Adrenoleukodystrophy
MONDO:0018544A peroxisomal disorder resulting in cerebral demyelination, axonal dysfunction in the spinal cord leading to spastic paraplegia, adrenal insufficiency and in some cases testicular insufficiency.
Also known as: ABCD1 deficiency, ALD, Bronze-Schilder disease, Siemerling-Creutzfeldt disease, X-ALD, X-Linked Adrenoleukodystrophy, X-linked ALD, X-linked adrenoleukodystrophy
20 clinical trials for this condition and its sub-types.
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Broader categories
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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: PHASE3 • 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 TerminatedThis 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: PHASE1 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Can a common MS drug help people with a rare nerve disease walk better?
Disease control OngoingThis clinical trial is testing whether dimethyl fumarate, a drug already used for multiple sclerosis, can improve balance and walking in adults with adrenomyeloneuropathy (AMN), a rare nerve disease. Forty participants will take either the drug or a placebo daily for 36 months, w…
Phase: PHASE2, PHASE3 • Sponsor: Pujol, Aurora, M.D. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Restless leg drug tested for rare disease in women
Symptom relief OngoingThis study looks at whether pramipexole, a drug used for restless leg syndrome, can ease leg symptoms in women with X-linked adrenoleukodystrophy (ALD). About 24 women will take the drug or a placebo for two months. The goal is to see if it improves sleep, walking, and quality of…
Phase: PHASE4 • Sponsor: Massachusetts General Hospital • Aim: Symptom relief
Last updated Jul 03, 2026 00:00 UTC