Treatment of Rett Syndrome With Recombinant Human IGF-1
Status:
Completed
Trial end date:
2016-11-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Boston Children's Hospital Boston Children’s Hospital