3 terminated, 2 completed, 2 recruiting, 2 unknown. 8 Phase 2, 4 Phase 3, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Terminated 3, Completed 2, Recruiting 2, Unknown 1 |
| Phase 3 | 4 | Recruiting 2, Enrolling by invitation 1, Unknown 1 |
| Phase 1 | 2 | Terminated 2 |
Follow Sanfilippo Syndrome: new trials by email Literature Review of Sanfilippo Syndrome trials Research Report on Sanfilippo Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| MPS IIIA Disease Monitoring Program No Intervention - MPS IIIA, Sanfilippo Syndrome | Not yet recruiting | Ultragenyx Pharmaceutical Inc | 55 | 2026-11-01 | 2026-10-02 | |
| Study of Cannabidiol in Sanfilippo Syndrome Epidiolex, Placebo - Sanfilippo Syndrome, Mucopolysaccharidosis III | Phase 2 / Phase 3 | Recruiting | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 35 | 2026-04-01 | 2026-09-15 |
| An 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 III Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day - Sanfilippo Syndrome, MPS3 | Phase 2 / Phase 3 | Unknown | Ozlem Goker-Alpan | 10 | 2024-12-01 | 2025-08-20 |
| The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3) Sanfilippo Syndrome, MPS3 | Unknown | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | 6 | 2023-05-01 | 2024-03-12 | |
| Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy - Mucopolysaccharidosis IIIA, MPS IIIA | Phase 3 | Enrolling by invitation | Ultragenyx Pharmaceutical Inc | 41 | 2020-09-28 | 2026-07-17 |
| Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease ABO-102 - MPS IIIA, Sanfilippo Syndrome | Phase 1 / Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 5 | 2019-09-18 | 2023-07-25 |
| Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy - MPS IIIA, Sanfilippo Syndrome | Phase 2 / Phase 3 | Recruiting | Ultragenyx Pharmaceutical Inc | 36 | 2016-04-25 | 2026-09-22 |
| An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. HGT-1410 - Sanfilippo Syndrome, Mucopolysaccharidosis (MPS) | Phase 2 | Terminated | Shire | 17 | 2015-04-08 | 2021-06-11 |
| Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease Recombinant human heparan N-sulfatase [rhHNS] - Sanfilippo Syndrome | Phase 2 | Completed | Shire | 21 | 2014-02-26 | 2021-06-08 |
| Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) rhHNS-10 mg, rhHNS-45 mg, rhHNS-90 mg - Sanfilippo Syndrome | Phase 1 / Phase 2 | Terminated | Shire | 12 | 2011-03-01 | 2021-06-14 |
| HSCT for High Risk Inherited Inborn Errors Clofarabine, Total body Irradiation, Melphalan - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 38 | 2006-09-01 | 2019-07-11 |
Mucopolysaccharidosis III (11) · Lysosomal Storage Diseases (2) · Adrenoleukodystrophy (1) · Gangliosidosis, GM1 (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Metabolism, Inborn Errors (1) · Mucolipidoses (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis II (1) · Sandhoff Disease (1) · Tay-Sachs Disease (1) · Wolman Disease (1)
Carbohydrate Metabolism, Inborn Errors (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Connective Tissue Diseases (11) · Genetic Diseases, Inborn (11) · Metabolic Diseases (11) · Mucinoses (11) · Nutritional and Metabolic Diseases (11) · Skin and Connective Tissue Diseases (11) · Metabolism, Inborn Errors (10) · Mucopolysaccharidoses (10) · Lysosomal Storage Diseases (9) · Genetic Diseases, X-Linked (2) · Heredodegenerative Disorders, Nervous System (2) · Intellectual Disability (2) · Nervous System Diseases (2)
Therapeutics (2) · Alemtuzumab (1) · Ambroxol (1) · Cannabidiol (1) · Clofarabine (1) · Hematopoietic Stem Cell Transplantation (1) · Hydroxyurea (1) · Melphalan (1) · Tablets (1) · Whole-Body Irradiation (1)
Ultragenyx Pharmaceutical Inc (4) · Shire (3) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · Lysosomal and Rare Disorders Research and Treatment Center, Inc. (1) · Masonic Cancer Center, University of Minnesota (1) · Ozlem Goker-Alpan (1)
United States (9) · Spain (5) · Australia (3) · Netherlands (3) · United Kingdom (3) · France (2) · Germany (2) · Italy (2) · Argentina (1)
Ozlem Goker-Alpan, MD (1) · Paul Orchard, MD (1)
PMID 21586746, PMID 22547151, PMID 25421091, PMID 30528227, PMID 32884151, PMID 34600820
2023 (2), 2014 (1), 2015 (1), 2018 (1), 2019 (1)