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Treatment of Rett Syndrome With Recombinant Human IGF-1

NCT01777542 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2018-03-26

Study results available
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Summary

Investigators are recruiting children for a clinical trial using the medication recombinant human IGF-1 (a.k.a. mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT). While IGF-1 is approved by the Food \& Drug Administration (FDA) for certain use in children, it is considered an investigational drug in this trial because it has not previously been used to treat RTT. Information from this study will help determine if IGF-1 effectively treats RTT but will not necessarily lead to FDA approval of IGF-1 as a treatment for RTT.

Conditions

Interventions

DRUG

Recombinant Human Insulin Growth Factor 1 (rhIGF-1)

Subjects will receive twice daily subcutaneous injections of IGF-1.

DRUG

Placebo

Subjects will receive twice daily subcutaneous injections of a saline solution (placebo).

Sponsors & Collaborators

Principal Investigators

  • Mustafa Sahin, MD, PhD · Boston Children's Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Max Age
10 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-01-31
Primary Completion
2016-07-31
Completion
2016-11-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases
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 NCT01777542 on ClinicalTrials.gov