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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01207908
• Other study ID #: 2010-1491
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: November 2010
• Est. completion date: June 2013

Study information

• Verified date: December 2020
• Source: Children's Hospital Medical Center, Cincinnati
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 44
• Est. completion date: June 2013
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• IGF-1
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio

Sponsors (3)

Lead Sponsor	Collaborator
Children's Hospital Medical Center, Cincinnati	Charley's Fund, Ipsen

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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

External Links

•   Duchenne and Becker Muscular Dystrophy
•   Genetics Home Reference
•   Medline
•   Muscular Dystrophy