A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism

PHASE2CompletedINTERVENTIONAL
Enrollment

32

Participants

Timeline

Start Date

January 19, 2022

Primary Completion Date

January 28, 2025

Study Completion Date

February 5, 2025

Conditions
Phelan-McDermid SyndromeAutism Spectrum Disorder (ASD)
Interventions
DRUG

Growth Hormone

Growth hormone will be administered subcutaneously once daily. A starting dose of 0.15 mg/kg/week divided daily for 2 weeks to ensure safety and tolerance. The dose will then be increased to 0.3 mg/kg/week for 10 weeks. Doses will be titrated based on IGF-1 levels and monitored every four weeks up to a maximum dose of 0.45 mg/kg/week based on the package insert.

DRUG

Saline

Placebo (saline) will be administered subcutaneously once daily.

Trial Locations (1)

10029

Seaver Autism Center for Research & Treatment, New York

All Listed Sponsors
collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

lead

Icahn School of Medicine at Mount Sinai

OTHER