Children's Hospital, Boston, to Begin Clinical Trials
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 participate in the study your child must be female, between the ages of 2 to 12 and have a genetic diagnosis (MECP2 deletion or mutation) of Rett Syndrome. As you may know, there is no treatment for this illness. Currently, the standard management of Rett syndrome is supportive, which means attempting to prevent complications and treatment of symptoms.
This study involves testing an investigational drug, which means that even though IGF-1 is approved by the Food and Drug Administration (FDA) for use in children, it has not been used before to treat Rett syndrome specifically. Information from this research will help determine whether the drug should be approved by the FDA in the future for the treatment of Rett Syndrome.
Treatment of Rett Syndrome with IGF-1 - CURRENTLY ENROLLING
For more information about this clinical trial, please contact:
Email: rettresearch@childrens.harvard.edu
Phone: 617-355-5230
For more information, please visit this site.
Children's Hospital, Boston, Begins New Rett Syndrome Program
The Rett Syndrome Program at Children's Hospital Boston provides specialized care for children and adults with Rett syndrome and related conditions. The program is staffed by a team of specialists from various fields to ensure that each individual receives the best care. Specialties include:
Neurology
Developmental pediatrics
Gastroenterology
Pulmonology cardiology
Endocrinology
Genetics
Psychiatry
- Orthopedics
Clinical Trials
If you would like to learn more about clinical trials for Rett syndrome, please call Kate Barnes at 617-355-5230. Read about the Rett Syndrome Program.