39 completed, 14 terminated, 6 active not recruiting, 6 recruiting. 28 Phase 1, 25 Phase 2, 7 Phase 3, 4 Phase 4, 3 Not applicable.
Follow Mucopolysaccharidosis I: new trials by email Literature Review of Mucopolysaccharidosis I trials Research Report on Mucopolysaccharidosis I
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 Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I JWK008 Single intravenous infusion administration - Mucopolysaccharidosis Type I | Phase 1 | Recruiting | West China Hospital | 6 | 2024-07-22 | 2024-07-25 |
| A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT Experimental: OTL-203, Active Comparator: Allo-HSCT - MPS-IH (Hurler Syndrome) | Phase 3 | Active not recruiting | Orchard Therapeutics | 41 | 2023-12-11 | 2025-12-22 |
| Long Term Follow-Up for RGX-111 No Intervention - Mucopolysaccharidosis I | Enrolling by invitation | REGENXBIO Inc. | 21 | 2023-07-24 | 2026-04-29 | |
| ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I Autologous Plasmablasts (B cells) - Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS | Phase 1 | Recruiting | Immusoft of CA, Inc. | 11 | 2023-04-12 | 2026-07-17 |
| Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH. Laronidase therapy and a stem cell transplant - Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I | Active not recruiting | Masonic Cancer Center, University of Minnesota | 13 | 2022-11-17 | 2025-10-30 | |
| 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 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Registry of Patients Diagnosed With Lysosomal Storage Diseases There is no intervention - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Recruiting | University of California, San Francisco | 250 | 2022-05-31 | 2026-04-08 | |
| Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy SIG-005 (hIDUA Producing Spheres) - MPS I, MPS1-S | Phase 1 / Phase 2 | Withdrawn | Sigilon Therapeutics, Inc. | 2021-11-15 | 2023-04-11 | |
| An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I) JR-171 - Mucopolysaccharidosis I | Phase 1 / Phase 2 | Completed | JCR Pharmaceuticals Co., Ltd. | 14 | 2021-10-28 | 2025-08-28 |
| China Post-marketing Surveillance (PMS) Study of Aldurazyme® Laronidase - Mucopolysaccharidosis I | Phase 4 | Completed | Genzyme, a Sanofi Company | 12 | 2021-10-28 | 2025-09-17 |
| A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting Pompe Disease, Mucopolysaccharidosis Type I (MPS I) | Completed | Sanofi | 57 | 2021-10-14 | 2024-02-14 | |
| Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX SB-318, SB-913, SB-FIX - Hemophilia B, Mucopolysaccharidosis I | Terminated | Sangamo Therapeutics | 13 | 2020-11-03 | 2026-08-13 | |
| A Study of JR-171 in Patients With Mucopolysaccharidosis I JR-171 (lepunafusp alfa) - Mucopolysaccharidosis I | Phase 1 / Phase 2 | Completed | JCR Pharmaceuticals Co., Ltd. | 18 | 2020-09-01 | 2025-03-19 |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 | |
| A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456 Safety and efficacy assessments - Inherited Metabolic Disorders (IMD) | Terminated | Magenta Therapeutics, Inc. | 3 | 2019-05-21 | 2021-03-15 | |
| RGX-111 Gene Therapy in Patients With MPS I RGX-111 - Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome | Phase 1 / Phase 2 | Suspended | REGENXBIO Inc. | 21 | 2019-04-03 | 2026-03-12 |
| MRS to Determine Neuroinflammation and Oxidative Stress in MPS I Mucopolysaccharidosis Type I | Completed | University of Minnesota | 30 | 2018-11-01 | 2019-11-01 | |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2) Hepato-splenomegaly, Dysostosis Multiplex | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 |
Showing 20 of 74. Sign in to see all of them.
Mucopolysaccharidosis II (20) · Mucopolysaccharidosis VI (18) · Leukodystrophy, Metachromatic (15) · Mucopolysaccharidoses (15) · Leukodystrophy, Globoid Cell (13) · Metabolism, Inborn Errors (10) · Adrenoleukodystrophy (9) · Lysosomal Storage Diseases (8) · Mucopolysaccharidosis VII (8) · Sudden Infant Death (8) · Anemia, Diamond-Blackfan (7) · Anemia, Sickle Cell (7) · Fucosidosis (7) · Granulomatous Disease, Chronic (7)
Carbohydrate Metabolism, Inborn Errors (74) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (74) · Connective Tissue Diseases (74) · Metabolic Diseases (74) · Mucinoses (74) · Nutritional and Metabolic Diseases (74) · Skin and Connective Tissue Diseases (74) · Genetic Diseases, Inborn (71) · Lysosomal Storage Diseases (66) · Metabolism, Inborn Errors (64) · Mucopolysaccharidoses (59) · Nervous System Diseases (36) · Central Nervous System Diseases (26) · Genetic Diseases, X-Linked (26) · Neurobehavioral Manifestations (25)
Iduronidase (17) · Stem Cell Transplantation (6) · Alemtuzumab (4) · valanafusp alpha (4) · Busulfan (3) · Cyclophosphamide (3) · Adalimumab (2) · Bone Marrow Transplantation (2) · Cyclosporine (2) · Growth Hormone (2) · Hydroxyurea (2) · Injections (2) · Melphalan (2) · Thiotepa (2) · fludarabine (2)
Genzyme, a Sanofi Company (10) · Masonic Cancer Center, University of Minnesota (9) · ArmaGen, Inc (4) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (3) · Patricia I. Dickson, M.D. (3) · University of Minnesota (3) · CENTOGENE GmbH Rostock (2) · JCR Pharmaceuticals Co., Ltd. (2) · Magenta Therapeutics, Inc. (2) · Orchard Therapeutics (2) · Paul Szabolcs (2) · REGENXBIO Inc. (2) · Sangamo Therapeutics (2) · Aldagen (1) · Bellicum Pharmaceuticals (1)
United States (53) · Brazil (11) · Germany (7) · Italy (7) · Canada (6) · United Kingdom (6) · Japan (5) · Netherlands (5) · France (4) · Belgium (3) · Egypt (3) · India (3) · China (2) · Finland (2) · Russia (2)
Paul Orchard, MD (6) · Joanne Kurtzberg, MD (2) · Patrice P Rioux, MD PhD (2) · Patrice Rioux, MD PhD (2) · Patricia I Dickson, MD (2) · Paul Orchard, M.D. (2) · Paul Szabolcs, MD (2) · Peter Bauer, Prof. (2) · Agnes Chen, MD (1) · Ali Rabani, Professor (1) · Ashley Schneider (1) · Biljana Horn, MD (1)
PMID 29976218, PMID 33874971, PMID 19117887, PMID 21586746, PMID 22867884, PMID 22974573, PMID 24296492, PMID 24368159, PMID 25541100, PMID 25864295, PMID 26095521, PMID 26303610, PMID 26873528, PMID 27114913, PMID 27114914, PMID 28119821, PMID 28983455, PMID 29751845, PMID 31315600, PMID 31575939, PMID 32278382, PMID 32634238, PMID 32750127, PMID 33008435, PMID 35012890, PMID 35142618, PMID 36299240, PMID 37624739, PMID 38204164, PMID 40830667, PMID 41163043, PMID 41272769
U54NS065768 (5), 2017 (1)