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Clinical trial

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

NCT06369974 is registered in ClinicalTrials.gov with Massachusetts General Hospital as lead sponsor.

NCT NCT06369974
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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
Massachusetts General Hospital
Phase
Phase 1/2
Overall status
Enrolling by invitation
Start date
September 18, 2024
Completion date
June 30, 2026
Enrollment
1 (estimated)
Sites
1

Conditions

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

genetic disease