STUDY: Effect of myo-Inositol on Cerebellar and Immune
Function in Patients with Ataxia-Telangiectasia
STUDY LOCATION: The Children's Hospital of Philadelphia (CHOP)
34th Street & Civic Center Boulevard
Philadelphia, Pennsylvania USA 19104-4399
PRINCIPLE INVESTIGATOR: Gerard Berry, M.D. (Metabolism/Biochemical Genetics)
CO-INVESTIGATORS: Peter Bingham, M.D. (Neurology)
Kathleen Sullivan, M.D., Ph.D. (Immunology)
FOCUS OF STUDY: Investigation of pharmacological doses of a
nutrient, identified as deficient in patients with
A-T, with critical roles in cell signaling and
cellular metabolism. Pre and post-assessment of
immune, neurological and activities of daily living
STUDY DESIGN: Double blind placebo-controlled randomized-crossover
FUNDING SOURCE: NIH Clinical Research Center Funds
WHAT IS REQUIRED: Four(4) trips to Philadelphia:
- 5 days at CHOP for "pre" measurements &
establishment of therapeutic dose of the
intervention per your child's weight and metabolism.
- Return home with liquid intervention.
- Take either placebo or nutrient for 1 month (both
- 2 days at CHOP 1 month later for "post"
- at least 1 week in-between, then repeat steps 1-4.
COST: - No cost for study and assessments.
- Minimal out-of-pocket for meals, recreation.
- Lodging at Ronald McDonald House.
- Assistance with airfares provided by The A-T
Project (Austin) with support from The A-T Medical
Research Foundation (Los Angeles).
BENEFITS OF STUDY: To participants & families:
- Complete immune & neurological work-ups including
- Determination of your child's A-T genotype
- Upon study completion, details of your child's
neurological and immune response to the intervention.
To all affected by A-T:
- Contributing to treatment options for A-T.
- Correlation of phenotype(symptoms & seriousness)
- Determination of levels of critical cell signaling
phospholipids in specific cell membranes(never before
CONTACT: Gerard Berry, M.D. 215-590-3372
Michelle Bergman, R.N. 215-590-1399 - Clinical
Research Center Coordinatior (Ms. Bergman can
provide referrals to other families who have
Pam Bower <pbower at inforamp.net>
InterNAF Board Member
Halifax, Nova Scotia Canada
INTERnational Network of Ataxia Friends
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