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 26, 2026 05:42 UTC
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Blood samples shed light on rare brain disorders
Knowledge-focused Recruiting nowThis study looks at how immune cells called macrophages behave in people with certain rare brain diseases (leukodystrophies). Researchers will collect one blood sample from about 100 participants during a regular check-up. The goal is to learn how these cells might affect disease…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 26, 2026 05:49 UTC
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Massive leukodystrophy biobank aims to unlock secrets of rare brain diseases
Knowledge-focused Recruiting nowThis study is creating a large collection of medical data and biological samples from people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this information to discover new genetic causes, develop better diagnostic tools, and track…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 26, 2026 05:49 UTC
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New ALD registry aims to unlock secrets of rare disease
Knowledge-focused Recruiting nowThis study creates a national registry for people with adrenoleukodystrophy (ALD) and their families. Researchers will collect medical records and survey data every six months to understand how the disease progresses and to find biological markers. Up to 1,000 participants of any…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Knowledge-focused
Last updated May 26, 2026 05:39 UTC