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Condition: Mps Mucopolysaccharidosis Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Mps Mucopolysaccharidosis

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Cardiac Structure and Function in MPS
Phase N/A · 3 location(s) · Children's Hospital of Orange County · NCT07361536An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Phase 1/Phase 2 · 3 location(s) · JCR Pharmaceuticals Co., Ltd. · NCT06488924Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT06749366Registry of Patients Diagnosed With Lysosomal Storage Diseases
Phase N/A · 1 location(s) · University of California, San Francisco · NCT05619900ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Phase 1 · 2 location(s) · Immusoft of CA, Inc. · NCT05682144UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
Phase N/A · 2 location(s) · Phoenix Nest · NCT05825131A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharido
Phase 1 · 1 location(s) · West China Hospital · NCT06519552Study of Cannabidiol in Sanfilippo Syndrome
Phase 2/Phase 3 · 1 location(s) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA · NCT06333041A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Phase 2/Phase 3 · 5 location(s) · AO GENERIUM · NCT05208281Effects of Bisphosphonates on OI-Related Hearing Loss
Phase 4 · 1 location(s) · Hospital for Special Surgery, New York · NCT04152551Effect of Chronic Feeding of Inulin And Methylcellulose on Colonic Fermentation
NA · 2 location(s) · University of Nottingham · NCT06551961Prospective Clinical Assessment Study in Children With Hypochondroplasia
Phase N/A · 25 location(s) · QED Therapeutics, a BridgeBio company · NCT06410976A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Ind
NA · 1 location(s) · Kiwi Health Inc · NCT07591584MPS (RaDiCo Cohort) (RaDiCo-MPS)
Phase N/A · 23 location(s) · Institut National de la Santé Et de la Recherche Médicale, F · NCT06036693Decoding the Genetic Landscape of Skeletal Diseases
Phase N/A · 1 location(s) · Karolinska Institutet · NCT05876416Assessment of the Impact of Intestinal Gas Emission Quality on the Postoperative Course After Abdominal Surgery
Phase N/A · 1 location(s) · University Hospital, Angers · NCT06675097Non-invasive Functional Assessment and Pathogenesis of Morquio A
Phase N/A · 1 location(s) · Nemours Children's Clinic · NCT05284006PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Phase 1 · 1 location(s) · University of California, San Francisco · NCT04532047Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Phase N/A · 1 location(s) · Paul Szabolcs · NCT04528355A Study to Evaluate the Effects of a Butyrate-Polyphenol Formulation on Gut Health and Associated Symptoms
NA · 1 location(s) · Supplement Formulators, Inc. · NCT07371975Data Collection of Patients With Rare Bone Diseases
Phase N/A · 1 location(s) · Luca Sangiorgi · NCT05247645Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Phase 2/Phase 3 · 5 location(s) · Ultragenyx Pharmaceutical Inc · NCT02716246A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult P
Phase 2/Phase 3 · 32 location(s) · Denali Therapeutics Inc. · NCT05371613Mucopolysaccharidosis VII Disease Monitoring Program
Phase N/A · 14 location(s) · Ultragenyx Pharmaceutical Inc · NCT03604835Study of Skeletal Disorders
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT05031507A Natural History Study in Children With a Type II Collagen Disorder With Short Stature
Phase N/A · 2 location(s) · Innoskel · NCT05408715Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Phase 1 · 5 location(s) · GC Biopharma Corp · NCT06567769An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambrox
Phase 2/Phase 3 · 1 location(s) · Ozlem Goker-Alpan · NCT06614894Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients
NA · 1 location(s) · University Hospital, Montpellier · NCT05858606
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.