P.O. Box 50030 London, ON N6A 6H8 info@rett.ca (519) 474-6877

News Release – Boston IGF – 1 Trial

Boston Children’s Hospital has begun the Phase 2 clinical trial entitled “Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation with IGF-1.” They are currently recruiting girls with Rett syndrome for this study.


This phase of the trial will include 30 children with Rett syndrome who are female, between the ages of 2 – 10 years old.  They must have a clinical diagnosis of Classic Rett syndrome, a documented genetic MECP2 mutation, and they must be in a “stable” stage of the disease.


You may apply if you live in Canada and your primary language is English.  You must have access to the internet to correspond online.  Several forms must be completed for the initial application.  If chosen for the in-person screening, families will be required to travel to Boston for a screening visit.  If chosen for the trial, families must commit to a total of 7 trips to Boston.  Travel and lodging costs will not be covered.  It is the family’s responsibility to pay for travel and lodging if enrolled in this trial.  The time commitment for the trial is at least one year.  Individuals should not be in more than one clinical drug trial at the same time.  The child must also be stable on current medications.


The Boston clinical trial will be one of several opportunities open to Canadian families in the coming years. Different trials will have different eligibility criteria.  The Canadian Rett Syndrome Registry will be implemented in 2014, allowing for clinical trials to begin in Canada.  The Ontario Rett Syndrome Association (O.R.S.A.) is the founder of this registry and has been working tirelessly to ensure this registry is implemented properly.  Several clinicians and researchers are attracted to the clinical trials and research opportunities soon to be available in Canada.


For more information about the Rett Syndrome Research Program at Boston Children’s Hospital, please email rettresearch@childrens.harvard.edu.


For more information about clinical trials in Canada or the Canadian Rett Syndrome Registry, please call Terry Boyd, O.R.S.A. President at 519-474-6877 or email terry@rett.ca.