Duchenne Muscular DystrophyPhase 2/3NCT01126697
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
A Phase 2/3 study testing Coenzyme Q10 and Lisinopril in people with Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 7 sites in 3 countries.
Trial summary
The study will include 120 participants aged 8 and up with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies that have no clinical cardiac symptoms. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- What is being tested?
- Coenzyme Q10 and Lisinopril
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- 7 sitesin 3 countries
- Study dates
- Feb 2010to Dec 2017
- Phase
- Phase 2/3
- 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 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 8 years of age or older
- Confirmed genetic diagnosis of Duchenne, Becker, or Limb Girdle muscular dystrophy
- Beta-blocker naïve
- Screening Doppler echocardiographic MPI measurement greater than or equal to 0.40 for the highest MPI value (spectral and tissue) or circumferential strain measured by STE that is less negative than or equal to - 23
- Normal left ventricular fractional shortening (≥28%) and no clinical cardiac symptoms
- Has not participated in other therapeutic research protocol within the last 6 months prior to screening
- Ability to swallow tablets
Who cannot join
- Spine curvature greater than 30% (based on the x-ray performed at screening)
- History of significant concomitant illness or significant impairment of renal or hepatic function
- History of hypersensitivity to ACE inhibitors
- History of idiopathic or hereditary angioedema or a history of angioedema with prior ACE inhibitor use
- Use of carnitine, creatine, glutamine, or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) in the 3-months prior to enrollment
- CoQ10 and/or ACE inhibitor use for a duration greater than 6 months
Show all 9Show fewer
- CoQ10 and/or ACE inhibitor use in the 3-months prior to enrollment
- CoQ10 serum level of 2.5 ug/ml or higher
- Investigator assessment of inability to comply with protocol
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
United States5 sites
Carolinas Medical Center
Charlotte, North Carolina
Lurie Children's Hospital
Chicago, Illinois
University of Tennessee
Memphis, Tennessee
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
The study will include 120 participants aged 8 and up with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies that have no clinical cardiac symptoms. Participants will be randomized to one of four arms: Arm 1 CoQ10 alone, Arm 2 Lisinopril alone, Arm 3 CoQ10 and Lisinopril or Arm 4 No study medication. Randomization will be stratified by ambulatory status and corticosteroid use. The primary outcome for the study is the myocardial performance index (MPI), measured by standard Doppler echocardiography. The study will last 24 months with visits at Months 0.5,1.5, 6, 12, 18 and 24.
Following completion of the Clinical Trial of Coenzyme Q10 and Lisinopril, participants will be offered participation in a companion protocol: PITT1215 A Natural History Companion Study to PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies. The objective of this study is to evaluate the longitudinal natural history of DMD, BMD, and LGMD2I and to evaluate the effects of Coenzyme Q10 and/or Lisinopril on prevention of cardiac dysfunction in these disorders.This will be an 18-month longitudinal natural history study designed to accompany the Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies.
- Being tested
- Coenzyme Q10 and Lisinopril
- Keywords
- Cardiac, Muscular Dystrophy, Randomized
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Limb-Girdle, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Coenzyme Q10 and LisinoprilDrug
Arm 1. Coenzyme Q10: taken once a day each morning by mouth OR Arm 2. Lisinopril: taken once a day each morning by mouth OR Arm 3. Coenzyme Q10 and lisinopril: each taken once a day in the morning by mouth OR Arm 4. Enhanced Standard Care (more doctor visits, muscle and breathing testing, and x-rays for monitoring, but no study medication).
| Study group | Role | What they receive |
|---|---|---|
| Enhanced standard of care | No intervention | Not described |
| Lisinopril | Active comparator | Not described Receives: Drug: Coenzyme Q10 and Lisinopril |
| Coenzyme Q10 | Active comparator | Not described Receives: Drug: Coenzyme Q10 and Lisinopril |
| Coenzyme Q10 and Lisinopril | Active comparator | Not described Receives: Drug: Coenzyme Q10 and Lisinopril |
What is being measured
The primary outcome is the main question the study is designed to answer.
myocardial performance index (MPI)
The MPI is a sensitive, quantifiable, noninvasive measure of global ventricular function that is independent of cardiac geometry and heart rate. MPI is collected through standard echocardiogram assessment. MPI is a ratio of the total time spent in isovolumic activity (isovolumic contraction time and isovolumic relaxation time) to the time spent in ventricular ejection.
Measured: every 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2010 (estimated)
- Study startFeb 2010
- Primary completionDec 2017
- Study completionDec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Enrolment
- 63 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01126697
- Sponsor's protocol ID
- PITT0908
- Status last verified
- Jun 2018
- First submitted
- 18 May 2010
- Last updated
- 15 Jun 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
- ClinicalTrials.gov ID
- NCT01126697
- Acronym / sponsor ID
- PITT0908
- Keywords
- Cardiac, Muscular Dystrophy, Randomized
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Collaborators
- United States Department of Defense
- Study start
- Feb 2010
- Primary completion
- Dec 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 20 May 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.
The study will include 120 participants aged 8 and up with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies that have no clinical cardiac symptoms. Participants will be randomized to one of four arms: Arm 1 CoQ10 alone, Arm 2 Lisinopril alone, Arm 3 CoQ10 and Lisinopril or Arm 4 No study medication. Randomization will be stratified by ambulatory status and corticosteroid use. The primary outcome for the study is the myocardial performance index (MPI), measured by standard Doppler echocardiography. The study will last 24 months with visits at Months 0.5,1.5, 6, 12, 18 and 24.
Following completion of the Clinical Trial of Coenzyme Q10 and Lisinopril, participants will be offered participation in a companion protocol: PITT1215 A Natural History Companion Study to PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies. The objective of this study is to evaluate the longitudinal natural history of DMD, BMD, and LGMD2I and to evaluate the effects of Coenzyme Q10 and/or Lisinopril on prevention of cardiac dysfunction in these disorders.This will be an 18-month longitudinal natural history study designed to accompany the Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Enhanced standard of care | No intervention | – |
| Lisinopril | Active comparator | Drug: Coenzyme Q10 and Lisinopril |
| Coenzyme Q10 | Active comparator | Drug: Coenzyme Q10 and Lisinopril |
| Coenzyme Q10 and Lisinopril | Active comparator | Drug: Coenzyme Q10 and Lisinopril |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 8 years of age or older
- Confirmed genetic diagnosis of Duchenne, Becker, or Limb Girdle muscular dystrophy
- Beta-blocker naïve
- Screening Doppler echocardiographic MPI measurement greater than or equal to 0.40 for the highest MPI value (spectral and tissue) or circumferential strain measured by STE that is less negative than or equal to - 23
- Normal left ventricular fractional shortening (≥28%) and no clinical cardiac symptoms
Key exclusion criteria (9 listed)
- Spine curvature greater than 30% (based on the x-ray performed at screening)
- History of significant concomitant illness or significant impairment of renal or hepatic function
- History of hypersensitivity to ACE inhibitors
- History of idiopathic or hereditary angioedema or a history of angioedema with prior ACE inhibitor use
- Use of carnitine, creatine, glutamine, or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) in the 3-months prior to enrollment
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 |
|---|---|---|
| Coenzyme Q10 and LisinoprilArm 1. Coenzyme Q10: taken once a day each morning by mouth OR Arm 2. Lisinopril: taken once a day each morning by mouth OR Arm 3. Coenzyme Q10 and lisinopril: each taken once a day in the morning by mouth OR Arm 4. Enhanced Standard Care (more doctor visits, muscle and breathing testing, and x-rays for monitoring, but no study medication). | Drug | Coenzyme Q10, Coenzyme Q10 and Lisinopril, Lisinopril |
Primary outcome
myocardial performance index (MPI)
Time frame: every 6 months
The MPI is a sensitive, quantifiable, noninvasive measure of global ventricular function that is independent of cardiac geometry and heart rate. MPI is collected through standard echocardiogram assessment. MPI is a ratio of the total time spent in isovolumic activity (isovolumic contraction time and isovolumic relaxation time) to the time spent in ventricular ejection.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2018.
- CINRG Network WebsiteLink provided by the sponsor.
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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.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Coenzyme Q10 and Lisinopril in people with Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 7 sites in 3 countries.
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 7 inclusion and 9 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: finished, as of the sponsor's last update on 15 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Canada, Japan. 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 combined 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.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
myocardial performance index (MPI) (time frame: every 6 months). 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 15 Jun 2018.