- Is participation in the Project Cure SMA clinical trials affected by
an alliance to any patient group?
No. Participation is based exclusively on patient
- 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
- 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
- Formal study consenting process with parents, patients and
- Rigorous safety assessments during the patientís participation in
the study in collaboration with their local pediatrician/attending
- 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.
- 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
- 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
To receive SMA research news and updates, please sign up
Families of SMA Elist.