NCT06369974View on ClinicalTrials.gov

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

PHASE1/PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

1

Participants

Timeline

Start Date

June 30, 2024

Primary Completion Date

June 30, 2026

Study Completion Date

June 30, 2026

Conditions
Genetic Disease
Interventions
DRUG

Antisense oligonucleotide treatment (ASO)

Drug: nL-TUBB4-001; Personalized antisense oligonucleotide

All Listed Sponsors
collaborator

n-Lorem Foundation

OTHER

lead

Massachusetts General Hospital

OTHER

NCT06369974 - Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy | Biotech Hunter | Biotech Hunter