Autism Spectrum Disorder (ASD) Clinical Trial
Official title:
Electrophysiological Markers for Interventions in Phelan-McDermid Syndrome and Idiopathic Autism
Verified date | May 2024 |
Source | Icahn School of Medicine at Mount Sinai |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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 | Enrolling by invitation |
Enrollment | 45 |
Est. completion date | March 2025 |
Est. primary completion date | March 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 12 Years |
Eligibility | Inclusion Criteria: - 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 Exclusion Criteria: - Closed epiphyses - Active or suspected neoplasia - Intracranial hypertension - Hepatic insufficiency - Renal insufficiency - Cardiomegaly/valvulopathy - History of allergy to growth hormone or any component of the formulation (mecasermin) - 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) - Visual problems that preclude the use of VEP |
Country | Name | City | State |
---|---|---|---|
United States | Seaver Autism Center for Research & Treatment | New York | New York |
Lead Sponsor | Collaborator |
---|---|
Icahn School of Medicine at Mount Sinai | National Institute of Neurological Disorders and Stroke (NINDS) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Visual Evoked Potentials (VEP) | A noninvasive technique to evaluate the functional integrity of visual pathways in the brain from the retina to the visual cortex via the optic nerve/optic radiations. The VEP is recorded from the head's surface, over the visual cortex, and is extracted from ongoing EEG through signal averaging. VEPs reflect the sum of excitatory and inhibitory postsynaptic potentials occurring on apical dendrites which modulate excitatory and inhibitory signals received by the pyramidal cells. | After 12 weeks of growth hormone therapy | |
Secondary | Aberrant Behavior Checklist (ABC) Social Withdrawal Subscale | A caregiver report symptom checklist. 58-item instrument into 5 subscales: Irritability (score 0-45); Lethargy/Social Withdrawal (score 0-48); Stereotypic Behavior (score 0-21); Hyperactivity (score 0-48); Inappropriate Speech (score 0-12). Total scale 0-174, with higher score indicating more aberrant behavior. | After 12 weeks of growth hormone therapy | |
Secondary | Repetitive Behavior Scale-Revised (RBS-R) | A 43 item instrument with total score from 0-129, with higher score indicating more restricted, repetitive and stereotyped behaviors. | After 12 weeks of growth hormone therapy | |
Secondary | Sensory Profile | The Sensory Profile has 125 items. Parents use a Likert scale to rate how frequently their child demonstrates a particular behavior (ranging from 1 = always to 5 = never). Total scale from 125-625, with a lower score indicates greater deviation from typically developing children and indicates more sensory reactivity symptoms. | After 12 weeks of growth hormone therapy | |
Secondary | Sensory Assessment for Neurodevelopmental Disorders (SAND) | A clinician-administered assessment and corresponding caregiver interview that is not dependent on verbal or cognitive ability and is therefore appropriate for severely affected or nonverbal individuals with PMS. Responses are rated by a trained examiner on an algorithm. Scores are dichotomous, 0 (not present) or 1 (present) and are based on a summary of observed sensory behaviors throughout the duration of the observation. A total SAND score ranging from 0 to 90. Higher scores represent a higher level of sensory reactivity symptoms. | After 12 weeks of growth hormone therapy |
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