~1 spots leftby Mar 2026

Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)

Recruiting in Palo Alto (17 mi)
Overseen ByAlexander Kolevzon, MD
Age: < 18
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Waitlist Available
Sponsor: Icahn School of Medicine at Mount Sinai
Prior Safety Data

Trial Summary

What is the purpose of this trial?The purpose of this study is to pilot the use of Insulin-Like Growth Factor-1 (IGF-1) treatment in 22q13 Deletion Syndrome (Phelan-McDermid Syndrome) caused by SHANK3 gene deficiency in order to evaluate safety, tolerability, and efficacy. IGF-1 is an injection under the skin that contains human IGF-1. IGF-1 is approved by the FDA under the brand name Increlex for the treatment of children with short stature due to primary IGF-1 deficiency. It is being used off-label in the current study and is not FDA approved, nor has it yet been studied in humans for the treatment of SHANK3 deficiency.

Eligibility Criteria

Participant Groups

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Insulin-Like Growth Factor-1 (IGF-1)Experimental Treatment1 Intervention
Injection
Group II: Normal salinePlacebo Group1 Intervention
Injection

Find A Clinic Near You

Research locations nearbySelect from list below to view details:
Seaver Austin Center, Icahn School of Medicine at Mount SinaiNew York, NY
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Who is running the clinical trial?

Icahn School of Medicine at Mount SinaiLead Sponsor
National Institute of Mental Health (NIMH)Collaborator

References