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
23 clinical trials for this condition and its sub-types, 19 tagged with Adrenoleukodystrophy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Part of
Sub-types of Adrenoleukodystrophy
-
Adrenomyeloneuropathy 7 trials
-
Isolated adrenal insufficiency 0 trials
-
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
-
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
-
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
-
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 4 • Sponsor: Massachusetts General Hospital • Aim: Symptom relief
Last updated Jul 03, 2026 00:00 UTC