10 active not recruiting. 10 Phase 1, 8 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 10 | Active not recruiting 10 |
| Phase 2 | 8 | Active not recruiting 8 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 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 |
| 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 |
| 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 |
Mucopolysaccharidosis II (4) · Mucopolysaccharidosis III (4) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis VI (2) · Mucopolysaccharidosis IV (1) · Sudden Infant Death (1)
Carbohydrate Metabolism, Inborn Errors (10) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Connective Tissue Diseases (10) · Genetic Diseases, Inborn (10) · Lysosomal Storage Diseases (10) · Metabolic Diseases (10) · Metabolism, Inborn Errors (10) · Mucopolysaccharidoses (10) · Nutritional and Metabolic Diseases (10) · Skin and Connective Tissue Diseases (10) · Genetic Diseases, X-Linked (4) · Heredodegenerative Disorders, Nervous System (4) · Intellectual Disability (4) · Nervous System Diseases (4)
Adalimumab (1) · Clinical Coding (1) · Injections (1) · Saline Solution (1) · vosoritide (1)
GC Biopharma Corp (2) · JCR Pharmaceuticals Co., Ltd. (2) · University of Manchester (2) · Denali Therapeutics Inc. (1) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · Orchard Therapeutics (1) · University of California, San Francisco (1)
United States (4) · United Kingdom (3) · South Korea (2) · Canada (1) · Germany (1) · Italy (1) · Japan (1) · Netherlands (1)
Robert Wynn (2) · Brian Bigger (1) · Ellen B Fung, PhD (1) · Lynda Polgreen, MD (1) · Sam Lu, MD (1)
2019 (1), 2023 (1)