- Last Updated: Friday, 23 May 2014 20:43
There are many definitions of clinical trials but generally they are studies done in people to treat or better understand disease. The decision to participate in clinical trials is a personal and private decision. NTSAD supports families in all their care choices. Our role is to provide families with the tools and resources to make informed decisions that they can live with and that are right for their family.
Click here to read more about Evaluating Experimental Therapies - US and Abroad.
Current Clinical Trials
Natural History for Tay-Sachs, Sandhoff and GM-1
Bone Marrow Transplant
Cholesteryl Ester Storage Disease
Synageva BioPharma Corp. has initiated a clinical study to evaluate the safety, tolerability, and pharmacokinetics of SBC-102 as an enzyme replacement therapy for cholesteryl ester storage disease (CESD). Visit clinicaltrials.gov to learn more.
Past Clinical Trials
Pyrimethamine for Late Onset Tay-Sachs
A small investigator-sponsored clinical trial of the molecular chaperone Pyrimethamine was done in adults with Late Onset Tay-Sachs. A protocol has been developed for the next phase of the trial to address proper dosage. Efforts have stalled due to regulatory requirements and funding challenges.
Zavesca for all forms of Tay-Sachs, Sandhoff and GM-1
Clinical trials in babies, children and adults with Tay-Sachs and Sandhoff of the substrate inhibitor ZavescaÂ® (miglustat) did not show therapeutic benefit. Zavesca is FDA approved for Gaucher, which provides families affected by other lysosomal diseases the option of using the drug for off-label use.