22 completed, 8 active not recruiting, 4 enrolling by invitation, 3 unknown. 43 Phase 3, 20 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 |
|---|---|---|---|---|---|---|
| A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP) garetosmab - Fibrodysplasia Ossificans Progressiva, FOP | Phase 3 | Not yet recruiting | Regeneron Pharmaceuticals | 18 | 2027-02-02 | 2026-10-20 |
| 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 |
| 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 |
| RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) RGX-121-3102 - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Active not recruiting | REGENXBIO Inc. | 2 | 2025-11-25 | 2026-06-04 |
| An Interventional Study of Infigratinib in Children With Hypochondroplasia infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day - Hypochondroplasia | Phase 2 / Phase 3 | Enrolling by invitation | QED Therapeutics, a BridgeBio company | 24 | 2025-04-22 | 2026-07-16 |
| 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 |
| Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment Idursulfase-IT, Elaprase - Hunter Syndrome | Phase 2 / Phase 3 | Active not recruiting | Takeda | 6 | 2024-03-05 | 2025-11-06 |
| A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT Experimental: OTL-203, Active Comparator: Allo-HSCT - MPS-IH (Hurler Syndrome) | Phase 3 | Active not recruiting | Orchard Therapeutics | 41 | 2023-12-11 | 2025-12-22 |
| A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day - Achondroplasia | Phase 3 | Completed | QED Therapeutics, a BridgeBio company | 114 | 2023-11-10 | 2026-03-17 |
| An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 tividenofusp alfa - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Enrolling by invitation | Denali Therapeutics Inc. | 99 | 2023-09-20 | 2026-10-27 |
| An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects JR-141 - Mucopolysaccharidosis II | Phase 3 | Enrolling by invitation | JCR Pharmaceuticals Co., Ltd. | 80 | 2023-02-22 | 2026-01-21 |
| Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients Laronidase, Antihistamine, Antipyretic - Mucopolysaccharidosis Type 1 | Phase 3 | Completed | Cinnagen | 12 | 2022-09-10 | 2024-08-06 |
| A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) tividenofusp alfa, idursulfase - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Active not recruiting | Denali Therapeutics Inc. | 65 | 2022-07-21 | 2026-09-09 |
| A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) JR-141, Idursulfase, JR-141 or Idursulfase - Mucopolysaccharidosis II | Phase 3 | Active not recruiting | JCR Pharmaceuticals Co., Ltd. | 86 | 2022-02-14 | 2026-10-02 |
| A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg - Mucopolysaccharidosis Type II, Metabolic Diseases | Phase 2 / Phase 3 | Active not recruiting | AO GENERIUM | 32 | 2021-11-30 | 2026-09-21 |
| 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 |
| Efficacy and Safety of mAnnitol in Bowel Preparation During Elective Colonoscopy and Comparison With Moviprep® Phase II: NTC015 low dose, Phase II: NTC015 medium dose, Phase II: NTC015 high dose - Elective Colonoscopy | Phase 2 / Phase 3 | Completed | NTC srl | 886 | 2020-06-18 | 2025-12-08 |
| An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II JR-141 - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Active not recruiting | JCR Pharmaceuticals Co., Ltd. | 27 | 2019-09-01 | 2026-09-03 |
| Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) LYS-SAF302 - Mucopolysaccharidosis Type IIIA | Phase 2 / Phase 3 | Unknown | LYSOGENE | 20 | 2018-12-17 | 2021-08-31 |
| CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) RGX-121 - Mucopolysaccharidosis Type II (MPS II) | Phase 2 / Phase 3 | Active not recruiting | REGENXBIO Inc. | 48 | 2018-09-27 | 2025-01-28 |
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Mucopolysaccharidosis II (19) · Sudden Infant Death (12) · Lysosomal Storage Diseases (10) · Mucopolysaccharidosis IV (8) · Mucopolysaccharidosis I (7) · Mucopolysaccharidosis III (7) · Mucopolysaccharidoses (5) · Mucopolysaccharidosis VI (4) · Dwarfism (3) · Genetic Diseases, Inborn (3) · Metabolic Diseases (3) · Bone Diseases (2) · Mucopolysaccharidosis VII (2) · Musculoskeletal Diseases (2) · Osteochondrodysplasias (2)
Carbohydrate Metabolism, Inborn Errors (43) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (43) · Nutritional and Metabolic Diseases (43) · Skin and Connective Tissue Diseases (43) · Connective Tissue Diseases (42) · Metabolism, Inborn Errors (42) · Genetic Diseases, Inborn (40) · Metabolic Diseases (40) · Mucopolysaccharidoses (38) · Lysosomal Storage Diseases (33) · Neurologic Manifestations (19) · Genetic Diseases, X-Linked (18) · Heredodegenerative Disorders, Nervous System (18) · Intellectual Disability (18)
idursulfase (6) · Iduronidase (3) · Chondroitinsulfatases (2) · GALNS protein, human (2) · N-Acetylgalactosamine-4-Sulfatase (2) · Therapeutics (2) · infigratinib (2) · vestronidase alfa (2) · Ambroxol (1) · Antilymphocyte Serum (1) · Antipyretics (1) · Cannabidiol (1) · Chondroitinases and Chondroitin Lyases (1) · Cyclophosphamide (1) · Enzyme Replacement Therapy (1)
BioMarin Pharmaceutical (5) · JCR Pharmaceuticals Co., Ltd. (4) · Shire (4) · Ultragenyx Pharmaceutical Inc (4) · Genzyme, a Sanofi Company (3) · Denali Therapeutics Inc. (2) · Green Cross Corporation (2) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (2) · QED Therapeutics, a BridgeBio company (2) · REGENXBIO Inc. (2) · AO GENERIUM (1) · Aldagen (1) · Cinnagen (1) · GC Biopharma Corp (1) · LYSOGENE (1)
United States (28) · France (14) · United Kingdom (14) · Canada (12) · Germany (12) · Spain (12) · Brazil (11) · Italy (11) · Argentina (7) · Australia (7) · Netherlands (7) · Japan (5) · South Korea (5) · Portugal (4) · Sweden (4)
Debra Lounsbury (2) · Alexander Schramm (1) · Ali Rabani, Professor (1) · Chester Whitley, MD (1) · Chris D'Adamo, PhD (1) · Dong-Kyu Jin (1) · DongKyu Jin, M.D., Ph.D. (1) · Eric He, MD (1) · Gianpiero Manes, Dr. (1) · James Hinson, MD (1) · Joanne Kurtzberg, MD (1)
PMID 33874971, PMID 32063397, PMID 35961250, PMID 37415957, PMID 37915038, PMID 19117887, PMID 21150784, PMID 22929184, PMID 23837440, PMID 25487082, PMID 27955919, PMID 28535791, PMID 29248359, PMID 29478819, PMID 30467742, PMID 32884151, PMID 36027721, PMID 36799346, PMID 40786127, PMID 40830667, PMID 42370681, PMID 42421116
2023 (4), 2015 (2), 2019 (2), 2004 (1), 2014 (1)