Page last updated: January 31, 2023 For web accessibility options: Click/tap the floating blue icon on the right.Trial Information Lysosomal and Rare Disorders Research and Treatment Center, Inc. is conducting a natural history study of patients with Sanfilippo...
Clinical Trial
A Natural History Study of Sanfilippo Syndrome Type D (MPSIIID) | Type IIID | Observational Study | Phoenix Nest
Page last updated: December 13, 2022 For web accessibility options: Click/tap the floating blue icon on the right. Trial Information Phoenix Nest and National Institute of Neurological Disorders and Stroke (NINDS) are conducting a observational research study, which...
Natural History of Biomarkers & Clinical Outcomes | Type IIIA | Observational Study | DenaliÂ
Trial Information Denali Therapeutics is conducting a observational research study, which can also be called a natural history study, into Sanfilippo syndrome Type A (MPS IIIA). The purpose of the research study is to collect blood and behavioral information from...
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...
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...
Open-label Study of Anakinra | Type IIIA, B, C, D | Phase II-III | Lundquist
Trial Information 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....
Modified Autologous Hematopoietic Stem Cells | Type IIIA | Phase I-II | Orchard Therapeutics
Page last updated: March 16, 2023 For web accessibility options: Click/tap the floating blue icon on the right.Trial Information This study is the first in-human clinical trial to explore the safety, tolerability, and clinical efficacy of ex vivo gene...
BMN-250 Enzyme Replacement | Type IIIB | Phase I-II | Biomarin/Allievex
Trial Information 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...
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...