Monday, April 4, 2011

FAQ about the IGF-1 Rett Syndrome Trial

"Treatment of Rett Syndrome with IGF-1"

PI: Omar Khwaja MD, PhD

Frequently Asked Questions (FAQ)
Download a PDF version of these FAQ
Q: Who is eligible for this clinical trial?
A: Eligible participants will be girls between the ages of 2 and 12 years, have a positive MECP2 genetic test and reside in the United States for the duration of the study.

Q: My child has a mutation in CDKL5, can we still participate?
A: No; all the work that has contributed to the development of this study has been conducted in mice with mutations in MECP2. Therefore, we do not know what the effects will be on children with CDKL5 mutations and at this time we are not enrolling children with this condition.

Q: What is the difference between Phase 1 and Phase 2?
A: Phase 1: A total of 10 girls will be enrolled in Phase 1. The objective of Phase 1 is to determine the tolerability of IGF-1 in the Rett Syndrome population. The visit schedule for Phase 1 includes three 24-hour inpatient stays and three outpatient visits (lasting approximately 3 hours each). At the end of the fourth week, participants will come to the hospital for a day-long visit lasting approximately 8 hours. Subjects that choose to participate in Phase 1 will automatically be enrolled in Phase 2, where they will receive an additional 20 weeks of treatment with IGF-1.

Phase 2: Phase 2 is a placebo controlled study lasting a total of 50 weeks. 30 girls will be enrolled in this phase. Participants will receive treatment (either IGF-1 or placebo) for 20-weeks, followed by 20 weeks of the alternate treatment (for example, if a subject received placebo for the first 20 weeks, she will be treated with IGF-1 for the following 20 weeks). There is a 6-week break between the two 20-week increments, during which participants will receive no treatment. In Phase 2, parents are required to bring their child to Children's Hospital, Boston every 4 weeks throughout the duration of the study.

Q: How will children be selected for participation?
A: The complete "inclusion criteria" is listed in the consent forms which have been sent to families interested in enrolling their children in this study (Note: if you have not received these documents, please e-mail and we will forward these to you). If the number of children that meet criteria exceeds 40; the potential participants will be randomized and chosen by a lottery. We will notify parents if their child has been selected to confirm that they still wish to enroll.

Q: When will the children be selected?
A: Children will be enrolled steadily over the first year of the study.

Phase 1: Enrollment for this phase is currently closed. For those who have sent in applications for Phase 1, we will schedule screening visits in March 2011 and begin treating participants in April.

Phase 2: Enrollment for this phase is currently open. We anticipate starting screening for this phase in August 2011.

Q: How do I apply for participation in this study?
A: Please send an email to and we will send you the necessary information and forms for enrollment.

Q: Once selections are made, will a more defined schedule of visits be provided?
A: Yes; at enrollment you will select a schedule schema. Once the first visit has occurred, this schedule is "set in stone" and cannot be altered under any circumstances.

Q: How soon after the initial screening/visits will the trial begin?
A: This will depend on you and your family's schedule. You may select when you would like the visits to begin.

Q: Can people outside of the Northeast enroll in Phase 1?
A: While Phase 1 is open to anyone, those who choose to enroll will most likely reside in the New England area as participants are required to visit the hospital twice a week for safety monitoring.

Q: If we choose not to participate in Phase 1, will this reduce our daughter's chance of being chosen for Phase 2?
A: No; participation in Phase 1 will in no way affect her eligibility for Phase 2.

Q: If I do not get selected for Phase 1, do I have to apply again for Phase 2?
A: No, the girls that are not selected for Phase 1 will automatically roll-over for enrollment in Phase 2.

Q: Can I go to my local Rett syndrome clinic for the evaluations?
A: No; the only site approved for clinical evaluations is Children's Hospital Boston.

Q: I tried to sign the consent forms, but they say "Not Valid"; now what?
A: These consent forms are intended for your review only. The official consent form will be signed in-person after your daughter has been selected and you have met with the research team to confirm her eligibility.

Q: Is there a hotel close to the hospital?
A: Yes, the closest hotel, the Inn at Longwood Medical, is next door to the hospital.

Q: Are there any discounted rates on airfare or hotel stay if you are traveling with a special needs child that is receiving treatment in a hospital setting such as this trial?
A: For airfare, check out Miracle Flights for Kids or Angel Flight. For families that cannot afford a hotel, there are a handful of rooms resourced within the hospital. However, these rooms are only available on a first-come, first-served basis and cannot be reserved well in advance. Unfortunately, we are not able to compensate families for lodging or travel.
For further information about this study, please contact:
Phone: 617-355-5230
Fax: 617-730-4669

Source: Children's Hospital Boston

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