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Trial record 3 of 46 for:    Asklepios BioPharmaceutical

Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00428935
Recruitment Status : Completed
First Posted : January 30, 2007
Last Update Posted : February 5, 2013
Sponsor:
Collaborator:
Asklepios Biopharmaceutical, Inc.
Information provided by (Responsible Party):
Jerry R. Mendell, Nationwide Children's Hospital

Tracking Information
First Submitted Date  ICMJE January 26, 2007
First Posted Date  ICMJE January 30, 2007
Last Update Posted Date February 5, 2013
Study Start Date  ICMJE March 2006
Actual Primary Completion Date March 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 4, 2013)
Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: followed for 2 years post injection ]
Physical Exams assessing major organ systems and safety labs (GGT, Bilirubin, Glucose, Amylase, CBC/Diff, AFP, Platelets, PT/PTT, Creatinine, Electrolytes, Total protein, Alkaline phosphatase, and Urinalysis)
Original Primary Outcome Measures  ICMJE
 (submitted: January 26, 2007)
Safety
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 4, 2013)
  • mini-dystrophin gene expression at the site of gene transfer [ Time Frame: 90 days post injection ]
  • Maximal Volume Isometric Contraction Testing as a measure of muscle strength [ Time Frame: out to 2 years post injection ]
Original Secondary Outcome Measures  ICMJE
 (submitted: January 26, 2007)
  • mini-dystrophin gene expression at the site of gene transfer
  • muscle strength evaluated by Maximal Volume Isometric Contraction Testing
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
Official Title  ICMJE Phase 1 Clinical Trial of rAAV2.5-CMV-mini-Dystrophin Gene Vector in Duchenne Muscular Dystrophy
Brief Summary The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
Detailed Description This phase I randomized double blind dose escalation study investigates the safety and efficacy of the mini-dystrophin gene transferred to the biceps muscle for Duchenne muscular dystrophy patients, ages 5 to 12 years of age, using a recombinant adeno-associated virus. Eligible participants must have a known dystrophin gene mutation and may be concurrently treated with corticoid steroids. The mini-dystrophin gene or a placebo agent (normal saline or empty viral capsids) are injected directly into both biceps muscles while under conscious sedation. Following the gene transfer, patients are admitted to the hospital for 48 hours of observation followed by weekly outpatient visits at the Columbus Children's Hospital Neuromuscular Clinic. A bilateral muscle biopsy is preformed following 6 weeks with long term follow up will consisting of bi-annual visits for the next 2 years.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Biological: rAAV2.5-CMV-minidystrophin (d3990)
Recombinant adeno-associated virus (AAV) carrying a truncated human dystrophin gene (mini-dystrophin) expressed from a cytomegalovirus (CMV) promoter.
Study Arms  ICMJE
  • Experimental: Low Dose
    Low dose cohort - 2.0E10 vg/kg
    Intervention: Biological: rAAV2.5-CMV-minidystrophin (d3990)
  • Experimental: High Dose
    High Dose - 1.0E11 vg/kg
    Intervention: Biological: rAAV2.5-CMV-minidystrophin (d3990)
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 26, 2007)
6
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE July 2010
Actual Primary Completion Date March 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Known null mutation of the Dystrophin gene
  • Male age of 5 years or older
  • If taking corticosteroids, must have dose unchanged for the past 3 months
  • Serum creatine kinase elevation greater than 10x normal value (established by Children's Hospital)
  • Progressive, symmetrical proximal muscle weakness of arms and legs

Exclusion Criteria:

  • Unable to cooperate for muscle strength testing
  • Joint contractures that prohibit muscle strength testing
  • Concomitant illness
  • Individuals predisposed to excessive vagal responses (bradyarrhythmia or hypotension)
  • Controlled substance abuse
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 5 Years to 15 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00428935
Other Study ID Numbers  ICMJE CCRI IRB05-00118
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Jerry R. Mendell, Nationwide Children's Hospital
Study Sponsor  ICMJE Nationwide Children's Hospital
Collaborators  ICMJE Asklepios Biopharmaceutical, Inc.
Investigators  ICMJE
Principal Investigator: Jerry R. Mendell, MD Nationwide Children's Hospital
PRS Account Nationwide Children's Hospital
Verification Date February 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP