22 completed. 22 Phase 3, 7 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 22 | Completed 22 |
| Phase 2 | 7 | Completed 7 |
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 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 |
| 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 |
| 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 |
| A Study of JR-141 in Patients With Mucopolysaccharidosis II JR-141 - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Completed | JCR Pharmaceuticals Co., Ltd. | 28 | 2018-08-01 | 2022-11-14 |
| An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) Hunterase - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Completed | GC Biopharma Corp | 30 | 2018-05-23 | 2026-01-15 |
| A Study of GC1111 in Hunter Syndrom Patients GC1111, Comparator - Hunter Syndrome | Phase 3 | Completed | Green Cross Corporation | 32 | 2017-03-09 | 2024-04-16 |
| A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) UX003 - Sly Syndrome, MPS VII | Phase 3 | Completed | Ultragenyx Pharmaceutical Inc | 12 | 2015-11-10 | 2020-07-30 |
| Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 Idursulfase-IT, Elaprase - Hunter Syndrome | Phase 2 / Phase 3 | Completed | Shire | 56 | 2015-04-14 | 2025-06-19 |
| Probiotic Supplementation Among Adults With Recurrent Gastrointestinal Symptoms Probiotic dietary supplement (Trenev Trio®), placebo - Gastrointestinal Symptoms, Bloating | Phase 2 / Phase 3 | Completed | University of Maryland, Baltimore | 80 | 2014-12-01 | 2021-09-14 |
| A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) UX003, Placebo - MPS 7, Sly Syndrome | Phase 3 | Completed | Ultragenyx Pharmaceutical Inc | 12 | 2014-12-01 | 2020-07-30 |
| Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment idursulfase-IT, No IT treatment - Hunter Syndrome | Phase 2 / Phase 3 | Completed | Shire | 58 | 2014-03-24 | 2021-06-11 |
| BMN 110 Phase 3B in Australian Patients BMN 110 - Mucopolysaccharidosis IVA (Morquio A Syndrome) | Phase 3 | Completed | BioMarin Pharmaceutical | 13 | 2013-07-01 | 2019-09-26 |
| Safety and Efficacy of Hunterase Hunterase - Hunter Syndrome | Phase 3 | Completed | Green Cross Corporation | 6 | 2012-07-01 | 2014-07-08 |
| Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 - Weekly, BMN 110 - Every Other Week - Mucopolysaccharidosis IV A, Morquio A Syndrome | Phase 3 | Completed | BioMarin Pharmaceutical | 173 | 2011-07-01 | 2021-07-21 |
| A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 Weekly, Placebo, BMN 110 Every Other Week - MPS IV A | Phase 3 | Completed | BioMarin Pharmaceutical | 177 | 2011-02-01 | 2014-07-07 |
| Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease Aldurazyme (Recombinant Human Alpha-L-Iduronidase) - Mucopolysaccharidosis I, Hurler Syndrome | Phase 3 | Completed | Genzyme, a Sanofi Company | 3 | 2005-12-01 | 2014-03-07 |
| Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase Idursulfase - Hunter Syndrome, Mucopolysaccharidosis II (MPS II) | Phase 2 / Phase 3 | Completed | Shire | 94 | 2004-09-13 | 2021-06-10 |
| Study of rhASB in Patients With Mucopolysaccharidosis VI N-acetylgalactosamine 4-sulfatase, Placebo/rhASB - Mucopolysaccharidosis VI | Phase 3 | Completed | BioMarin Pharmaceutical | 39 | 2004-02-01 | 2010-02-02 |
| Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) Iduronate-2-sulfatase enzyme replacement therapy, iduronate-2-sulfatase enzyme replacement therapy, Placebo - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Completed | Shire | 96 | 2003-09-18 | 2021-06-10 |
| Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI Placebo, N-acetylgalactosamine 4-sulfatase - Mucopolysaccharidosis VI | Phase 3 | Completed | BioMarin Pharmaceutical | 39 | 2003-09-01 | 2009-12-24 |
Showing 20 of 22. Sign in to see all of them.
Mucopolysaccharidosis II (8) · Lysosomal Storage Diseases (6) · Mucopolysaccharidosis IV (6) · Sudden Infant Death (5) · Mucopolysaccharidosis I (4) · Metabolic Diseases (2) · Mucopolysaccharidoses (2) · Mucopolysaccharidosis VI (2) · Mucopolysaccharidosis VII (2) · Rare Diseases (2) · Abdominal Pain (1) · Achondroplasia (1) · Bone Diseases (1) · Dwarfism (1) · Dyspepsia (1)
Carbohydrate Metabolism, Inborn Errors (22) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (22) · Connective Tissue Diseases (22) · Metabolism, Inborn Errors (22) · Nutritional and Metabolic Diseases (22) · Skin and Connective Tissue Diseases (22) · Genetic Diseases, Inborn (21) · Metabolic Diseases (20) · Mucopolysaccharidoses (20) · Lysosomal Storage Diseases (16) · Neurologic Manifestations (9) · Genetic Diseases, X-Linked (8) · Heredodegenerative Disorders, Nervous System (8) · Intellectual Disability (8)
Iduronidase (3) · idursulfase (3) · Chondroitinsulfatases (2) · GALNS protein, human (2) · N-Acetylgalactosamine-4-Sulfatase (2) · vestronidase alfa (2) · Antipyretics (1) · Chondroitinases and Chondroitin Lyases (1) · Enzyme Replacement Therapy (1) · Galns protein, mouse (1) · Histamine Antagonists (1) · Iduronate Sulfatase (1) · MoviPrep (1) · infigratinib (1)
BioMarin Pharmaceutical (5) · Shire (4) · Genzyme, a Sanofi Company (3) · Green Cross Corporation (2) · Ultragenyx Pharmaceutical Inc (2) · Cinnagen (1) · GC Biopharma Corp (1) · JCR Pharmaceuticals Co., Ltd. (1) · NTC srl (1) · QED Therapeutics, a BridgeBio company (1) · University of Maryland, Baltimore (1)
United States (13) · Canada (8) · United Kingdom (8) · France (7) · Germany (7) · Brazil (6) · Italy (6) · South Korea (5) · Spain (5) · Australia (4) · Japan (4) · Argentina (3) · Mexico (3) · Netherlands (3) · Portugal (3)
Debra Lounsbury (2) · 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) · Mislen Bauer, MD (1) · Paul Harmatz, MD (1) · Raymond Wang, 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 36027721, PMID 36799346, PMID 40786127, PMID 40830667, PMID 42370681, PMID 42421116
2004 (1), 2014 (1), 2015 (1), 2019 (1)