welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
study id #: NCT01126697
condition: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
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.
intervention: Coenzyme Q10 and Lisinopril
mechanism of action: Antioxidant to reduce inflammation and ACE inhibitor to prevent cardiac funtion deterioration
last updated: November 22, 2018
start date: February 2010
estimated completion: December 2017
phase of development: Phase 2/Phase 3
size / enrollment: 63
- 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.
• 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
• 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
Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystroph...The proposed clinical trial is an outgro...
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, ran...Background: Duchenne muscular dystrophy...
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular DystrophyThe main purpose of this study is to tes...
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)The purpose of this study is to see whet...
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Musc...This is a multi-center, randomized, doub...
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)This is a Phase 2, open-label, single ar...
Study of SRP-4045 and SRP-4053 in DMD PatientsThe main objective of this study is to e...
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-infl...We report a first-in-patient study of va...
MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dys...NF-κB is activated from infancy in DMD,...
First Patient Dosed in Phase II Clinical Trial in Duchenne Muscular DystrophyAntisense Therapeutics today announced d...
Catabasis Quarterly: Updates on edasalonexent and clinical trialsHappy New Year! As 2019 kicks off, our P...