Previous human clinical trials of insulin-like growth factor type I (IGF-1) in amyotrophic lateral sclerosis (ALS) have been inconsistent. This phase III, randomized, double-blind, placebo-controlled study was undertaken to address whether IGF-1 benefited patients with ALS.
A total of 330 patients from 20 medical centers were randomized to receive 0.05 mg/kg body weight of human recombinant IGF-1 given subcutaneously twice daily or placebo for 2 years. The primary outcome measure was change in their manual muscle testing score. Secondary outcome measures included tracheostomy-free survival and rate of change in the revised ALS functional rating scale. Intention to treat analysis was used.
There was no difference between treatment groups in the primary or secondary outcome measures after the 2-year treatment period.
Insulin-like growth factor type I does not provide benefit for patients with amyotrophic lateral sclerosis.
= amyotrophic lateral sclerosis;
= revised ALS functional rating scale;
= area under the curve;
= deep venous thromboses;
= insulin-like growth factor type I;
= manual muscle testing;
= pulmonary embolisms.