Clinical trials for Mucopolysaccharidosis type 1
9 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- RECRUITINGPHASE1, PHASE2NCT06488924An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIBJCR Pharmaceuticals Co., Ltd.
- RECRUITINGPhase 1NCT06519552A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type IWest China Hospital · PI: Xingchen Peng, Ph.D
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT06181136Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)Denali Therapeutics Inc. · PI: Ana-Claire Meyer, MD
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT06095388Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIAJCR Pharmaceuticals Co., Ltd.
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT05665166Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIUniversity of Manchester · PI: Robert Wynn
- ACTIVE NOT RECRUITINGNCT05634512Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.Masonic Cancer Center, University of Minnesota · PI: Paul Orchard
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT04201405Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIAUniversity of Manchester · PI: Brian Bigger
- ACTIVE NOT RECRUITINGPHASE2, PHASE3NCT03566043CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)REGENXBIO Inc.
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT03488394Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler VariantOrchard Therapeutics