40 completed, 15 active not recruiting, 9 terminated, 4 recruiting. 31 Phase 2, 20 Phase 1, 19 Phase 3, 4 Phase 4.
Follow Mucopolysaccharidosis II: new trials by email Literature Review of Mucopolysaccharidosis II trials Research Report on Mucopolysaccharidosis II
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 |
|---|---|---|---|---|---|---|
| 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 |
| Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment Idursulfase-IT, Elaprase - Hunter Syndrome | Phase 2 / Phase 3 | Active not recruiting | Takeda | 6 | 2024-03-05 | 2025-11-06 |
| An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 tividenofusp alfa - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Enrolling by invitation | Denali Therapeutics Inc. | 99 | 2023-09-20 | 2026-06-11 |
| Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII - Mucopolysaccharidosis II | Phase 1 / Phase 2 | Active not recruiting | University of Manchester | 5 | 2023-06-01 | 2026-03-27 |
| A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers verenafusp alfa 6 mg/kg, verenafusp alfa 9 mg/kg, verenafusp alfa 12 mg/kg - Mucopolysaccharidosis Type II, Metabolic Diseases | Phase 1 | Completed | AO GENERIUM | 18 | 2023-04-06 | 2024-07-03 |
| A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II) ELAPRASE, Rituximab, Methotrexate - Mucopolysaccharidosis (MPS), Hunter Syndrome | Phase 4 | Withdrawn | Takeda | 2023-02-28 | 2025-12-09 | |
| An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects JR-141 - Mucopolysaccharidosis II | Phase 3 | Enrolling by invitation | JCR Pharmaceuticals Co., Ltd. | 80 | 2023-02-22 | 2026-01-21 |
| A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ GC1123 - Mucopolysaccharidosis II, Hunter Syndrome | Phase 1 | Active not recruiting | GC Biopharma Corp | 12 | 2022-09-20 | 2024-10-15 |
| Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II Genetic HMI-203 - Mucopolysaccharidosis II | Phase 1 | Withdrawn | Homology Medicines, Inc | 2022-09-08 | 2023-08-28 | |
| 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 | |
| A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) tividenofusp alfa, idursulfase - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Active not recruiting | Denali Therapeutics Inc. | 65 | 2022-07-21 | 2026-09-09 |
| 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 | |
| A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India Elaprase - Hunter Syndrome | Phase 4 | Completed | Takeda | 5 | 2022-04-21 | 2025-01-23 |
| MPS II Immunophenotyping MPS II, Mucopolysaccharidosis II | Terminated | Duke University | 6 | 2022-04-01 | 2024-09-19 | |
| Mucopolysaccharidosis Type II Observational Observational - Mucopolysaccharidosis II | Withdrawn | REGENXBIO Inc. | 2022-03-01 | 2022-10-10 | ||
| A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) JR-141, Idursulfase, JR-141 or Idursulfase - Mucopolysaccharidosis II | Phase 3 | Active not recruiting | JCR Pharmaceuticals Co., Ltd. | 86 | 2022-02-14 | 2026-10-02 |
| A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg - Mucopolysaccharidosis Type II, Metabolic Diseases | Phase 2 / Phase 3 | Active not recruiting | AO GENERIUM | 32 | 2021-11-30 | 2026-09-21 |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program Confirmatory Testing - Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2 | Enrolling by invitation | Albert Einstein College of Medicine | 100000 | 2021-05-10 | 2026-07-28 | |
| Long-term Follow-Up for RGX-121 Long-term Follow-Up - Mucopolysaccharidosis II | Active not recruiting | REGENXBIO Inc. | 27 | 2021-03-14 | 2026-08-19 | |
| RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) RGX-121 - Mucopolysaccharidosis Type II (MPS II) | Phase 1 / Phase 2 | Completed | REGENXBIO Inc. | 6 | 2021-03-11 | 2025-01-28 |
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Sudden Infant Death (33) · Mucopolysaccharidosis I (20) · Lysosomal Storage Diseases (15) · Mucopolysaccharidoses (15) · Mucopolysaccharidosis VI (15) · Leukodystrophy, Metachromatic (9) · Leukodystrophy, Globoid Cell (8) · Mucopolysaccharidosis III (8) · Mucopolysaccharidosis VII (8) · Mucopolysaccharidosis IV (7) · Adrenoleukodystrophy (6) · alpha-Mannosidosis (6) · Metabolism, Inborn Errors (5) · Gaucher Disease (4)
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idursulfase (14) · Alemtuzumab (3) · Stem Cell Transplantation (3) · Adalimumab (2) · Hydroxyurea (2) · Melphalan (2) · Thiotepa (2) · fludarabine (2) · Busulfan (1) · Cyclophosphamide (1) · Cyclosporine (1) · Growth Hormone (1) · Human Growth Hormone (1) · Iduronate Sulfatase (1) · Immunoglobulins, Intravenous (1)
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PMID 35961250, PMID 37915038, PMID 21150784, PMID 22867884, PMID 22929184, PMID 22974573, PMID 23497636, PMID 23837440, PMID 24296492, PMID 24368159, PMID 25541100, PMID 25902842, PMID 26095521, PMID 26303610, PMID 26873528, PMID 27114913, PMID 27114914, PMID 28243577, PMID 28983455, PMID 29751845, PMID 30528227, PMID 30595526, PMID 31315600, PMID 32634238, PMID 32750127, PMID 33008435, PMID 33874971, PMID 35012890, PMID 35142618, PMID 35588317, PMID 36027721, PMID 36299240, PMID 37974184, PMID 40598289, PMID 41467650, PMID 42421116
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