Status:
COMPLETED
Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
Lead Sponsor:
Boston Children's Hospital
Collaborating Sponsors:
International Rett Syndrome Foundation
Autism Speaks
Conditions:
Rett Syndrome
Eligibility:
FEMALE
2-12 years
Phase:
PHASE1
Brief Summary
The investigators are recruiting children for a research study using a medication known as IGF-1 (mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT). To part...
Detailed Description
There are two treatment periods in the trial. The multiple ascending dose (MAD) period is an intensive 4-week pharmacokinetic study which will require 3 inpatient stays and 4 half-day outpatient visit...
Eligibility Criteria
Inclusion
- female
- with RTT (typical or variant) as defined using the internationally agreed 2010 RettSearch criteria.
- genetically defined mutation or deletion of the MECP2 gene.
- Girls will have the following prepubertal status: (1) Tanner stage 1 or 2 breast development; (2) Tanner stage 1 or 2 pubic hair development; (3) and younger than 12 years by bone age.
- Chronological age must be 2 years or older
Exclusion
- prior therapeutic use of IGF-1, growth hormone, Lupron® or sex steroids
- allergy to the trial product
- co-morbid or chronic illness beyond that known to be associated with Rett Syndrome: diabetes mellitus, fatty acid oxidation disorder, chromosomal aneuploidy, syndromes associated with high risk of malignancy, current or previous exposure to spinal irradiation or history of malignancy.
- severe scoliosis (defined as a spinal curve of 70 degrees or more as measured on clinical and radiological examination)
Key Trial Info
Start Date :
December 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2013
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT01253317
Start Date
December 1 2010
End Date
January 1 2013
Last Update
June 22 2017
Active Locations (1)
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1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115