Treatment of Rett Syndrome With Recombinant Human IGF-1

Completed · Phase 2 · Has a placebo group

Conditions studied: Rett Syndrome

In brief

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.

Key facts

Study ID

NCT01777542

Run by

Boston Children's Hospital

People needed

30

Starts

2013-01-01

Expected to finish

2016-11-01

Last updated by the study team

2018-03-26

Who can join

Age: 2 and older, up to 10. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of "classic" (or "typical") Rett Syndrome
• Genetic documentation of MECP2 mutation
• Subject must be post-regression (Hagberg Stage 2)
• Subject and caregiver's primary language must be English
• Subject must reside in North America (US and Canada)
• Caregiver must have internet access and be able to complete questionnaires online and communicate via email
• Subject is stable on current medications for at least 4 weeks
• Subject's regimen of non-pharmacological interventions (physical therapy, speech therapy, etc.) is stable for at least 90 days

You may not qualify if…

• Severe scoliosis (curvature >40 degrees)
• Bone-age greater than 11 years
• Cardiomegaly (enlarged heart)
• Tanner stage 2 or higher breast development
• Allergy to IGF-1
• Prior use of IGF-1, growth hormone, or sex steroids

Where it is running

• Boston Children's Hospital — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.