Browse recruiting and upcoming clinical trials for metachromatic leukodystrophy.
Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026
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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 inherited metabolic brain diseases and leukodystrophies, such as adrenoleukodystrophy, Batten disease, Krabbe, MLD, MPS II, Tay‑Sachs.
What is being studied AITests intrathecal DUOC-01 given after umbilical cord blood transplant for safety.
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 AIChildren and people with X‑ALD (AMN/CALD), metachromatic leukodystrophy, ALSP, presymptomatic ABCD1/CSF1R carriers, or healthy controls.
What is being studied AICollects a single blood sample to study macrophage activation, metabolism and gene expression.
Who can join AIAdults (≥18 years) with ultra-rare inherited metabolic or degenerative neurological diseases, or with undiagnosed neurological disease suspected to be inherited.
What is being studied AICollects clinical, lab and instrumental data over time for ultra-rare neurological diseases
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 of any age with a suspected or confirmed leukodystrophy or related white-matter disorder, and healthy people without such disorders.
What is being studied AICollects clinical data and biological samples to build a leukodystrophy biorepository and study natural history.