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The A-T Children's Project raises funds to support and
coordinate biomedical research projects, scientific conferences and a clinical
center aimed at finding a cure for ataxia-telangiectasia, a rare genetic disease
that causes progressive loss of motor control, cancer and
immune system problems.
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Attention researchers -
for grant proposal guidelines click here. |
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2005 WALT DISNEY WORLD® Marathon
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Join the Marathon A-TEAM if you are registered to run
another marathon.
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Donate
to help find a cure for A-T and support the Marathon
A-TEAM's efforts. |
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Clinical Trial Opens at Hopkins |
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Who will participate in the trial?
20 confirmed A-T patients who meet the following criteria:·
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Age 12 and older (during the
initial phase to ensure safety with plans to include younger patients in
the near future) |
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Able to travel to Baltimore,
Maryland monthly |
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Able to swallow medicine
capsules |
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No nutritional supplements
other than standard daily multivitamin taken within the previous 30 days |
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No need for insulin or oral
hypoglycemic agent |
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No previous adverse reaction
to alpha lipoic acid or
nicotinamide |
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Preference will be given to
subjects who previously have been evaluated at the Johns Hopkins A-T
Clinical Center |
How long is the study?
8 ½ months
What drugs will be studied?
An antioxidant (alpha lipoic acid) and a PARP-1 inhibitor
(nicotinamide)
When will it start?
This clinical trial will start immediately.
Where will it be held?
A-T Clinical Center at Johns Hopkins Hospital in Baltimore, Maryland
Why is this clinical trial
being held?
To determine the safety and preliminary effectiveness of a combination of
drugs to slow the brain cell death that occurs in patients with A-T
Who is funding this study?
This study is being funded by the A-T Children’s Project. Additional
funding is currently being sought from the NIH-funded General Clinical
Research Center at Johns Hopkins.
When will the study include
more patients, and younger patients?
If a beneficial effect is found at the end of the study, a second trial
will be planned to include a larger number of patients of a wider age
range. Patients would be recruited nationally and travel reimbursement
would be included in the budget.
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 Whom can I contact with questions?
A-T Clinical Center
The Johns Hopkins Hospital
Room CMSC 1102
600 N. Wolfe St.
Baltimore, MD 21287
Howard Lederman, MD PhD, Director
Tel. 410-955-5883
E-mail:
hlederm1@jhem.jhmi.edu
Karen Rosquist, RN, Coordinator
Tel. 800-610-5691
E-mail: krosquis@jhmi.edu
Special Note:
The safety and efficacy of these
drugs for patients with A-T are unknown. While these drugs are readily
available, the doses used for the clinical trial will not be made public
and these drugs are not recommended at this time for use by patients with
A-T outside of this clinical trial.
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