drugset / Trial / NCT05187377
A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism
Phase 2
Completed
32 enrolled
Icahn School of Medicine at Mount Sinai
National Institute of Neurological Disorders and Stroke (NINDS) · collab
RandomizedCrossoverQuadruple-blindTreatment
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.
Timeline
- Start
- 2022-01-19
- Primary completion
- 2025-01-28
- Completion
- 2025-02-05
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Somatropin | Protein / enzyme biologic | 0.3 mg/kg | Subcutaneous |