welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
CoQ10 and Prednisone in Non-Ambulatory DMD
study id #: NCT00308113
condition: 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.
intervention: Prednisone, Coenzyme Q10
mechanism of action: Glucocorticoid to delay decline in muscle strength; Antioxidant to reduce inflammation
last updated: November 21, 2018
start date: April 2007
estimated completion: November 2010
phase of development: Phase 3
size / enrollment: 3
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.
- 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.
- Compare Side Effect Profiles of the Three Study Groups [Time Frame: 12 months]
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.
• 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
• 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
Givinostat in Duchenne’s Muscular Dystrophy Long-term Safety and Tolerability StudyThis is an open label, long-term safety,...
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double...Background: Duchenne muscular dystrophy...
Catabasis Quarterly: Updates on edasalonexent and clinical trialsWe are pleased to announce that we have ...
Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilo...Wave Life Sciences Ltd., a biotechnology...
Study of ataluren in previously treated participants with nonsense mutation dystrophinopathy (nmDBMD)The objective of this study is to assess...
PTC Therapeutics Phase III Duchenne Muscular Dystrophy (DMD) Clinical TrialPTC Therapeutics, Inc. is seeking pa...
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular DystrophyThe purpose of this study is to explore ...
Design of a phase 3 trial to evaluate the long-term efficacy and safety of ataluren in patients with nonsense mutati...Ataluren is conditionally approved by th...