45 completed, 11 terminated, 10 active not recruiting, 5 recruiting. 76 Phase 1, 52 Phase 2.
Follow Mucinoses: new trials by email Literature Review of Mucinoses trials Research Report on Mucinoses
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 |
| 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 |
| 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 |
| A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I JWK008 Single intravenous infusion administration - Mucopolysaccharidosis Type I | Phase 1 | Recruiting | West China Hospital | 6 | 2024-07-22 | 2024-07-25 |
| Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) DNL126, Prophylactic immunomodulatory therapy - Mucopolysaccharidosis Type IIIA | Phase 1 / Phase 2 | Recruiting | Denali Therapeutics Inc. | 30 | 2023-12-07 | 2026-11-02 |
| 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 |
| Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI Vosoritide Injection [Voxzogo] - MPS IVA, MPS VI | Phase 1 / Phase 2 | Active not recruiting | University of California, San Francisco | 6 | 2023-09-25 | 2026-03-10 |
| Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII - Mucopolysaccharidosis II | Phase 1 / Phase 2 | Active not recruiting | University of Manchester | 5 | 2023-06-01 | 2026-03-27 |
| ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I Autologous Plasmablasts (B cells) - Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS | Phase 1 | Recruiting | Immusoft of CA, Inc. | 11 | 2023-04-12 | 2026-07-17 |
| A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers verenafusp alfa 6 mg/kg, verenafusp alfa 9 mg/kg, verenafusp alfa 12 mg/kg - Mucopolysaccharidosis Type II, Metabolic Diseases | Phase 1 | Completed | AO GENERIUM | 18 | 2023-04-06 | 2024-07-03 |
| A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ GC1123 - Mucopolysaccharidosis II, Hunter Syndrome | Phase 1 | Active not recruiting | GC Biopharma Corp | 12 | 2022-09-20 | 2024-10-15 |
| Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II Genetic HMI-203 - Mucopolysaccharidosis II | Phase 1 | Withdrawn | Homology Medicines, Inc | 2022-09-08 | 2023-08-28 | |
| Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy SIG-005 (hIDUA Producing Spheres) - MPS I, MPS1-S | Phase 1 / Phase 2 | Withdrawn | Sigilon Therapeutics, Inc. | 2021-11-15 | 2023-04-11 | |
| An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I) JR-171 - Mucopolysaccharidosis I | Phase 1 / Phase 2 | Completed | JCR Pharmaceuticals Co., Ltd. | 14 | 2021-10-28 | 2025-08-28 |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) RGX-121 - Mucopolysaccharidosis Type II (MPS II) | Phase 1 / Phase 2 | Completed | REGENXBIO Inc. | 6 | 2021-03-11 | 2025-01-28 |
| A Study of JR-171 in Patients With Mucopolysaccharidosis I JR-171 (lepunafusp alfa) - Mucopolysaccharidosis I | Phase 1 / Phase 2 | Completed | JCR Pharmaceuticals Co., Ltd. | 18 | 2020-09-01 | 2025-03-19 |
| A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome tividenofusp alfa - Mucopolysaccharidosis II | Phase 1 / Phase 2 | Active not recruiting | Denali Therapeutics Inc. | 47 | 2020-07-16 | 2025-08-07 |
| Comparison of Thread Technique Versus Aspiration Plus Steroid Injection for Wrist Ganglion Recurrence methyl prednisolone acetate, Silk suture - Ganglion Cyst | Phase 1 / Phase 2 | Completed | Benazir Bhutto Hospital, Rawalpindi | 60 | 2020-06-01 | 2025-01-24 |
| A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers GNR-055 - Mucopolysaccharidosis Type II, Metabolic Diseases | Phase 1 | Completed | AO GENERIUM | 20 | 2020-02-28 | 2020-10-14 |
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Mucopolysaccharidosis I (28) · Mucopolysaccharidosis II (20) · Mucopolysaccharidosis III (20) · Lysosomal Storage Diseases (14) · Mucopolysaccharidoses (12) · Sudden Infant Death (10) · Metabolic Diseases (8) · Mucopolysaccharidosis IV (8) · Genetic Diseases, Inborn (7) · Mucopolysaccharidosis VI (7) · Metabolism, Inborn Errors (6) · Anemia, Diamond-Blackfan (5) · Adrenoleukodystrophy (4) · Anemia, Sickle Cell (4) · Granulomatous Disease, Chronic (4)
Skin and Connective Tissue Diseases (75) · Connective Tissue Diseases (74) · Nutritional and Metabolic Diseases (74) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (73) · Carbohydrate Metabolism, Inborn Errors (72) · Metabolism, Inborn Errors (68) · Genetic Diseases, Inborn (67) · Metabolic Diseases (66) · Mucopolysaccharidoses (62) · Lysosomal Storage Diseases (60) · Nervous System Diseases (32) · Genetic Diseases, X-Linked (23) · Pathological Conditions, Signs and Symptoms (23) · Heredodegenerative Disorders, Nervous System (22)
Iduronidase (6) · valanafusp alpha (4) · GALNS protein, human (3) · Adalimumab (2) · idursulfase (2) · Abatacept (1) · Alemtuzumab (1) · Azathioprine (1) · Botulinum Toxins, Type A (1) · Clinical Coding (1) · Clinical Trials, Phase I as Topic (1) · Clinical Trials, Phase II as Topic (1) · Combined Modality Therapy (1) · Cyclosporine (1) · GAA protein, human (1)
ArmaGen, Inc (5) · JCR Pharmaceuticals Co., Ltd. (5) · BioMarin Pharmaceutical (3) · Shire (3) · AO GENERIUM (2) · Alexion Pharmaceuticals, Inc. (2) · Denali Therapeutics Inc. (2) · GC Biopharma Corp (2) · Joanne Kurtzberg, MD (2) · LYSOGENE (2) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (2) · Masonic Cancer Center, University of Minnesota (2) · Patricia I. Dickson, M.D. (2) · REGENXBIO Inc. (2) · Sangamo Therapeutics (2)
United States (44) · United Kingdom (14) · Brazil (7) · Canada (6) · France (5) · Germany (5) · Japan (5) · Netherlands (4) · Spain (4) · Turkey (Türkiye) (4) · Russia (3) · South Korea (3) · Finland (2) · Italy (2) · Australia (1)
Patrice Rioux, MD PhD (3) · Celeste Decker, MD (2) · Dugald Seely, ND (2) · Joanne Kurtzberg, MD (2) · Patrice P Rioux, MD PhD (2) · Paul Harmatz, MD (2) · Paul Orchard, MD (2) · Robert Wynn (2) · Abdul Basit, MBBS, FCPS (1) · Ana-Claire Meyer, MD (1) · Biljana Horn, MD (1)
PMID 22547151, PMID 25421091, PMID 29526614, PMID 29976218, PMID 35835061, PMID 36299240, PMID 15324318, PMID 19948615, PMID 21637107, PMID 23497636, PMID 23510062, PMID 24296492, PMID 27211612, PMID 28119821, PMID 28374935, PMID 28657663, PMID 28969890, PMID 30177353, PMID 30337566, PMID 30467742, PMID 30595526, PMID 31575939, PMID 32102934, PMID 32278382, PMID 32583902, PMID 32884151, PMID 34040605, PMID 34600820, PMID 35588317, PMID 35709957, PMID 36413418, PMID 37624739, PMID 37955580, PMID 38204164, PMID 38319253, PMID 38907160, PMID 39547230, PMID 39891377, PMID 41467650, PMID 9918480
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