Custom-Made drug targets rare genetic brain disorder in Single-Child trial
NCT ID NCT06369974
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a personalized drug called nL-TUBB4-001, designed specifically for one child with TUBB4A-related leukodystrophy, a rare genetic disease that damages the brain. The drug is an antisense oligonucleotide, which aims to correct the genetic error. The trial will monitor the child's development, movement, feeding, and safety over 24 months.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
nL-TUBB4-001 (personalized antisense oligonucleotide)
What this could lead to
If it works, this could point toward a treatment for TUBB4A-related leukodystrophy, a rare genetic brain disease.
What could go wrong
This is an extremely early trial with only one participant, so results may not apply to others. The treatment is experimental and may not improve symptoms or could cause side effects.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States