Subcutaneous IGF-1 is not beneficial in 2-year ALS trial
E J Sorenson, A J Windbank, J N Mandrekar, W R Bamlet, S H Appel, C Armon, P E Barkhaus, P Bosch, K Boylan, W S David, E Feldman, J Glass, L Gutmann, J Katz, W King, C A Luciano, L F McCluskey, S Nash, D S Newman, R M Pascuzzi, E Pioro, L J Sams, S Scelsa, E P Simpson, S H Subramony, E Tiryaki, C A Thornton, E J Sorenson, A J Windbank, J N Mandrekar, W R Bamlet, S H Appel, C Armon, P E Barkhaus, P Bosch, K Boylan, W S David, E Feldman, J Glass, L Gutmann, J Katz, W King, C A Luciano, L F McCluskey, S Nash, D S Newman, R M Pascuzzi, E Pioro, L J Sams, S Scelsa, E P Simpson, S H Subramony, E Tiryaki, C A Thornton
Abstract
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.
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Source: PubMed