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

NCT01525901 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2022-05-12

Study results available
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Summary

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.

Conditions

  • 22q13 Deletion Syndrome
  • Phelan-McDermid Syndrome

Interventions

DRUG

Insulin-Like Growth Factor-1 (IGF-1)

IGF-1 and placebo will each be administered for 3 months with a four-week washout period in between. IGF-1 will be administered for 3 months subcutaneously.

DRUG

Normal saline

Saline solution will be administered for three months subcutaneously.

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
5 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-02-29
Primary Completion
2016-08-23
Completion
2016-08-23

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 NCT01525901 on ClinicalTrials.gov