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

NCT05187377 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2025-04-20

No results posted yet for this study

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.

Conditions

  • Phelan-McDermid Syndrome
  • Autism 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.

Sponsors & Collaborators

Principal Investigators

  • Alexander Kolevzon, MD · Icahn School of Medicine at Mount Sinai

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-01-19
Primary Completion
2025-01-28
Completion
2025-02-05
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05187377 on ClinicalTrials.gov