The Center for Information and Study on Clinical Research Participation (CISCRP) defines a clinical trial as, "a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people and ways to improve health. Interventional trials determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments. Observational trials address health issues in large groups of people or populations in natural settings."
The decision to participate in a clinical trial 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
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IntraBio |
Passage Bio |
Sanofi Genzyme |
Sio Gene Therapies I |
Sio Gene Therapies II |
Taysha Gene Therapies |
Primary focus |
Late Onset GM2 |
GM1 Gangliosidosis |
Late Onset GM2 |
GM2 Sandhoff/Tay-Sachs |
GM1 Gangliosidosis |
GM2 Sandhoff/Tay-Sachs |
Program |
Small molecule |
Gene Therapy |
Small molecule/substrate inhibitor |
Gene Therapy |
Gene Therapy |
Gene Therapy |
Status |
Recruiting |
NOT yet recruiting |
Recruiting |
Recruiting |
Recruiting |
NOT yet recruiting |
Clinical Trials posting |
Not yet posted |
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Other Resources
FAQ About Clinical Trials at clinicaltrials.gov
Clinical Trials at clinicaltrials.gov
Food and Drug Administration at fda.gov