Custom-Made drug targets rare genetic brain disorder in Single-Child trial

NCT ID NCT06369974

ENROLLING_BY_INVITATION Disease control Sponsor: Massachusetts General Hospital Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

hereditary disease TUBB4A-related neurologic disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GENETIC DISEASE

Contacts and locations

Locations

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

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