Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Clinical Trials (Current)

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/Juvenile

GM2 Sandhoff/Tay-Sachs
Infantile

Program

Small molecule

Gene Therapy

Gene Therapy

Small molecule/substrate inhibitor

Gene Therapy

Gene Therapy

Gene Therapy

Status

Recruiting

Not yet 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

Not yet posted

 

 

 

 

 

 

 

Other Resources

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

An Understanding

Courageous Parents Network has a guided pathway about clinical trials with helpful videos addressing what to expect, how to navigate and process the news whether a trial is a fit for your family or not. 

If you have questions that are not answered here, please reach out to NTSAD's Family Services team here.

Balancing Hope and Expectation

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