Phase 2
N=44
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT01207908 ↗Enrolled (actual)
44
Serious AEs
2.3%
Results posted
Jan 2021
Primary outcome: Primary: Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline — 3.4; -5.1 meters — p=0.53
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- IGF-1 (Drug)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- Male
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- Oct 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline |
3.4; -5.1 | 0.53 |
| SECONDARY Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline |
2.60; -0.06 | <0.0001 sig |
| SECONDARY Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline |
-0.94; -0.4 | 0.53 |
Summary
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
Eligibility 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): 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
Data sourced from ClinicalTrials.gov (NCT01207908). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.