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

IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy

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

Study Overview

• Status: Completed
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Drug: IGF-1

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.

• Study Type: Interventional
• Enrollment (Actual): 44
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / 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 Names: Increlex (mecasermin [rDNA origin] injection)
No Intervention: Standard steroid treatment alone; Standard daily steroid treatment for DMD

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline	Outcome Measure Data Table shows change of 6-Minute Walk Distance at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline	Outcome Measure Data Table shows change of height velocity at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).	6 months
Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline	Outcome Measure Data Table shows change of North Star Ambulatory Assessment (NSAA) score at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1). The NSAA is a 17-item scale that grades performance of various functional skills on a scale from 0 (unable), 1 (complete independently but with modifications), and 2 (complete without compensation). The range of NSAA score is from 0 to 34. The higher score indicates better motor function.	6 months

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborators: Ipsen; Charley's Fund
• Investigators: Principal Investigator: Meilan Rutter, MD, Children's Hospital Medical Center, Cincinnati

Publications and helpful links

Helpful Links

• genetics home reference
• Duchenne and Becker Muscular Dystrophy
• Muscular Dystrophy
• Medline

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2010
• Primary Completion (Actual): October 1, 2012
• Study Completion (Actual): June 1, 2013

Study Registration Dates

• First Submitted: September 22, 2010
• First Submitted That Met QC Criteria: September 22, 2010
• First Posted (Estimate): September 23, 2010

Study Record Updates

• Last Update Posted (Actual): January 20, 2021
• Last Update Submitted That Met QC Criteria: December 28, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Keywords: Duchenne Muscular Dystrophy; DMD; 6 minute walk test; Insulin like growth factor
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne; Physiological Effects of Drugs; Growth Substances; Mecasermin
• Other Study ID Numbers: 2010-1491