Trials / Conditions / Sanfilippo Syndrome

Sanfilippo Syndrome

10 registered clinical trials studyying Sanfilippo Syndrome — 4 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	Study of Cannabidiol in Sanfilippo Syndrome NCT06333041	Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center	Phase 2 / Phase 3
Recruiting	An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High D NCT06614894	Ozlem Goker-Alpan	Phase 2 / Phase 3
Unknown	The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3) NCT05705674	Lysosomal and Rare Disorders Research and Treatment Center, Inc.	—
Enrolling By Invitation	Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA NCT04360265	Ultragenyx Pharmaceutical Inc	Phase 3
Terminated	Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease NCT04088734	Ultragenyx Pharmaceutical Inc	Phase 1 / Phase 2
Recruiting	Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH NCT02716246	Ultragenyx Pharmaceutical Inc	Phase 2 / Phase 3
Terminated	An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Part NCT02350816	Shire	Phase 2
Completed	Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD NCT02060526	Shire	Phase 2
Terminated	Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A NCT01299727	Shire	Phase 1 / Phase 2
Completed	HSCT for High Risk Inherited Inborn Errors NCT00383448	Masonic Cancer Center, University of Minnesota	Phase 2