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Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
Tercica- Subsidiary of Ipsen
Charley's Fund
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT01207908
First received: September 22, 2010
Last updated: June 12, 2012
Last verified: June 2012
History of Changes
  Purpose

The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).


Condition Intervention Phase
Duchenne Muscular Dystrophy
 Drug: IGF-1
Other: Standard steroid treatment
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:
  • Six Minute Walk Test (6MWT) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    IGF-1 treatment motor function assessment by the difference in the distance traveled using the 6MWT.


Secondary Outcome Measures:
  • Growth Rate [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Difference in motor function [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Assessed by North Star Ambulatory Assessment, timed functional tests

  • Safety parameters [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    Measuring pulmonary function, cardiac function, and safety labs.


Estimated Enrollment: 40
Study Start Date: November 2010
Estimated Study Completion Date: June 2013
Estimated Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: IGF-1
IGF-1 plus standard steroid treatment
 Drug: IGF-1
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
Other Name: Increlex (mecasermin [rDNA origin] injection)
No Intervention: Standard steroid treatment alone Other: Standard steroid treatment
Will complete all study related procedures and evaluations

Detailed Description:

Detailed Description:

DMD is a progressive degenerative muscle disorder for which there is no current cure. Glucocorticoids (GC) are often used to improve motor function and survival but have significant side effects such as growth failure, weight gain, insulin resistance and osteoporosis. IGF-1 stimulates both the proliferation and differentiation of skeletal muscle cells and is thus important for muscle repair and regeneration. IGF-1 offers potential as a therapeutic agent for DMD as it may improve or preserve motor function and reduce GC side effects such as growth failure and insulin resistance.

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • DMD diagnosed with mutational testing and/or complete absence of dystrophin on muscle biopsy
  • Proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
  • Male
  • Age > 5 years of age
  • Bone maturation (assess by bone age x-ray): </= 11 years of age
  • Daily GC (prednisone or deflazacort) therapy for > 12 months
  • Ambulatory
  • Informed consent
  • Willingness and ability to comply with all protocol requirements and procedures

Exclusion Criteria:

  • Current or prior treatment with growth hormone or IGF-1 therapy
  • Non-ambulatory
  • Pubertal (based on clinical Tanner staging examination)
  • Congestive cardiac failure
  • History of intracranial hypertension
  • Daytime ventilatory dependence (non-invasive or tracheostomy)
  • Concomitant therapy - any other medications/supplements that would be considered, in the opinion of the investigators, to affect muscle function, need to have been started 3 months prior to enrollment
  • Patients enrolled in other clinical drug trials
  • Any physical or mental conditions which may, in the investigators'opinions, render the subject unable to complete the tasks of the study appropriately
  • There will be no selection by ethnicity
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01207908

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Tercica- Subsidiary of Ipsen
Charley's Fund
Investigators
Principal Investigator: Meilan Rutter, MD Children's Hospital Medical Center, Cincinnati
  More Information

Additional Information:
Genetics Home Reference  This link exits the ClinicalTrials.gov site
Duchenne and Becker Muscular Dystrophy  This link exits the ClinicalTrials.gov site
Muscular Dystrophy  This link exits the ClinicalTrials.gov site
Medline  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01207908     History of Changes
Other Study ID Numbers: 2010-1491
Study First Received: September 22, 2010
Last Updated: June 12, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by Children's Hospital Medical Center, Cincinnati:
DMD
Duchenne Muscular Dystrophy
Insulin like growth factor
6 minute walk test

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 19, 2013