Custom-Made genetic drug targets rare brain disorder in one patient
NCT ID NCT06816498
First seen Jul 13, 2026 · Last updated Jul 14, 2026 · Updated 1 time
Summary
This study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with autosomal dominant leukodystrophy (ADLD), a rare genetic brain disease. The treatment aims to slow or stop the disease by targeting the underlying genetic mutation. The participant will be followed for two years to measure changes in gait, neurological function, and brain structure.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- personalized antisense oligonucleotide (nL-LMNB1-001)
- What this could lead to
- If successful, this approach could slow or halt the progression of a rare, debilitating neurological disease and pave the way for similar personalized treatments for other genetic disorders.
- What could go wrong
- This is a very early, single-participant study, so results may not apply to others. The treatment is experimental and carries unknown risks, including potential side effects from the antisense oligonucleotide.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
1 person
The number who actually took part.
- Started
-
Mar 2025
- Expected to finish
-
Mar 2027
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
51 to 51 years
- Sex
-
Male participants only
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Informed consent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s). * Autosomal dominant adult-onset leukodystrophy (ADLD) caused by an LMNB1 duplication mutation * Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records. * Willingness to follow contraceptive guidance during the intervention period and for at least 40 weeks after the last dose of study intervention Exclusion Criteria: * Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures
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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
Locations
-
Mayo Clinic
Rochester, Minnesota, 55905, United States