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Subcutaneous IGF-1 is not beneficial in 2-year ALS trial
E J Sorenson1, A J Windbank, J N Mandrekar
1Department of Neurology, Mayo Clinic, 200 1st St. SW, Rochester, MN 55905, USA. sorenson.eric@mayo.edu
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.
