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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.

TrialTitlePhaseParticipantsResults first postedConditionsDrugsLinked bySource
NCT00069641Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)Phase 2/3962015-05-13Mucopolysaccharidosis IIIDURSULFASEIts record names the diseaseClinicalTrials.gov
NCT02412787Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094Phase 2/3562025-06-19Hunter SyndromeIDURSULFASEIts record names the diseaseClinicalTrials.gov
NCT00607386Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase TherapyPhase 4282013-11-07Hunter Syndrome, Mucopolysaccharidosis II, MPS IIIDURSULFASEIts record names the diseaseClinicalTrials.gov
NCT05058391A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in IndiaPhase 452025-01-23Hunter SyndromeIDURSULFASEIts record names the diseaseClinicalTrials.gov
NCT00748969Clinical Trial of Growth Hormone in MPS I, II, and VIPhase 2/322014-11-24Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VISOMATROPINIts record names the diseaseClinicalTrials.gov
NCT02437253Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VIPhase 1/222017-09-15Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VIADALIMUMABIts record names the diseaseClinicalTrials.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 2/399Mucopolysaccharidosis IITIVIDENOFUSP ALFAIts record names the diseaseClinicalTrials.gov
NCT04573023A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)Phase 386Mucopolysaccharidosis IIPABINAFUSP ALFAOpen Targets' reading of its recordClinicalTrials.gov
NCT05594992An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) SubjectsPhase 380Mucopolysaccharidosis IIPABINAFUSP ALFAIts record names the diseaseClinicalTrials.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 2/365Mucopolysaccharidosis IITIVIDENOFUSP ALFAIts record names the diseaseClinicalTrials.gov
NCT04251026A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter SyndromePhase 1/247Mucopolysaccharidosis IITIVIDENOFUSP ALFAIts record names the diseaseClinicalTrials.gov
NCT03920540A Study of GC1111 in Hunter Syndrom PatientsPhase 332Hunter SyndromeIDURSULFASEIts record names the diseaseClinicalTrials.gov
NCT01301898To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome PatientsPhase 1/231Mucopolysaccharidosis IIIDURSULFASEIts record names the diseaseClinicalTrials.gov
NCT07344376An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)Phase 330MPS II, Hunter Syndrome (MPS II), Hunter Syndrome, Hunterase, GC1111IDURSULFASE BETAIts record names the diseaseClinicalTrials.gov
NCT03568175A Study of JR-141 in Patients With Mucopolysaccharidosis IIPhase 2/328Mucopolysaccharidosis IIPABINAFUSP ALFAOpen Targets' reading of its recordClinicalTrials.gov
NCT04348136An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type IIPhase 2/327Mucopolysaccharidosis IIPABINAFUSP ALFAIts record names the diseaseClinicalTrials.gov
NCT02663024Study of Idursulfase-beta (GC1111) in Hunter SyndromePhase 220Mucopolysaccharidosis IIIDURSULFASEIts record names the diseaseClinicalTrials.gov
NCT03708965An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis IIPhase 219Mucopolysaccharidosis IIPABINAFUSP ALFAOpen Targets' reading of its recordClinicalTrials.gov
NCT03359213A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)Phase 218Mucopolysaccharidosis IIPABINAFUSP ALFAOpen Targets' reading of its recordClinicalTrials.gov
NCT06475404A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersPhase 118Mucopolysaccharidosis Type II, Metabolic DiseasesVERENAFUSP ALFAIts record names the diseaseClinicalTrials.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.

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