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.

Treatment Trials

	IntraBio	Lysogene	Passage Bio	Sanofi Genzyme	Sio Gene Therapies	NHGRI/Sio Gene Therapies	Queens University/Taysha Gene Therapies
Disease	Late Onset GM2	GM1 Gangliosidosis Infantile and late infantile	GM1 Gangliosidosis Late infantile followed by infantile	Late Onset GM2	GM2 Sandhoff/Tay-Sachs Infantile/Juvenile	GM1 Gangliosidosis Infantile	GM2 Sandhoff/Tay-Sachs Infantile
Program	Small molecule	Gene Therapy	Gene Therapy	Small molecule/substrate inhibitor	Gene Therapy	Gene Therapy	Gene Therapy
Status	Recruiting	Recruiting	Recruiting in U.S.	Recruiting	Recruiting	Recruiting	Recruiting in Canada
Clinical Trials posting	CT Posting	CT Posting	CT Posting	CT Posting	CT Posting	CT posting	CT posting Queens site
Brochure					Download pdf here (1.10 MB)		Download pdf here (428 KB)

 

Natural History Studies

- coming soon -

Other Resources

FAQ About Clinical Trials at clinicaltrials.gov
Clinical Trials at clinicaltrials.gov
Food and Drug Administration at fda.gov