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 Contact@CureSanfilippoFoundation.org.
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...
ABO-101 Gene Therapy Transfer | 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...
ABO-102 Gene Therapy Transfer | Type IIIA | Phase I-II | Abeona
Page last updated Feb. 19, 2021 Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) will be delivered one time through a venous catheter inserted into a patient's peripheral limb...
LYS-SAF302 Gene Therapy | Type IIIA | Phase II-III | Lysogene
Page Last Updated Feb. 15, 2021 LYS-SAF302 is a gene therapy by Lysogene which is intended to deliver a working copy of the SGSH gene to deliver the gene therapy to the patient's brain. The normal gene is placed inside the shell of a non-disease-causing virus...
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...
BMN-250 Enzyme Replacement | Type IIIB | Phase I-II | Biomarin/Allievex
The study's primary objectives are to evaluate the safety and tolerability of AX 250 administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the impact of AX 250 on cognitive function in patients with MPS IIIB as assessed by the...
Clincal Trials: Stem Cell Therapy
Modified Autologous Hematopoietic Stem Cells | Type IIIA | Phase I-II | Orchard Therapeutics
Page last updated Feb. 19, 2021 This study is the first in-human clinical trial to explore the safety, tolerability, and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene) in MPSIIIA...
Clincal Trials: Substrate Reduction Therapy
Open-label Study of Anakinra | Type IIIA, B, C, D | Phase II-III | Lundquist
Inflammation has been connected with disease pathogenesis in the MPS disorders. Therapies aimed at decreasing inflammation are currently being studied in many MPS disorders and benefits in both brain and other parts of the body have been reported.Decreasing...