Browse recruiting and upcoming clinical trials for Hunter syndrome.
Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026
About this listHide
ClinicalTrials.gov is the complete and most up-to-date source. Trials may be missing, and trial information may have changed since our last check. Always verify important information in the official registry.
Who can join AIPeople with certain inborn metabolic brain diseases and leukodystrophies (e.g., adrenoleukodystrophy, Batten disease, Krabbe, metachromatic leukodystrophy, and others).
What is being studied AITests safety and feasibility of intrathecal DUOC-01 given after umbilical cord blood transplant.
Who can join AIPeople aged 2 months–55 years with non‑malignant disorders treatable by hematopoietic stem‑cell transplant (examples: immune, metabolic, hereditary anemias, inflammatory conditions).
What is being studied AITests reduced-intensity conditioning before stem cell transplant vs standard myeloablative conditioning.
Who can join AIPeople with mucopolysaccharidosis II (MPS II), including neuronopathic (nMPS II) and non‑neuronopathic (nnMPS II), who completed specified parent studies DNLI-E-0002 or DNLI-E-0007.
What is being studied AITests long-term safety, tolerability, and effects of tividenofusp alfa (DNL310) in MPS II.
Who can join AIPeople with mucopolysaccharidosis type II (Hunter syndrome) who completed the parent JR-141 study and are judged by the investigator to have no safety concerns.
What is being studied AITests long-term safety and effects of weekly IV JR-141 on brain and body measures.
Who can join AIPeople aged 2 months–60 years with non-malignant disorders (e.g., primary immunodeficiency, bone marrow failure, inherited metabolic disorders, hereditary anemias, inflammatory conditions) undergoing UCB, bone marrow, or peripheral blood stem cell transplant with a reduced-intensity conditioning regimen.
What is being studied AICollects clinical outcomes after alemtuzumab-based reduced-intensity conditioning stem cell transplants.
Who can join AIPeople with rare genetic neurodegenerative disorders (for example MLD, Krabbe disease, ALD, Batten disease, Gaucher disease, Niemann‑Pick disease, and others).
What is being studied AIRegular clinical evaluations, developmental testing, MRI, and collection of blood, CSF, and urine biomarkers.
Who can join AIPeople (prenatally or postnatally diagnosed) with Mucopolysaccharidosis I, II, IV A, VI, VII; infantile-onset Pompe; neuronopathic Gaucher; or Wolman disease, ages 0–64.
What is being studied AICollects clinical and laboratory data to describe natural history and prenatal outcomes of lysosomal diseases.