4 recruiting. 4 Phase 2, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Recruiting 4 |
| Phase 3 | 2 | Recruiting 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 |
|---|---|---|---|---|---|---|
| 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 |
| Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration - Achondroplasia | Phase 2 | Recruiting | QED Therapeutics, a BridgeBio company | 77 | 2025-11-19 | 2026-07-13 |
| Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy - MPS IIIA, Sanfilippo Syndrome | Phase 2 / Phase 3 | Recruiting | Ultragenyx Pharmaceutical Inc | 36 | 2016-04-25 | 2026-09-22 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
Mucopolysaccharidosis III (2) · Achondroplasia (1) · Adrenoleukodystrophy (1) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Arthritis, Juvenile (1) · Autoimmune Lymphoproliferative Syndrome (1) · Bone Diseases (1) · Chediak-Higashi Syndrome (1) · Congenital Bone Marrow Failure Syndromes (1) · Congenital amegakaryocytic thrombocytopenia (1) · Crohn Disease (1) · Dwarfism (1) · Dyskeratosis Congenita (1) · Gaucher Disease (1)
Carbohydrate Metabolism, Inborn Errors (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Connective Tissue Diseases (4) · Genetic Diseases, Inborn (4) · Lysosomal Storage Diseases (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Nutritional and Metabolic Diseases (4) · Skin and Connective Tissue Diseases (4) · Mucopolysaccharidoses (3) · Bone Diseases, Developmental (2) · Endocrine System Diseases (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2)
Administration, Oral (1) · Alemtuzumab (1) · Cannabidiol (1) · Hydroxyurea (1) · Melphalan (1) · Therapeutics (1) · Thiotepa (1) · fludarabine (1) · fludarabine phosphate (1) · infigratinib (1)
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · Paul Szabolcs (1) · QED Therapeutics, a BridgeBio company (1) · Ultragenyx Pharmaceutical Inc (1)
United States (4) · Australia (2) · Spain (2) · Canada (1) · Norway (1) · Singapore (1) · United Kingdom (1)
PMID 32634238, PMID 32750127, PMID 32884151
2015 (1), 2023 (1)