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Clinical trial

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

NCT07222371 is registered in ClinicalTrials.gov with University of California, San Diego as lead sponsor.

NCT NCT07222371
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy
Lead sponsor
University of California, San Diego
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
September 19, 2025
Completion date
September 30, 2027
Enrollment
1 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

genetic disease, tubb4a-related leukodystrophy