14 completed, 4 terminated, 3 active not recruiting, 2 withdrawn. 25 Phase 2, 16 Phase 1, 1 Phase 3.
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 |
|---|---|---|---|---|---|---|
| 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of Encapsulated Allogeneic MPS-1 Therapy SIG-005 (hIDUA Producing Spheres) - MPS I, MPS1-S | Phase 1 / Phase 2 | Withdrawn | Sigilon Therapeutics, Inc. | 2021-11-15 | 2023-04-11 | |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 |
| 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 |
| 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 |
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Thiotepa--single daily dose, Thiotepa--escalated dose - Bone Marrow Failure Syndrome, Thalassemia | Phase 1 / Phase 2 | Completed | University of Florida | 6 | 2018-06-15 | 2023-09-15 |
| Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium. - Mucopolysaccharidosis IH | Phase 1 / Phase 2 | Active not recruiting | Orchard Therapeutics | 8 | 2018-05-11 | 2026-07-16 |
| MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) MGTA-456 - Inherited Metabolic Disorders (IMD) | Phase 2 | Completed | Magenta Therapeutics, Inc. | 8 | 2018-02-09 | 2021-11-03 |
| S 81694 Plus Paclitaxel in Metastatic Breast Cancer Combination therapy (S81694 + paclitaxel) phase I, Paclitaxel, Combination therapy (S81694 + paclitaxel) phase II - Metastatic Breast Cancer, Metastatic Triple Negative Breast Cancer | Phase 1 / Phase 2 | Completed | Institut de Recherches Internationales Servier | 22 | 2018-01-04 | 2021-05-25 |
| Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Umbilical cord blood transplantation with HSC835 - Inherited Metabolic Disorders IMD | Phase 1 / Phase 2 | Withdrawn | Novartis Pharmaceuticals | 2017-10-10 | 2018-11-28 | |
| Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI Adalimumab Injection [Humira], Saline Solution for Injection - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Phase 1 / Phase 2 | Active not recruiting | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 14 | 2017-06-05 | 2026-02-02 |
| Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I SB-318 - MPS I | Phase 1 / Phase 2 | Terminated | Sangamo Therapeutics | 3 | 2017-05-24 | 2023-01-26 |
| Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I AGT-181 - Mucopolysaccharidosis I | Phase 1 / Phase 2 | Completed | ArmaGen, Inc | 19 | 2016-10-01 | 2023-03-15 |
| Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I AGT-181 - Mucopolysaccharidosis I | Phase 1 / Phase 2 | Completed | ArmaGen, Inc | 21 | 2015-10-01 | 2023-03-15 |
| Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI Adalimumab, Placebo - Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II | Phase 1 / Phase 2 | Completed | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 2 | 2015-05-01 | 2017-09-15 |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-01-07 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
| Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 46 | 2009-12-01 | 2018-02-05 |
| Clinical Trial of Growth Hormone in MPS I, II, and VI Somatropin (DNA origin) - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Phase 2 / Phase 3 | Terminated | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 2 | 2008-11-01 | 2018-09-05 |
| Immune Tolerance Study With Aldurazyme® (Laronidase) Laronidase, Cyclosporine A (CsA), Azathioprine (Aza) - Mucopolysaccharidosis I | Phase 1 / Phase 2 | Completed | Genzyme, a Sanofi Company | 7 | 2008-09-01 | 2014-07-02 |
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Leukodystrophy, Metachromatic (7) · Mucopolysaccharidosis II (7) · Mucopolysaccharidosis VI (7) · Leukodystrophy, Globoid Cell (6) · alpha-Mannosidosis (5) · Adrenoleukodystrophy (4) · Fucosidosis (4) · Mucopolysaccharidoses (4) · Anemia, Sickle Cell (3) · Aspartylglucosaminuria (3) · Granulomatous Disease, Chronic (3) · Mucopolysaccharidosis VII (3) · Osteopetrosis (3) · Peroxisomal Disorders (3)
Carbohydrate Metabolism, Inborn Errors (25) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (25) · Connective Tissue Diseases (25) · Lysosomal Storage Diseases (25) · Metabolic Diseases (25) · Mucinoses (25) · Nutritional and Metabolic Diseases (25) · Skin and Connective Tissue Diseases (25) · Genetic Diseases, Inborn (24) · Metabolism, Inborn Errors (23) · Mucopolysaccharidoses (21) · Nervous System Diseases (13) · Brain Diseases (10) · Brain Diseases, Metabolic (10) · Brain Diseases, Metabolic, Inborn (10)
Stem Cell Transplantation (6) · Alemtuzumab (3) · Busulfan (3) · Cyclophosphamide (3) · Iduronidase (3) · Adalimumab (2) · Bone Marrow Transplantation (2) · Cyclosporine (2) · valanafusp alpha (2) · Azathioprine (1) · Clinical Coding (1) · Clinical Trials, Phase I as Topic (1) · Clinical Trials, Phase II as Topic (1) · Combined Modality Therapy (1) · Growth Hormone (1)
Masonic Cancer Center, University of Minnesota (5) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (3) · ArmaGen, Inc (2) · Genzyme, a Sanofi Company (2) · JCR Pharmaceuticals Co., Ltd. (2) · Children's Hospital Los Angeles (1) · Institut de Recherches Internationales Servier (1) · Magenta Therapeutics, Inc. (1) · Novartis Pharmaceuticals (1) · Orchard Therapeutics (1) · Paul Szabolcs (1) · REGENXBIO Inc. (1) · Sangamo Therapeutics (1) · Sigilon Therapeutics, Inc. (1) · Talaris Therapeutics Inc. (1)
United States (17) · Brazil (7) · Japan (3) · France (2) · Netherlands (2) · United Kingdom (2) · Belgium (1) · Germany (1) · Italy (1) · Russia (1)
Paul Orchard, MD (4) · Patrice P Rioux, MD PhD (2) · Biljana Horn, MD (1) · Hisham Abdel-Azim, MD (1) · Lynda E Polgreen, M.D. (1) · Lynda E Polgreen, MD, MS (1) · Lynda Polgreen, MD (1) · Paul Orchard, M.D. (1) · Paul Szabolcs, MD (1) · Suzanne T Ildstad, MD (1)
PMID 29976218, PMID 21586746, PMID 28119821, PMID 32278382, PMID 32634238, PMID 32750127, PMID 36299240, PMID 37624739, PMID 38204164
2017 (1)