NCT07197268View on ClinicalTrials.gov

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

1

Participants

Timeline

Start Date

May 19, 2025

Primary Completion Date

May 31, 2027

Study Completion Date

May 31, 2027

Conditions
Bainbridge-Ropers Syndrome
Interventions
DRUG

nL-ASXL3-001

Personalized antisense oligonucleotide

Trial Locations (1)

27599

University of North Carolina Chapel Hill, Chapel Hill

All Listed Sponsors
collaborator

University of North Carolina, Chapel Hill

OTHER

lead

n-Lorem Foundation

OTHER

NCT07197268 - Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation | Biotech Hunter | Biotech Hunter