatory (primary mode of transportation is via wheelchair for 3 years or less)
- Confirmed DMD diagnosis
- Steroid-naive for the 6 months prior to screening
- Stable dose of b-blocker or ACE inhibitor medication for the 6 months prior to screening, if taking either of these medications
- Ability to provide reproducible repeat QMT grip score within 15% of first assessment score
- Has not participated in other therapeutic research protocol within the last 6 months prior to screening
- Ability to swallow tablets
Who cannot join
- Failure to achieve one or more of the diagnostic inclusion criteria cited above
- Symptomatic DMD carrier
- Use of carnitine, other amino acids, creatine, glutamine, CoQ10 or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) within the last 3 months
- History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy
- Positive PPD
- No prior exposure to chickenpox and no immunization against chicken pox
- Baseline serum CoQ10 level of 5.0mg/ml or greater
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
About this study
Official title: PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. This is a 13-month, prospective, randomized study comparing a daily prednisone arm (0.75mg/kg/day), a CoQ10 arm (serum of greater than 2.5 ug/mL) and a combination arm (prednisone and CoQ10) with an enhanced standard of care arm in wheelchair confined males age 10 to 18 years with an established DMD diagnosis.
Read the detailed description
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy affecting 1:3500 male births worldwide. Despite an increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Improvement in the treatment of DMD will depend upon the development of better therapies. Affected boys become symptomatic at 3 to 5 years of age with proximal leg weakness that impairs mobility, ability to get up from a squat, and precludes a normal ability to run. By 8 years of age, some affected boys begin to lose the ability to walk and resort to a wheelchair for mobility. This shift from the ambulant to non-ambulant phase occurs in all boys with a diagnosis of DMD by age 12 years. In this study, participants will be randomized into groups after being screened to determine eligibility. Participants will then be followed for a 12-month investigation period.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Prednisone, Coenzyme Q10
- Keywords
- Muscular dystrophy, Duchenne, CoQ10, prednisone
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
Prednisone 0/75 mg/kg/day.
Coenzyme Q10Dietary supplement
Also called CoQ10
serum levels of greater or equal to 2.5 micrograms/mL.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | CoenzymeQ10 taken once a day each morning by mouth. Receives: Dietary Supplement: Coenzyme Q10 |
| 2 | Active comparator | Prednisone taken once a day each morning by mouth Receives: Drug: Prednisone |
| 3 | Active comparator | CoenzymeQ10 and prednisone each taken once a day in the morning by mouth. Receives: Drug: Prednisone; Dietary Supplement: Coenzyme Q10 |
| 4 | No intervention | Enhanced standard of care. |
What is being measured
The primary outcome is the main question the study is designed to answer.
One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.
Comparing change from baseline of mean systolic wall stress and rate-corrected mean velocity of circumferential shortening in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year. The values are obtained via an echocardiogram read locally at each site.
Measured: 12 months
One Year Change in Pulmonary Function (Forced Expiratory Volume, FEV1 and Forced Vital Capacity, FVC)
Comparing change from baseline levels in pulmonary function (FEV1 and FVC) in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year.
Measured: 12 months
Secondary outcomes (1)
Compare Side Effect Profiles of the Three Study Groups
To compare side effect profiles of the three regimens to the enhanced standard of care group, to include height, weight, weight/height ratio, body mass index, cataract formation, blood glucose, blood pressure, and behavioral changes.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2006 (estimated)
- Study startApr 2007
- Primary completionNov 2010
- Study completionNov 2010
Why it stopped: New enrollment has been suspended, currently following previously enrolled participants
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Duchenne Muscular DystrophyStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Collaborators
- United States Department of Defense
- Responsible party
- Sponsor
- Investigators
- Paula R Clemens, M.D., Study chair, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00308113
- Sponsor's protocol ID
- PITT0503
- Status last verified
- Oct 2013
- First submitted
- 27 Mar 2006
- Last updated
- 8 Nov 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00308113
- Acronym / sponsor ID
- PITT0503
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Muscular dystrophy, Duchenne, CoQ10, prednisone
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: New enrollment has been suspended, currently following previously enrolled participants
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Collaborators
- United States Department of Defense
- Study start
- Apr 2007
- Primary completion
- Nov 2010 (actual)
- Study completion
- Nov 2010 (actual)
- First posted
- 29 Mar 2006 (estimated)
- Results
- Posted 11 Oct 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. This is a 13-month, prospective, randomized study comparing a daily prednisone arm (0.75mg/kg/day), a CoQ10 arm (serum of greater than 2.5 ug/mL) and a combination arm (prednisone and CoQ10) with an enhanced standard of care arm in wheelchair confined males age 10 to 18 years with an established DMD diagnosis.
Detailed description
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy affecting 1:3500 male births worldwide. Despite an increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Improvement in the treatment of DMD will depend upon the development of better therapies. Affected boys become symptomatic at 3 to 5 years of age with proximal leg weakness that impairs mobility, ability to get up from a squat, and precludes a normal ability to run. By 8 years of age, some affected boys begin to lose the ability to walk and resort to a wheelchair for mobility. This shift from the ambulant to non-ambulant phase occurs in all boys with a diagnosis of DMD by age 12 years. In this study, participants will be randomized into groups after being screened to determine eligibility. Participants will then be followed for a 12-month investigation period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Dietary Supplement: Coenzyme Q10CoenzymeQ10 taken once a day each morning by mouth. |
| 2 | Active comparator | Drug: PrednisonePrednisone taken once a day each morning by mouth |
| 3 | Active comparator | Drug: Prednisone, Dietary Supplement: Coenzyme Q10CoenzymeQ10 and prednisone each taken once a day in the morning by mouth. |
| 4 | No intervention | –Enhanced standard of care. |
Who may qualify
- Ages
- 10 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Age 10-18 years
- Non-ambulatory (primary mode of transportation is via wheelchair for 3 years or less)
- Confirmed DMD diagnosis
- Steroid-naive for the 6 months prior to screening
- Stable dose of b-blocker or ACE inhibitor medication for the 6 months prior to screening, if taking either of these medications
Key exclusion criteria (7 listed)
- Failure to achieve one or more of the diagnostic inclusion criteria cited above
- Symptomatic DMD carrier
- Use of carnitine, other amino acids, creatine, glutamine, CoQ10 or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) within the last 3 months
- History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy
- Positive PPD
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 |
|---|---|---|
| PrednisonePrednisone 0/75 mg/kg/day. | Drug | 2, 3 |
| Coenzyme Q10Also: CoQ10serum levels of greater or equal to 2.5 micrograms/mL. | Dietary supplement | 1, 3 |
Primary outcome
One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.
Time frame: 12 months
Comparing change from baseline of mean systolic wall stress and rate-corrected mean velocity of circumferential shortening in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year. The values are obtained via an echocardiogram read locally at each site.
One Year Change in Pulmonary Function (Forced Expiratory Volume, FEV1 and Forced Vital Capacity, FVC)
Time frame: 12 months
Comparing change from baseline levels in pulmonary function (FEV1 and FVC) in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year.
1 secondary outcome
Compare Side Effect Profiles of the Three Study Groups
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Paula R Clemens, M.D. study chair
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Prednisone and Coenzyme Q10 in people with Duchenne Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 1 site in United States.
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 8 inclusion and 7 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 8 Nov 2013. 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 States. 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Oct 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation. (time frame: 12 months). The study lists 2 primary outcomes in all. 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 8 Nov 2013.