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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01207908
• Date of registration: 22/09/2010
• Prospective Registration: Yes
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
• Scientific title: IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy
• Date of first enrolment: November 2010
• Target sample size: 40
• Recruitment status: Active, not recruiting
• URL: http://clinicaltrials.gov/show/NCT01207908
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Meilan Rutter, MD
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion 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):
- 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



Age minimum: 5 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Other: Standard steroid treatment

Drug: IGF-1

Primary Outcome(s)

Six Minute Walk Test (6MWT) [Time Frame: 6 months]

Secondary Outcome(s)

Growth Rate [Time Frame: 6 months]

Difference in motor function [Time Frame: 6 months]

Safety parameters [Time Frame: 6 months]

Secondary ID(s)

2010-1491

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Tercica- Subsidiary of Ipsen

Charley's Fund