A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism
Status:
Recruiting
Trial end date:
2024-07-01
Target enrollment:
Participant gender:
Summary
This clinical trial will use growth hormone as a novel treatment for Phelan-McDermid syndrome
(PMS) and idiopathic autism. A double-blind, placebo-controlled crossover trial design will
be used in 30 children with idiopathic autism and 15 children with PMS to evaluate the the
effects of growth hormone on visual evoked potentials (VEPs), socialization, language, and
repetitive behaviors. The researchers expect to provide evidence for the feasibility of using
VEPs in PMS, and to show support for growth hormone in ameliorating clinical symptoms of ASD.
Phase:
Phase 2
Details
Lead Sponsor:
Icahn School of Medicine at Mount Sinai
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)