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Current Clinical Trials & Studies for Sanfilippo

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

Clinical trials for Sanfilippo are the crucial step of determining whether earlier research (basic and translational) shows the same therapeutic benefits in people.

Participation in observational studies and research surveys are opportunities to contribute to scientists’ understand of symptoms of Sanfilippo and how things change over time for the children. Participation also helps researchers develop new and improved outcome measures and more.

We do our best to keep the following list of clinical trials and studies for Sanfilippo up to date. However, we recommend periodically searching for “Sanfilippo” or “MPS III” on ClinicalTrials.gov and ClinicalTrialsRegister.eu in order for you to have the most-current information and to stay on top of newly-emerging clinical trial listings.

Contact us at Contact@CureSanfilippoFoundation.org if you would like more information about any of these clinical trial or other earlier-stage research underway.

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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ABO-101 Gene Therapy | Type IIIB | Phase I-II | Abeona

Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one time through a venous catheter inserted into a patient's peripheral limb vein. A tapering course of prophylactic...

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ABO-102 Gene Therapy | Type IIIA | Phase I-II | Abeona

Page last updated May 18, 2022 The ABO-102 Gene Therapy uses a self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) delivered one time through a venous catheter inserted into a...

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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.

All Research on Sanfilippo

Beyond clinical trials, there is basic and translational research (earlier-stage research) exploring multiple therapies about how to address the systemic impact and symptoms of Sanfilippo in order to improve the quality of life for a child with Sanfilippo. Learn more about the entire ecosystem of Sanfilippo reseach, as well as current scientific news.

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.

Learn more about the Foundation

We are the nation’s largest 501(c)3 nonprofit dedicated to advocating and funding research to bring about treatments and/or a cure for Sanfilippo Syndrome. Cure Sanfilippo has funded research grants around the world which has helped clinical trials get underway, treating children with Sanfilippo Syndrome. The Foundation creates and explores innovative and breakthrough research to ensure multiple promising paths are being explored in the effort to save children and better their quality of life.

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Dr. Cara O’Neill, Chief Science Officer for the Foundation, meeting with researchers conducting important work regarding Sanfilippo in their lab to talk about progress and ideas.

Team working on Sanfilippo Syndrome caregiver preference study

Parents of children with Sanfilippo meeting to contribute to the Foundation’s work to create materials to help scientists and regulators better include the patient and caregiver perspective in research.