16 active not recruiting. 16 Phase 2, 9 Phase 1, 5 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 16 | Active not recruiting 16 |
| Phase 1 | 9 | Active not recruiting 9 |
| Phase 3 | 5 | Active not recruiting 5 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 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 |
| 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 |
| Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene - Mucopolysaccharidosis Type IIIA | Phase 1 / Phase 2 | Active not recruiting | University of Manchester | 5 | 2020-01-07 | 2025-03-30 |
| 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 |
| An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II JR-141 - Mucopolysaccharidosis II | Phase 2 | Active not recruiting | JCR Pharmaceuticals Co., Ltd. | 19 | 2019-01-01 | 2026-09-08 |
| 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 |
| Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium. - Mucopolysaccharidosis IH | Phase 1 / Phase 2 | Active not recruiting | Orchard Therapeutics | 8 | 2018-05-11 | 2026-07-16 |
| Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI Adalimumab Injection [Humira], Saline Solution for Injection - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Phase 1 / Phase 2 | Active not recruiting | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 14 | 2017-06-05 | 2026-02-02 |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-01-07 |
Mucopolysaccharidosis II (10) · Mucopolysaccharidosis III (4) · Sudden Infant Death (4) · Mucopolysaccharidosis I (3) · Mucopolysaccharidosis VI (3) · Lysosomal Storage Diseases (2) · Alpha-Methylacyl-CoA Racemase Deficiency (1) · Aspartylglucosaminuria (1) · Cognition Disorders (1) · Cognitive Dysfunction (1) · Connective Tissue Diseases (1) · Fucosidosis (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1) · Hereditary Diffuse Leukoencephalopathy with Spheroids (1)
Carbohydrate Metabolism, Inborn Errors (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Metabolism, Inborn Errors (16) · Mucopolysaccharidoses (16) · Nutritional and Metabolic Diseases (16) · Skin and Connective Tissue Diseases (16) · Connective Tissue Diseases (15) · Genetic Diseases, Inborn (15) · Metabolic Diseases (15) · Lysosomal Storage Diseases (14) · Genetic Diseases, X-Linked (9) · Heredodegenerative Disorders, Nervous System (9) · Intellectual Disability (9) · Nervous System Diseases (9)
idursulfase (2) · Adalimumab (1) · Clinical Coding (1) · Injections (1) · Regimen B (1) · Saline Solution (1) · Stem Cell Transplantation (1) · vosoritide (1)
JCR Pharmaceuticals Co., Ltd. (4) · Denali Therapeutics Inc. (3) · University of Manchester (2) · AO GENERIUM (1) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · Masonic Cancer Center, University of Minnesota (1) · Orchard Therapeutics (1) · REGENXBIO Inc. (1) · Takeda (1) · University of California, San Francisco (1)
United States (7) · United Kingdom (4) · Brazil (3) · Canada (3) · France (2) · Germany (2) · Italy (2) · Japan (2) · Netherlands (2) · Argentina (1) · Belgium (1) · Czechia (1) · Russia (1) · Spain (1) · Sweden (1)
Robert Wynn (2) · Ana-Claire Meyer, MD (1) · Brian Bigger (1) · Ellen B Fung, PhD (1) · Jose Alcantara Rodriguez, PharmD (1) · Lynda Polgreen, MD (1) · Oksana A. Markova, MD, MSc (1) · Paul Orchard, M.D. (1) · Sam Lu, MD (1)
2023 (2), 2019 (1)