NCT07215416View on ClinicalTrials.gov

Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

PHASE1/PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

November 30, 2025

Primary Completion Date

December 31, 2028

Study Completion Date

December 31, 2035

Conditions
Ataxia Telangiectasia
Interventions
DRUG

Antisense oligonucleotide targeting the ATM gene

Atipeksen is a fully modified PS-2'MOE splice-switching antisense oligonucleotide that is designed to restore normal splicing patterns in patients with the ATM c.7865C\>T mutation.

All Listed Sponsors
lead

Timothy Yu

OTHER

NCT07215416 - Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T) | Biotech Hunter | Biotech Hunter