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Safety and Efficacy Study of IGF-1 in 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. Identifier: NCT01207908
Recruitment Status : Unknown
Verified June 2012 by Children's Hospital Medical Center, Cincinnati.
Recruitment status was:  Active, not recruiting
First Posted : September 23, 2010
Last Update Posted : June 13, 2012
Charley's Fund
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

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

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

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy
Study Start Date : November 2010
Estimated Primary Completion Date : October 2012
Estimated Study Completion Date : June 2013

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. Six Minute Walk Test (6MWT) [ Time Frame: 6 months ]
    IGF-1 treatment motor function assessment by the difference in the distance traveled using the 6MWT.

Secondary Outcome Measures :
  1. Growth Rate [ Time Frame: 6 months ]
  2. Difference in motor function [ Time Frame: 6 months ]
    Assessed by North Star Ambulatory Assessment, timed functional tests

  3. Safety parameters [ Time Frame: 6 months ]
    Measuring pulmonary function, cardiac function, and safety labs.

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01207908

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United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Charley's Fund
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Principal Investigator: Meilan Rutter, MD Children's Hospital Medical Center, Cincinnati
Additional Information:
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Responsible Party: Children's Hospital Medical Center, Cincinnati Identifier: NCT01207908    
Other Study ID Numbers: 2010-1491
First Posted: September 23, 2010    Key Record Dates
Last Update Posted: June 13, 2012
Last Verified: June 2012
Keywords provided by Children's Hospital Medical Center, Cincinnati:
Duchenne Muscular Dystrophy
Insulin like growth factor
6 minute walk test
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Growth Substances
Physiological Effects of Drugs