NCT05187377CompletedPhase 2
A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism
Electrophysiological Markers for Interventions in Phelan-McDermid Syndrome and Idiopathic Autism
Source: ClinicalTrials.gov · Last updated 20 Apr 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 32 (actual)
- First submitted
- 28 Dec 2021
- Start date
- 19 Jan 2022
- Primary completion date
- 28 Jan 2025
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Diseases
- Autism
- NCT number
- NCT05187377
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 32
- From 19 Jan 2022 to the primary completion date, 28 Jan 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patients with comorbid conditions who are deemed too medically compromised to tolerate the risk of experimental treatment with growth hormone (including severe obesity, sleep apnea, and various acute health conditions)
Other illness
- Intracranial hypertension
- Hepatic insufficiency
- Renal insufficiency
- History of allergy to growth hormone or any component of the formulation (mecasermin)
Other
- Closed epiphyses
- Active or suspected neoplasia
- Cardiomegaly/valvulopathy
- Visual problems that preclude the use of VEP
Who the study is looking for
- Children between 2 and 12 years of age
- Open epiphyses on bone age x ray
- Must be on stable medication and psychosocial treatment regimens for at least three months prior to enrollment, assuming the concomitant medication is safe for use with Growth Hormone
- No prior use of Growth Hormone or IGF-1
- ASD group: Meet DSM-5 criteria for Autism Spectrum Disorder confirmed by the Autism Diagnostic Interview-Revised (ADI-R) and the Autism Diagnostic Observation Schedule, Second Edition (ADOS-2); absence of known pathogenic copy number variants
- PMS group: Known pathogenic deletions or mutations in SHANK3 gene diagnosed by array CGH and/or direct sequencing
Age in the registry: 2 years to 12 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Seaver Autism Center for Research & Treatment Coordinator of this site? Confirm its status | New York | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.