Clinical Trials
Live snapshot from ClinicalTrials.gov, Sanfilippo Syndrome clinical trials (Recruiting / Not yet recruiting / Active, not recruiting).
13 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
- 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
- NCT07579910 NOT_YET_RECRUITINGA Phase 3, Randomized, Single-blind, Parallel Group, Controlled, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebroventricular Tralesinidase Alfa Treatment Compared to Standard of Care in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)Phase: PHASE3 Updated:
- NCT06333041 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
- 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
- NCT03333200 RECRUITINGLongitudinal Study of Neurodegenerative DisordersUpdated:Locations: UPMC Children's Hospital of Pittsburgh, Pittsburgh, United States
- 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
- 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
Disclaimer: Information changes frequently and may lag behind the registry. For medical decisions, consult investigators.