4 enrolling by invitation. 4 Phase 2, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Enrolling by invitation 4 |
| Phase 3 | 2 | Enrolling by invitation 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 |
|---|---|---|---|---|---|---|
| Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia Infigratinib - Hypochondroplasia | Phase 2 | Enrolling by invitation | QED Therapeutics, a BridgeBio company | 135 | 2026-04-23 | 2026-08-26 |
| 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 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 |
| Extension Study of Infigratinib in Children With Achondroplasia (ACH) Infigratinib - Achondroplasia | Phase 2 | Enrolling by invitation | QED Therapeutics, a BridgeBio company | 325 | 2021-12-06 | 2026-10-26 |
Bone Diseases (3) · Genetic Diseases, Inborn (3) · Mucopolysaccharidosis IV (3) · Musculoskeletal Diseases (3) · Osteochondrodysplasias (3) · Dwarfism (2) · Hypochondroplasia (2) · Achondroplasia (1) · Mucopolysaccharidosis II (1) · Sudden Infant Death (1)
Carbohydrate Metabolism, Inborn Errors (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Connective Tissue Diseases (4) · Lysosomal Storage Diseases (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Mucopolysaccharidoses (4) · Nutritional and Metabolic Diseases (4) · Skin and Connective Tissue Diseases (4) · Bone Diseases, Developmental (3) · Endocrine System Diseases (2) · Death (1) · Death, Sudden (1) · Dwarfism (1)
infigratinib (3)
QED Therapeutics, a BridgeBio company (3) · Denali Therapeutics Inc. (1)
Canada (4) · France (4) · Spain (4) · United Kingdom (4) · United States (4) · Australia (3) · Norway (3) · Singapore (3) · Sweden (3) · Argentina (2) · Italy (2) · Portugal (2) · Belgium (1) · Brazil (1) · Czechia (1)
2023 (1)