Clinical Trials
Live snapshot from ClinicalTrials.gov, Sanfilippo Syndrome clinical trials (Recruiting / Not yet recruiting / Active, not recruiting).
14 trials
- NCT02716246 RECRUITINGPhase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIAPhase: PHASE2, PHASE3 Updated:Locations: Nationwide Children's Hospital, Columbus, United States; Children's Hospital of Pittsburgh, Pittsburgh, United States; Women's and Children's Hospital, North Adelaide, Australia
- NCT06181136 ACTIVE_NOT_RECRUITINGA Phase 1/2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)Phase: PHASE1, PHASE2 Updated:Locations: UCSF Benioff Children's Hospital Oakland, Oakland, United States; University of Iowa Stead Family Children's Hospital, Iowa City, United States; University of North Carolina at Chapel Hill, Chapel Hill, United States
- NCT02254863 RECRUITINGAugmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like CellsPhase: PHASE1 Updated:Locations: Duke University Medical Center, Durham, United States
- NCT06488924 RECRUITINGAn Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIBPhase: PHASE1, PHASE2 Updated:Locations: Hiroshima University Hospital, Hiroshima, Japan; University of the Ryukyus Hospital, Okinawa, Japan; National Center for Child Health and Development, Tokyo, Japan
- NCT06567769 RECRUITINGA Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)Phase: PHASE1 Updated:Locations: UCSF Benioff Children's Hospital, Oakland, United States; University of Minnesota, Minneapolis, United States; National Center for Child Health and Development, Setagaya City, Japan
- NCT06095388 ACTIVE_NOT_RECRUITINGPhase I/II Study of Weekly Infusions of JR-441 in Patients With Mucopolysaccharidosis Type IIIAPhase: PHASE1, PHASE2 Updated:Locations: Universitätsklinikum Hamburg-Eppendorf, Hamburg, Germany
- NCT06614894 RECRUITINGAn Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS IIIPhase: PHASE2, PHASE3 Updated:Locations: Lysosomal & Rare Disorders Research & Treatment Center, Inc., Fairfax, United States
- NCT05825131 RECRUITINGA Combination Retrospective and Prospective Natural History Study of Participants With Sanfilippo Syndrome Mucopolysaccharidosis Type IIIC (MPS IIIC)Updated:Locations: The University of Texas Southwestern Medical Center, Dallas, United States; Hospices Civils De Lyon, Bron, France
- NCT04201405 ACTIVE_NOT_RECRUITINGA Phase I-II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced Ex Vivo with CD11b Lentiviral Vector Encoding for Human SGSH in Patients with Mucopolysaccharidosis Type IIIA (MPS IIIa, Sanfilippo Syndrome Type A)Phase: PHASE1, PHASE2 Updated:Locations: Manchester University NHS Foundation Trust, Manchester, United Kingdom
- NCT06333041 NOT_YET_RECRUITINGRandomized, Double-Blind, Placebo-Controlled Cross-Over Study to Evaluate the Effects of Cannabidiol on Neurobehavioral and Function Outcomes in Sanfilippo SyndromePhase: PHASE2, PHASE3 Updated:Locations: The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, United States
- NCT03333200 RECRUITINGLongitudinal Study of Neurodegenerative DisordersUpdated:Locations: UPMC Children's Hospital of Pittsburgh, Pittsburgh, United States
- NCT06036693 RECRUITINGMucopolysaccharidosis Patients in France in the Era of Specific TherapeuticsUpdated:Locations: Centre Hospitalier Universitaire d'Angers, Angers, France; Hôpital des Enfants - Groupe Hospitalier Pellegrin, Bordeaux, France; Hôpital Morvan, Brest, France
- NCT05705674 RECRUITINGThe Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)Updated:Locations: LDRTC, Fairfax, United States
- NCT03227042 ACTIVE_NOT_RECRUITINGA Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)Updated:Locations: UCSF Benioff Children's Hospital Oakland, Oakland, United States; Hospital Universitario Austral, Pilar, Argentina; Murdoch Childrens Research Institute and Royal Children's Hospital, Melbourne, Australia
Disclaimer: Information changes frequently and may lag behind the registry. For medical decisions, consult investigators.