169 completed, 39 terminated, 30 recruiting, 30 unknown. 104 Phase 2, 76 Phase 1, 43 Phase 3, 38 Not applicable, 17 Phase 4.
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 Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP) garetosmab - Fibrodysplasia Ossificans Progressiva, FOP | Phase 3 | Not yet recruiting | Regeneron Pharmaceuticals | 18 | 2027-02-02 | 2026-10-20 |
| Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB rAAV9-CMV-hNAGLUop - Sanfilippo Syndrome (MPS III) | Phase 1 / Phase 2 | Not yet recruiting | NeuroGT | 9 | 2027-01-18 | 2026-09-14 |
| Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B) Tralesinidase alfa (TA) - MPS IIIB | Phase 3 | Not yet recruiting | Spruce Biosciences | 14 | 2026-12-01 | 2026-09-21 |
| MPS IIIA Disease Monitoring Program No Intervention - MPS IIIA, Sanfilippo Syndrome | Not yet recruiting | Ultragenyx Pharmaceutical Inc | 55 | 2026-11-01 | 2026-10-02 | |
| Natural History to Assess Disease in Patients With MPS IIIC Sanfilippo Syndrome Type C, Mucopolysaccharidosis (MPS) IIIC | Not yet recruiting | Phoenix Nest | 35 | 2026-11-01 | 2026-07-17 | |
| Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis intra-oral 3D optical impression - Osteogenesis Imperfecta, Rare Bone Disorders | Not applicable | Not yet recruiting | University Hospital, Bordeaux | 240 | 2026-09-01 | 2026-06-24 |
| A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating FODMAP-targeting digestive enzyme blend, Placebo - IBS (Irritable Bowel Syndrome), Bloating | Not applicable | Recruiting | Kiwi Health Inc | 150 | 2026-05-04 | 2026-05-15 |
| 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 |
| 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 |
| Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy Participants Bloat Gummies, Placebo - Digestive Health, Abdominal Discomfort | Not applicable | Not yet recruiting | Arrae | 100 | 2026-04-01 | 2026-04-13 |
| Physical Activity Intervention for MPS Interviews/focus groups/participatory workshops - Mucopolysaccharidosis (MPS) | Recruiting | Brunel University | 50 | 2026-03-20 | 2026-08-19 | |
| Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients Evaluation of the prevalence of truly (authentified) idiopathic short stature after multidisciplinary clinico-radiological evaluation, analysis in a multidisciplinary consultation meeting via a secure platform (ShareConfrère) for evaluation by multidisciplinary team, Whole genome analysis for authentified idiopathic short stature - Idiopathic Short Stature | Not applicable | Recruiting | University Hospital, Montpellier | 200 | 2026-03-16 | 2026-05-06 |
| Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes Probiotic, Placebo - Digestive Disease, Gut Health | Not applicable | Completed | Synbiotic Health | 92 | 2026-01-20 | 2026-10-19 |
| Ganglion Resection Effectiveness Trial Open resection, Arthroscopic resection, Conservative treatment - Wrist Ganglion, Ganglion | Not applicable | Recruiting | Tampere University Hospital | 220 | 2026-01-13 | 2026-10-22 |
| Radicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health Outcomes GI Health Active Product 1, GI Health Product Placebo Control - Gas and Bloating, Diarrhea | Not applicable | Completed | Radicle Science | 500 | 2025-12-16 | 2026-05-28 |
| RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) RGX-121-3102 - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Active not recruiting | REGENXBIO Inc. | 2 | 2025-11-25 | 2026-06-04 |
| 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 |
| A Study to Evaluate the Effects of a Butyrate-Polyphenol Formulation on Gut Health and Associated Symptoms Butyrate + Polyphenol Formulation, Placebo - Gut Health | Not applicable | Completed | Supplement Formulators, Inc. | 124 | 2025-10-16 | 2026-08-26 |
| Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital Mucopolysaccharidoses, Progressive Pseudorheumatoid Dysplasia | Not yet recruiting | Assiut University | 35 | 2025-09-01 | 2025-09-15 | |
| Cardiac Structure and Function in MPS MPS I, MPS IV A | Recruiting | Children's Hospital of Orange County | 240 | 2025-09-01 | 2026-10-21 |
Showing 20 of 333. Sign in to see all of them.
Mucopolysaccharidosis IV (101) · Mucopolysaccharidosis II (77) · Mucopolysaccharidosis I (74) · Mucopolysaccharidosis III (58) · Mucopolysaccharidoses (57) · Lysosomal Storage Diseases (42) · Sudden Infant Death (37) · Mucopolysaccharidosis VI (36) · Genetic Diseases, Inborn (21) · Leukodystrophy, Metachromatic (21) · Leukodystrophy, Globoid Cell (19) · Ganglion Cysts (18) · Mucopolysaccharidosis VII (18) · Metabolism, Inborn Errors (16) · Adrenoleukodystrophy (14)
Skin and Connective Tissue Diseases (332) · Connective Tissue Diseases (329) · Nutritional and Metabolic Diseases (313) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (312) · Carbohydrate Metabolism, Inborn Errors (309) · Metabolic Diseases (299) · Metabolism, Inborn Errors (295) · Genetic Diseases, Inborn (290) · Lysosomal Storage Diseases (269) · Mucopolysaccharidoses (254) · Nervous System Diseases (123) · Neurologic Manifestations (102) · Pathological Conditions, Signs and Symptoms (95) · Genetic Diseases, X-Linked (87)
Iduronidase (18) · idursulfase (15) · GALNS protein, human (11) · Stem Cell Transplantation (7) · Alemtuzumab (6) · infigratinib (6) · vestronidase alfa (6) · Chondroitinases and Chondroitin Lyases (4) · Cyclophosphamide (4) · Enzyme Replacement Therapy (4) · Galns protein, mouse (4) · Melphalan (4) · N-Acetylgalactosamine-4-Sulfatase (4) · valanafusp alpha (4) · Busulfan (3)
Shire (20) · BioMarin Pharmaceutical (18) · Masonic Cancer Center, University of Minnesota (12) · JCR Pharmaceuticals Co., Ltd. (11) · Genzyme, a Sanofi Company (10) · Ultragenyx Pharmaceutical Inc (10) · QED Therapeutics, a BridgeBio company (8) · REGENXBIO Inc. (8) · CENTOGENE GmbH Rostock (7) · Takeda (6) · Alexion Pharmaceuticals, Inc. (5) · Allievex Corporation (5) · ArmaGen, Inc (5) · Denali Therapeutics Inc. (5) · Green Cross Corporation (5)
United States (198) · United Kingdom (72) · France (46) · Germany (44) · Canada (38) · Spain (38) · Brazil (35) · Italy (29) · Australia (23) · Netherlands (21) · Turkey (Türkiye) (20) · Portugal (17) · Argentina (15) · Japan (13) · Taiwan (12)
Paul Orchard, MD (7) · Peter Bauer, Prof. (5) · Celeste Decker, MD (4) · Debra Lounsbury (3) · Joanne Kurtzberg, MD (3) · Patrice Rioux, MD PhD (3) · Paul Harmatz, MD (3) · Adeline Vanderver, MD (2) · Arndt Rolfs, Prof. Dr. (2)
PMID 33874971, PMID 30467742, PMID 35709957, PMID 37415957, PMID 21586746, PMID 22547151, PMID 25421091, PMID 29526614, PMID 29976218, PMID 32063397, PMID 32884151, PMID 34353864, PMID 35342457, PMID 35835061, PMID 35961250, PMID 36299240, PMID 37915038, PMID 11310367, PMID 11820919, PMID 15324318, PMID 18412124, PMID 19117887, PMID 19948615, PMID 20924685, PMID 21150784, PMID 21419704, PMID 21596994, PMID 21637107, PMID 21877895, PMID 22210669, PMID 22393902, PMID 22543891, PMID 22867884, PMID 22929184, PMID 22974573, PMID 23497636, PMID 23510062, PMID 23666771, PMID 23837440, PMID 24108527, PMID 24296492, PMID 24368159, PMID 24388732, PMID 24582005, PMID 24764221, PMID 25178307, PMID 25214650, PMID 25323764, PMID 25487082, PMID 25541100
U54NS065768 (7), 2019 (5), 2023 (5), 2015 (4), 2014 (3), 1RC1DK086182 (1), 1UB1NS122644-01A1 (1), 2004 (1), 2009 (1), 2016 (1), 2017 (1), 2018 (1), R01DK067071 (1), R01DK079866 (1), R01HD073292 (1)