24 completed, 9 terminated, 6 active not recruiting, 6 unknown. 27 Phase 2, 20 Phase 1, 7 Phase 3, 1 Not applicable, 1 Phase 4.
Follow this condition Literature Review of Mucopolysaccharidosis III trials Research Report on Mucopolysaccharidosis III
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 |
|---|---|---|---|---|---|---|
| Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB rAAV9-CMV-hNAGLUop - Sanfilippo Syndrome (MPS III) | Phase 1 / Phase 2 | Not yet recruiting | NeuroGT | 9 | 2027-01-18 | 2026-09-14 |
| Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B) Tralesinidase alfa (TA) - MPS IIIB | Phase 3 | Not yet recruiting | Spruce Biosciences | 14 | 2026-12-01 | 2026-09-21 |
| MPS IIIA Disease Monitoring Program No Intervention - MPS IIIA, Sanfilippo Syndrome | Not yet recruiting | Ultragenyx Pharmaceutical Inc | 55 | 2026-11-01 | 2026-10-02 | |
| Natural History to Assess Disease in Patients With MPS IIIC Sanfilippo Syndrome Type C, Mucopolysaccharidosis (MPS) IIIC | Not yet recruiting | Phoenix Nest | 35 | 2026-11-01 | 2026-07-17 | |
| 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 |
| Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA) GC1130A - Sanfilippo Syndrome Type A | Phase 1 | Active not recruiting | GC Biopharma Corp | 9 | 2024-11-21 | 2026-09-08 |
| Natural History Study of Participants With Sanfilippo Syndrome Type IIIC Sanfilippo Syndrome Type C | Recruiting | Phoenix Nest | 30 | 2024-11-10 | 2026-05-12 | |
| An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB JR-446 - Mucopolysaccharidosis III-B | Phase 1 / Phase 2 | Active not recruiting | JCR Pharmaceuticals Co., Ltd. | 10 | 2024-11-05 | 2026-10-01 |
| Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) DNL126 - Mucopolysaccharidosis Type IIIA | Phase 1 / Phase 2 | Active not recruiting | Denali Therapeutics Inc. | 20 | 2023-12-07 | 2026-07-13 |
| Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA JR-441 - Mucopolysaccharidosis III-A | Phase 1 / Phase 2 | Active not recruiting | JCR Pharmaceuticals Co., Ltd. | 15 | 2023-10-04 | 2026-09-04 |
| 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 | |
| A Natural History Study of Sanfilippo Syndrome Type D Sanfilippo Syndrome Type D | Completed | Phoenix Nest | 10 | 2023-01-22 | 2025-08-01 | |
| Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE AX 250 - MPS III B | Phase 4 | Enrolling by invitation | Allievex Corporation | 15 | 2022-12-02 | 2023-08-09 |
| A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome) No intervention - Mucopolysaccharidosis III-A | Completed | Sanguine Biosciences | 15 | 2022-09-27 | 2025-01-30 | |
| Natural History Observational Study of MPS IIIa in SMC assessment - Sanfilippo Syndrome A | Unknown | Samsung Medical Center | 25 | 2021-06-30 | 2021-06-09 | |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program Confirmatory Testing - Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2 | Enrolling by invitation | Albert Einstein College of Medicine | 100000 | 2021-05-10 | 2026-07-28 | |
| A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101 ABO-101 - Mucopolysaccharidosis III-B | Terminated | Abeona Therapeutics, Inc | 1 | 2020-10-28 | 2022-05-31 | |
| 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 |
| Open-label Study of Anakinra in MPS III anakinra - Mucopolysaccharidosis III | Phase 1 / Phase 2 | Completed | Lynda E Polgreen | 24 | 2020-01-30 | 2024-10-22 |
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Mucopolysaccharidoses (12) · Lysosomal Storage Diseases (9) · Mucopolysaccharidosis II (8) · Leukodystrophy, Globoid Cell (7) · Mucopolysaccharidosis I (7) · Leukodystrophy, Metachromatic (6) · Mucopolysaccharidosis VI (6) · Genetic Diseases, Inborn (5) · Metabolic Diseases (5) · Metabolism, Inborn Errors (5) · Sandhoff Disease (5) · Sudden Infant Death (5) · Tay-Sachs Disease (5) · Adrenoleukodystrophy (4)
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Alemtuzumab (2) · Clofarabine (2) · Hematopoietic Stem Cell Transplantation (2) · Melphalan (2) · Restraint, Physical (2) · Therapeutics (2) · Whole-Body Irradiation (2) · Ambroxol (1) · Cannabidiol (1) · Cyclosporine (1) · Hydroxyurea (1) · Interleukin 1 Receptor Antagonist Protein (1) · Magnetic Resonance Imaging (1) · Mycophenolic Acid (1) · Palliative Care (1)
Shire (6) · Alexion Pharmaceuticals, Inc. (5) · Allievex Corporation (5) · Ultragenyx Pharmaceutical Inc (4) · LYSOGENE (3) · Phoenix Nest (3) · Abeona Therapeutics, Inc (2) · JCR Pharmaceuticals Co., Ltd. (2) · Masonic Cancer Center, University of Minnesota (2) · Spruce Biosciences (2) · Swedish Orphan Biovitrum (2) · University of Minnesota (2) · Albert Einstein College of Medicine (1) · Aldagen (1) · Denali Therapeutics Inc. (1)
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PMID 35709957, PMID 22547151, PMID 25421091, PMID 32884151, PMID 35835061, PMID 21586746, PMID 24582005, PMID 25770355, PMID 26787381, PMID 26956723, PMID 27211612, PMID 29661560, PMID 30528227, PMID 31315600, PMID 33008435, PMID 34040605, PMID 34600820, PMID 35142618, PMID 36413418, PMID 38907160
2019 (3), U54NS065768 (3), 2015 (2), 2023 (2), 1UB1NS122644-01A1 (1), 2009 (1), 2014 (1), 2018 (1), R01HD073292 (1)