52 completed, 18 recruiting, 10 terminated, 9 unknown. 23 Not applicable, 20 Phase 2, 8 Phase 1, 8 Phase 3, 5 Phase 4.
Follow Mucopolysaccharidosis IV: new trials by email Literature Review of Mucopolysaccharidosis IV trials Research Report on Mucopolysaccharidosis IV
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 |
| 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 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 |
| 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 |
| 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 |
| 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 |
| 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 | |
| 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 |
| Assessment of the Impact of Intestinal Gas Emission Quality on the Postoperative Course After Abdominal Surgery Postoperative Complication, Recovery, Physiological | Recruiting | University Hospital, Angers | 200 | 2025-01-31 | 2026-08-10 | |
| Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing Enchondromatosis | Recruiting | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 100 | 2025-01-27 | 2026-10-01 | |
| Effect of Abdominal Massage on Gastrointestinal Symptoms After Head and Neck Cancer Surgery Abdominal Massage - Abdominal Massage, Enteral Nutrition | Not applicable | Completed | Maltepe University | 70 | 2024-10-30 | 2026-10-26 |
| Effect of Chronic Feeding of Inulin And Methylcellulose on Colonic Fermentation methylcellulose, inulin, maltodextrin - Irritable Bowel Syndrome | Not applicable | Recruiting | University of Nottingham | 35 | 2024-09-30 | 2024-11-15 |
| CO2 Supplement for Treatment of Acute Mountain Sickness Breathing with CO2 during daytime and night at high altitude, Sleep and doing exercise test under inhalation of air - Acute Mountain Sickness, High Altitude | Not applicable | Completed | State Key Laboratory of Respiratory Disease | 46 | 2024-06-07 | 2024-12-18 |
| Prospective Clinical Assessment Study in Children With Hypochondroplasia Hypochondroplasia | Recruiting | QED Therapeutics, a BridgeBio company | 150 | 2024-06-05 | 2026-07-16 | |
| Sevoflurane Wash in Method Using Conventional Flows Versus Low Flows of 0.5L Sevoflurane - Inhalation; Gas | Completed | Royal Hospital, Oman | 48 | 2024-01-30 | 2024-08-27 | |
| Efficacy of BIS Monitoring in Deep Hypnotic State BIS - Hypnotic; Withdrawal State With Delirium, Neurological Disorder | Not applicable | Unknown | Universitas Indonesia | 50 | 2023-12-15 | 2023-12-20 |
| 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 |
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Dwarfism (12) · Mucopolysaccharidosis VI (12) · Lysosomal Storage Diseases (11) · Bone Diseases (10) · Constipation (10) · Diarrhea (10) · Genetic Diseases, Inborn (10) · Osteochondrodysplasias (10) · Abdominal Pain (9) · Musculoskeletal Diseases (9) · Respiratory Aspiration (8) · Achondroplasia (7) · Mucopolysaccharidoses (7) · Mucopolysaccharidosis II (7)
Carbohydrate Metabolism, Inborn Errors (101) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (101) · Connective Tissue Diseases (101) · Metabolic Diseases (101) · Mucinoses (101) · Nutritional and Metabolic Diseases (101) · Skin and Connective Tissue Diseases (101) · Metabolism, Inborn Errors (99) · Mucopolysaccharidoses (94) · Genetic Diseases, Inborn (91) · Lysosomal Storage Diseases (90) · Pathological Conditions, Signs and Symptoms (38) · Neurologic Manifestations (25) · Signs and Symptoms (22) · Bone Diseases, Developmental (19)
GALNS protein, human (11) · infigratinib (6) · Chondroitinases and Chondroitin Lyases (4) · Enzyme Replacement Therapy (4) · Galns protein, mouse (4) · Sevoflurane (3) · Sulfatases (3) · Butyrates (2) · Chondroitinsulfatases (2) · Inulin (2) · Probiotics (2) · Administration, Oral (1) · Anesthesia (1) · Bupivacaine (1) · Cathartics (1)
BioMarin Pharmaceutical (11) · QED Therapeutics, a BridgeBio company (8) · University of California, San Francisco (3) · CENTOGENE GmbH Rostock (2) · Emory University (2) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (2) · Nadia Ali, PhD (2) · Pfizer (2) · Regeneron Pharmaceuticals (2) · State Key Laboratory of Respiratory Disease (2) · Supplement Formulators, Inc. (2) · The Canadian College of Naturopathic Medicine (2) · University of Nottingham (2) · Acuity Polymers, Inc. (1) · Ain Shams University (1)
United States (56) · United Kingdom (22) · Canada (21) · France (19) · Spain (13) · Australia (12) · Italy (12) · Germany (9) · Norway (8) · Portugal (8) · Singapore (7) · Argentina (6) · Taiwan (6) · Denmark (5) · Belgium (4)
Celeste Decker, MD (3) · Debra Lounsbury (3) · Dugald Seely, ND (2) · Nadia Ali, Ph.D. (2) · Yuanming Luo, PhD (2) · Adam Shaywitz, MD PhD (1) · Andrea Superti-Furga (1) · Andrew Swick, Ph.D (1) · Andrew Swick, PhD (1) · Anthony Beir, MD (1) · Arndt Rolfs, Prof. Dr. (1) · Ashley Mateus, Ph.D. (1)
PMID 29526614, PMID 34353864, PMID 35342457, PMID 11820919, PMID 15324318, PMID 18412124, PMID 19948615, PMID 21596994, PMID 21637107, PMID 21877895, PMID 22393902, PMID 22867884, PMID 22974573, PMID 23510062, PMID 24368159, PMID 25323764, PMID 25487082, PMID 25541100, PMID 26095521, PMID 26252852, PMID 26303610, PMID 26331768, PMID 26779697, PMID 26873528, PMID 27114913, PMID 27114914, PMID 27955919, PMID 28374935, PMID 28535791, PMID 28657663, PMID 28969890, PMID 28983455, PMID 29159458, PMID 29248359, PMID 29751845, PMID 30046155, PMID 30177353, PMID 30337566, PMID 31315600, PMID 31719214, PMID 32102934, PMID 34127041, PMID 35012890, PMID 35142618, PMID 36194207, PMID 36799346, PMID 37955580, PMID 38300481, PMID 38685585, PMID 39315524
2019 (2), 1RC1DK086182 (1), R01DK067071 (1), R01DK079866 (1), R01HD073292 (1), R01HD102545 (1), R01HL173371 (1), U01HG006487 (1), U54NS065768 (1), UL1RR024150 (1)