CLINICAL STUDY: Ataxia-Telangiectasia

Pam Bower pbower at inforamp.net
Tue Feb 24 20:11:16 EST 1998


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 
                        functioning.

STUDY DESIGN:           Double blind placebo-controlled randomized-crossover
                        study.

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
                        pleasant tasting).
  
                        - 2 days at CHOP 1 month later for "post" 
                        measurements.

                        - 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
brain MRI.
                        - 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)
                        with genotype.
                        - Determination of levels of critical cell signaling
                        phospholipids in specific cell membranes(never before 
                        measured).

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
                        participated).

              
                


~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~
Pam Bower <pbower at inforamp.net>
InterNAF Board Member
Halifax, Nova Scotia Canada
~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~
INTERnational Network of Ataxia Friends
http://internaf.merseyside.org/ 
To subscribe to InterNAF 
send an email to:  Majordomo at connect.org.uk
with a one line message:  Subscribe InterNAF
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