Deletion Syndrome > Insulin-Like Growth Factor-1 (IGF-1)

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

Recruiting in Palo Alto (17 mi)

Overseen By Alexander 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

Group I: Insulin-Like Growth Factor-1 (IGF-1) Experimental Treatment 1 Intervention

Injection

Group II: Normal saline Placebo Group 1 Intervention

Injection

Find A Clinic Near You

Research locations nearby Select from list below to view details:

Seaver Austin Center, Icahn School of Medicine at Mount Sinai New York, NY

Who is running the clinical trial?

Icahn School of Medicine at Mount Sinai Lead Sponsor

National Institute of Mental Health (NIMH) Collaborator

References