ADRENOLEUKODYSTROPHY
Clinical trials for ADRENOLEUKODYSTROPHY explained in plain language.
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Experimental cell shot aims to boost brain repair in kids with rare metabolic diseases
Disease control Recruiting nowThis early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…
Matched conditions: ADRENOLEUKODYSTROPHY
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Jun 26, 2026 14:27 UTC
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New registry aims to unlock secrets of rare brain disease ALD
Knowledge-focused Recruiting nowThis study is a national registry for people with adrenoleukodystrophy (ALD), a rare genetic disorder. Researchers will collect medical records and survey data from up to 1,000 patients and their families over time to understand how the disease progresses. The goal is to find bet…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:31 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC
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Blood tests may unlock secrets of rare brain disorders
Knowledge-focused Recruiting nowThis study collects blood samples from 100 people with rare brain diseases like adrenoleukodystrophy and metachromatic leukodystrophy. Researchers will study immune cells called macrophages to see if they affect how the disease progresses. No treatment is given; the goal is to le…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:51 UTC