Antisense oligonucleotide treatment (ASO)

BIOLOGICAL2 trials

Sponsors

Massachusetts General Hospital, Pirogov Russian National Research Medical University

Conditions

EpilepsyGNAO1Genetic DiseaseHyperkinesis

Phase 1

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

Enrolling by invitationNCT06369974

Massachusetts General HospitalGenetic Disease

Start: 2024-09-18End: 2026-06-01Target: 1Updated: 2026-03-02

Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.

RecruitingNCT07363603

Pirogov Russian National Research Medical UniversityEpilepsy, GNAO1, Hyperkinesis

Start: 2025-09-09End: 2026-12-31Target: 5Updated: 2026-01-23