Clinical Trial

JR-441 Enzyme Replacement | Type IIIA | Phase I-II | JCR

Page last updated: October 23, 2023 For web accessibility options: Click/tap the floating blue icon on the right. Trial Information Phase I/​II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA A Phase I/ II, open-label, randomized, 2-arm study,...

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...