Current Clinical Trials

Clinical trials are where the rubber meets the road, scientifically.

It’s the crucial step of determining whether earlier research (basic and translational) is able to show the same therapeutic benefits in people.To stay current on newly emerging clinical trial listings, you can periodically search for Sanfilippo or MPS III on these sites:

The following are clinical trials currently underway or preparation stages.

If you would like more information about any of these, or about other earlier-stage research underway, contact us at

Clinical Trials: Gene Therapy

EGT-101 Gene Therapy | Type IIIA | Phase I-II | Esteve

Update: April 2021 This Phase 1/2 trial has now fully enrolled and is not accepting additional patients. We are hopeful that a Phase 3 trial will be initiated in the future but no timeline is currently available. Trial Information EGT-101 is Esteve's potential...

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Clincal Trials: Enzyme Replacement

SOB-103 Enzyme Replacement | Type IIIA | Phase I-II | Sobi

Update: April 27, 2021 Last year Sobi decided to halt development of this ERT program. The patients enrolled in the trial were provided access to drug for the full 2 years of the planned trial period. All patients have now completed the trial and transitioned off of...

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Clincal Trials: Stem Cell Therapy

Clincal Trials: Substrate Reduction Therapy

There are not any clinical trials exploring substrate reduction currently happening.

Research Funded by Cure Sanfilippo Foundation

Cure Sanfilippo Foundation architects and funds promising research to accelerate discovery of a treatment or cure for Sanfilippo.

Explore the research funded by the Foundation.