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Project Cure Spinal Muscular Atrophy

Project Cure SMA is a collaborative initiative between Families of SMA and clinical investigators designed to help facilitate the rapid translation of promising new therapies to individuals with SMA. The primary goal of Project Cure SMA is to develop safe and well-tolerated clinical protocols to help identify effective therapies for SMA.




Frequently Asked Questions (FAQ)
  1. Is participation in the Project Cure SMA clinical trials affected by an alliance to any patient group?
    No. Participation is based exclusively on patient criteria.
  2. Can you be in multiple trials?
    Every clinical trial design has its own inclusion/exclusion criteria and you can expect to see, as one of the exclusion criteria items in most trials, that a study subject may not participate in an ongoing clinical trial if they are already taking another study medication. The possibility exists that this may confound results of a clinical therapeutic trial.
  3. What are the side effects and risks of the trial?
    Risks associated with study medication and side effects
    : The study medication your child receives can cause side effects. In other studies, 2%-6% of patients treated with VPA reported side effects including headache, stomach upset, weight gain, tremor, tiredness, dizziness, hair loss, or altered mood. Rare life-threatening and deadly reactions include liver failure and pancreas problems. Reported side effects for carnitine include; nausea, vomiting, abdominal cramps, and diarrhea. Since the study medication is experimental in children with SMA, there may be some side effects that researchers do not yet know about. It is important for you to tell your childís doctor/study staff immediately about any signs or symptoms your child experiences while using the study medication. The following measures have been put in place in order to minimize the risks and/or to alert the Investigators of any possible problems:
    1. Formal study consenting process with parents, patients and Investigator
    2. Rigorous safety assessments during the patientís participation in the study in collaboration with their local pediatrician/attending physician.
    3. A Medical Monitor (an M.D. with a subspecialty in pediatric Neurology) will supervise and review all laboratory data and note any abnormalities. A Data Safety and Monitoring Committee will work together with the Medical Monitor.
    4. Dietary/Supplement record and nutritional analysis by a registered dietician to monitor weight changes and to make appropriate suggestions for intervention. This information will be reviewed with parents and patient during their arrival or on the first day of testing for each visit.
  4. Why are there exclusionary criteria?
    We are definitely interested in getting a therapy to all children with SMA -- and it seems wrong that many children are excluded by these rules. This is a real concern to the investigators, but the rules of research require that there be careful consideration of which patients should be enrolled. The purpose of any study is to show whether or not a therapy works, and the best way to get an accurate study is to focus on those patients in whom changes are most easily measured. Certain features, such as age, or the presence of spinal rods, or the presence of other illnesses, can make it more difficult to measure a change even though it might occur. The problem is that in formal research studies it isn't enough simply to observe that there has been improvement -- it must be measured on a scale that is applied to all of the children in the trial in the same way. Unfortunately, there isn't a single way to measure changes across the full range of severity, age, and complexity that is the whole of SMA. Thus, our best chance to get a good study means that we must concentrate the research efforts on a smaller group where a single good measure is possible. We feel that this is the best way to get the correct answer that will be meaningful to all.

If you have a question, please email questions@projectcuresma.org

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April 22, 2008