X-linked cerebral adrenoleukodystrophy
MONDO:0010247A peroxisomal disease characterized by severe inflammatory demyelination in the brain, and often associated with adrenal insufficiency.
Also known as: X-linked cerebral adrenoleukodystrophy, ALD, ALD childhood cerebral form, Addison disease and cerebral sclerosis, Siemerling-Creutzfeldt disease, adrenoleukodystrophy, adrenoleukodystrophy X-linked cerebral form, adrenoleukodystrophy childhood cerebral form
7 clinical trials for this condition and its sub-types, 4 tagged with X-linked cerebral adrenoleukodystrophy itself.
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One-Time gene therapy for fatal childhood brain disease: does it last?
Cure OngoingResearchers are following 64 people with cerebral adrenoleukodystrophy (CALD) for 15 years after they received a one-time gene therapy called eli-cel in earlier studies. The therapy uses the patient's own stem cells, modified to carry a working copy of the gene that is faulty in …
Sponsor: Genetix Biotherapeutics Inc. • Aim: Cure
Last updated Sep 16, 2026 00:00 UTC
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Hope for rare brain disease: new drug trial aims to stall deadly decline
Disease control OngoingThis Phase 3 study tests whether the drug leriglitazone can help adult men with cerebral adrenoleukodystrophy (cALD), a rare and serious brain disease. The main goal is to see if the drug delays death or the need for permanent breathing support compared to a placebo. About 40 men…
Phase 3 • Sponsor: Minoryx Therapeutics, S.L. • Aim: Disease control
Last updated Jul 31, 2026 00:00 UTC
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Stem cell infusion aims to buy time for kids with fatal brain disease
Disease control PausedThis early-stage trial tests whether a single infusion of donated mesenchymal stem cells is safe for children with cerebral adrenoleukodystrophy (cALD), a rare and life-threatening brain disease. The stem cells are given as a temporary bridge to buy time before a more definitive …
Phase 1 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC