Disease · MONDO_0010674
Trials23
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 2 more are not shown, since the checks do not confirm them.
1 to 20 of 23
Download this page as JSON| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT00069641 | Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) | Phase 2/3 | 96 | 2015-05-13 | Mucopolysaccharidosis II | IDURSULFASE | Its record names the disease | ClinicalTrials.gov |
| NCT02412787 | Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 | Phase 2/3 | 56 | 2025-06-19 | Hunter Syndrome | IDURSULFASE | Its record names the disease | ClinicalTrials.gov |
| NCT00607386 | Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy | Phase 4 | 28 | 2013-11-07 | Hunter Syndrome, Mucopolysaccharidosis II, MPS II | IDURSULFASE | Its record names the disease | ClinicalTrials.gov |
| NCT05058391 | A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India | Phase 4 | 5 | 2025-01-23 | Hunter Syndrome | IDURSULFASE | Its record names the disease | ClinicalTrials.gov |
| NCT00748969 | Clinical Trial of Growth Hormone in MPS I, II, and VI | Phase 2/3 | 2 | 2014-11-24 | Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI | SOMATROPIN | Its record names the disease | ClinicalTrials.gov |
| NCT02437253 | Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI | Phase 1/2 | 2 | 2017-09-15 | Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI | ADALIMUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT06075537 | 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 | Phase 2/3 | 99 | Mucopolysaccharidosis II | TIVIDENOFUSP ALFA | Its record names the disease | ClinicalTrials.gov | |
| NCT04573023 | A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) | Phase 3 | 86 | Mucopolysaccharidosis II | PABINAFUSP ALFA | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT05594992 | An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects | Phase 3 | 80 | Mucopolysaccharidosis II | PABINAFUSP ALFA | Its record names the disease | ClinicalTrials.gov | |
| NCT05371613 | 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) | Phase 2/3 | 65 | Mucopolysaccharidosis II | TIVIDENOFUSP ALFA | Its record names the disease | ClinicalTrials.gov | |
| NCT04251026 | A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome | Phase 1/2 | 47 | Mucopolysaccharidosis II | TIVIDENOFUSP ALFA | Its record names the disease | ClinicalTrials.gov | |
| NCT03920540 | A Study of GC1111 in Hunter Syndrom Patients | Phase 3 | 32 | Hunter Syndrome | IDURSULFASE | Its record names the disease | ClinicalTrials.gov | |
| NCT01301898 | To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients | Phase 1/2 | 31 | Mucopolysaccharidosis II | IDURSULFASE | Its record names the disease | ClinicalTrials.gov | |
| NCT07344376 | An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) | Phase 3 | 30 | MPS II, Hunter Syndrome (MPS II), Hunter Syndrome, Hunterase, GC1111 | IDURSULFASE BETA | Its record names the disease | ClinicalTrials.gov | |
| NCT03568175 | A Study of JR-141 in Patients With Mucopolysaccharidosis II | Phase 2/3 | 28 | Mucopolysaccharidosis II | PABINAFUSP ALFA | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT04348136 | An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II | Phase 2/3 | 27 | Mucopolysaccharidosis II | PABINAFUSP ALFA | Its record names the disease | ClinicalTrials.gov | |
| NCT02663024 | Study of Idursulfase-beta (GC1111) in Hunter Syndrome | Phase 2 | 20 | Mucopolysaccharidosis II | IDURSULFASE | Its record names the disease | ClinicalTrials.gov | |
| NCT03708965 | An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II | Phase 2 | 19 | Mucopolysaccharidosis II | PABINAFUSP ALFA | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT03359213 | A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21) | Phase 2 | 18 | Mucopolysaccharidosis II | PABINAFUSP ALFA | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT06475404 | A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers | Phase 1 | 18 | Mucopolysaccharidosis Type II, Metabolic Diseases | VERENAFUSP ALFA | Its record names the disease | ClinicalTrials.gov |
diseases-2026-09-18-0.1.0
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0010674/trials