drugset / Trial / NCT06415344
Long-term Extension of GTX-102 in Angelman Syndrome
NaSingle-groupOpen-labelTreatment
Summary
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Timeline
- Start
- 2024-07-31
- Primary completion
- 2029-02
- Completion
- 2029-02
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | GTX-102 | Antisense oligonucleotide | — | Intrathecal |