ADRENOLEUKODYSTROPHY
Clinical trials for ADRENOLEUKODYSTROPHY explained in plain language.
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New cell booster may help cord blood transplants reach the brain in kids with rare diseases
Disease control Recruiting nowThis early-stage study tests whether adding special cells (DUOC-01) to a standard cord blood transplant is safe for children with inherited metabolic diseases that damage the brain. About 40 participants, from 1 week to 21 years old, will receive the cells via a spinal injection.…
Matched conditions: ADRENOLEUKODYSTROPHY
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated May 17, 2026 02:58 UTC
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New ALD registry aims to unlock secrets of rare disease
Knowledge-focused Recruiting nowThis study is building a national registry of people with adrenoleukodystrophy (ALD) and their family members. Researchers will collect medical records and survey data every six months to understand how the disease progresses and to find biological markers. No treatment is given;…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Knowledge-focused
Last updated May 15, 2026 11:56 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples from up to 12,000 people with leukodystrophies—rare brain disorders affecting white matter. Researchers will use this data to find new genetic causes, develop biomarkers, and better understand how these diseases progr…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 13, 2026 16:02 UTC
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Blood tests could unlock secrets of rare brain disorders
Knowledge-focused Recruiting nowThis study looks at how immune cells called macrophages behave in people with rare brain diseases like adrenoleukodystrophy and metachromatic leukodystrophy. Researchers will collect one blood sample from about 100 participants during a regular check-up. The goal is to learn if s…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 12, 2026 13:40 UTC