Browse recruiting and upcoming clinical trials for alpha-mannosidosis.
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 and leukodystrophy brain diseases, such as adrenoleukodystrophy, Krabbe, metachromatic leukodystrophy, Tay‑Sachs, and Sanfilippo.
What is being studied AITests safety and feasibility of intrathecal DUOC-01 added to 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 AIChildren under 3 years with confirmed alpha-mannosidosis who started velmanase alfa (Lamzede) at least six weeks before their 3rd birthday.
What is being studied AIMeasures how velmanase alfa affects blood levels of the disease marker GlcNAc(Man)2.
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.