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

What Was Tested

The trial (phase 1, phase 2) is built around IGF-1 LR3.

It had two arms (groups): IGF-1 (experimental) and Standard steroid treatment alone (no intervention).

IGF-1 plus standard steroid treatment.

Standard daily steroid treatment for DMD.

The registry classes the interventions as drug.

More on the Plan

IGF-1 stimulates both the proliferation and differentiation of skeletal muscle cells and is thus important for muscle repair and regeneration.

Who Took Part

Enrollment: 44 actual participants took part.

It was open to men only aged from 5 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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). 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.

The full record lists 9 inclusion and 10 exclusion criteria; among the exclusions: current or prior treatment with growth hormone or igf-1 therapy; pubertal (based on clinical tanner staging examination); congestive cardiac failure.

How the Study Was Run

The set-up was randomized, parallel-group and single-blind.

Random assignment meant chance, not the researchers, decided who got IGF-1 LR3 and who got the comparison, which limits bias.

The IGF-1 LR3 group and the comparison group ran side by side rather than switching.

In this single-blind IGF-1 LR3 study one party knew the assignments and the other did not.

Those kept unaware of assignments: outcomes assessor.

It started in 2010-11 with completion listed for 2012-10.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline (time frame: 6 months).

It also tracked 2 secondary outcomes, for example Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline and Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2, randomized trial of IGF-1 LR3 in 44 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the IGF-1 LR3 compound page, all human trials.