- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01126697
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
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.
Study Overview
Status
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 3
Contacts and Locations
Study Locations
-
-
Alberta
-
Calgary, Alberta, Canada
- Alberta Children's Hospital
-
-
-
-
-
Tokyo, Japan
- National Center of Neurology and Psychiatry
-
-
-
-
District of Columbia
-
Washington, District of Columbia, United States, 20010
- Children's National Medical Center
-
-
Illinois
-
Chicago, Illinois, United States
- Lurie Children's Hospital
-
-
North Carolina
-
Charlotte, North Carolina, United States
- Carolinas Medical Center
-
-
Pennsylvania
-
Pittsburgh, Pennsylvania, United States, 15213
- University of Pittsburgh
-
-
Tennessee
-
Memphis, Tennessee, United States
- University of Tennessee
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Factorial Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Active Comparator: Coenzyme Q10
|
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).
|
No Intervention: Enhanced standard of care
|
|
Active Comparator: Lisinopril
|
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).
|
Active Comparator: Coenzyme Q10 and Lisinopril
|
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).
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
every 6 months
|
Collaborators and Investigators
Collaborators
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Musculoskeletal Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Muscular Disorders, Atrophic
- Muscular Dystrophies
- Muscular Dystrophy, Duchenne
- Muscular Dystrophies, Limb-Girdle
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Antihypertensive Agents
- Enzyme Inhibitors
- Protease Inhibitors
- Protective Agents
- Cardiotonic Agents
- Micronutrients
- Vitamins
- Angiotensin-Converting Enzyme Inhibitors
- Coenzyme Q10
- Lisinopril
- Ubiquinone
Other Study ID Numbers
- PITT0908
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Duchenne Muscular Dystrophy
-
Cairo UniversityCompletedMuscular Dystrophy, Duchenne TypeEgypt
-
Medical University of GdanskRecruitingDuchenne Muscular Dystrophy (DMD)Poland
-
ItalfarmacoCompletedDuchenne Muscular Dystrophy (DMD)Italy
-
Santhera PharmaceuticalsTerminatedDuchenne Muscular Dystrophy (DMD)United States, Spain, Netherlands, Sweden, Germany, France, Belgium, United Kingdom, Italy, Ireland, Switzerland, Austria, Bulgaria, Hungary, Israel
-
Sarepta Therapeutics, Inc.CompletedDuchenne Muscular Dystrophy (DMD)United States
-
Hospital RudolfstiftungOesterreichische MuskelforschungCompletedCarrier of Duchenne Muscular DystrophyAustria
-
General Hospital of Chinese Armed Police ForcesUnknownDuchenne Muscular Dystrophy (DMD)China
-
Chaitanya Hospital, PuneUnknownMuscular Dystrophy | Duchenne Muscular Dystrophy,India
-
University of FloridaU.S. Army Medical Research and Development CommandRecruitingDuchenne Muscular Dystrophy (DMD)United States
-
PTC TherapeuticsCompletedNonsene Mutation Duchenne Muscular DystrophyUnited States
Clinical Trials on Coenzyme Q10 and Lisinopril
-
University of SumerCompletedMale InfertilityIraq
-
University of ShizuokaCompletedHemodialysis | Chronic Renal FailureJapan
-
Central Hospital, Nancy, FranceCompletedCyclic Vomiting SyndromeFrance
-
Columbia UniversityNational Institute of Neurological Disorders and Stroke (NINDS)CompletedAmyotrophic Lateral Sclerosis | Lou Gehrig's DiseaseUnited States
-
Hadassah Medical OrganizationUnknown
-
Peter HumaidanCompleted
-
Johns Hopkins UniversityNational Institute of Neurological Disorders and Stroke (NINDS)CompletedHuntington's DiseaseUnited States
-
Technische Universität DresdenDeutsche Parkinson-Vereinigung e.V.; MSE Pharmazeutika GmbH, Bad HomburgCompleted
-
Mclean HospitalNational Alliance for Research on Schizophrenia and DepressionCompleted
-
National University Hospital, SingaporeCompletedParkinson DiseaseSingapore