Background:
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.
Methods:
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.
Results:
There was no difference between treatment groups in the primary or secondary outcome measures after the 2-year treatment period.
Conclusions:
Insulin-like growth factor type I does not provide benefit for patients with amyotrophic lateral sclerosis.
GLOSSARY
= 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.