Apazunersen sodium clinical trials
Also written GTX-102.
How each study uses apazunersen sodium is sorted by a rule, right about nine times in ten. Readers can ask us to read every study one by one.Ask us to read each study
Approvals
The first approval found in each region, with its record
- United States
- Not in FDA's approval records
- European Union
- Not in EMA's list of EU-wide authorisations
From FDA's Drugs@FDA and Purple Book (through openFDA) and the European Medicines Agency, files of Sept 2026 and 27 Oct 2026. National approvals in Europe and other countries are not included. How this is worked out
Studies by the year they started
4 studies that test it; most in 2024 (2)
| Year | Studies |
|---|---|
| 2020 | 1 |
| 2024 | 2 |
| 2025 | 1 |
By phase
A phase 1/2 study counts in both.
What it is tested for
Each study’s main condition, where its own words name it, so a study counts once.
Who runs them
Industry 4 · Other 0
Studies
Open first, then newest update. How each study uses apazunersen sodium is read from its arms, and is right about nine times in ten. Ask us to read each study. These are the studies that list it as an intervention; a search of ClinicalTrials.gov also finds studies that mention it only in a description, such as earlier treatment.
Testing it · 4
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)TerminatedPhase 3Ultragenyx Pharmaceutical IncNCT06617429Written as GTX-102
- Long-term Extension of GTX-102 in Angelman SyndromeTerminatedPhase 3Ultragenyx Pharmaceutical IncNCT06415344Written as GTX-102
- A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)TerminatedPhase 2Ultragenyx Pharmaceutical IncNCT07157254Written as GTX-102
- A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeCompletedPhase 1/2Ultragenyx Pharmaceutical IncNCT04259281Written as GTX-102
What changed recently
From the registry's own updates since Oct 14, 2026.
Stopped3
- Long-term Extension of GTX-102 in Angelman SyndromeTerminatedNov 3, 2026NCT06415344
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)TerminatedNov 3, 2026NCT06617429
- A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)TerminatedNov 3, 2026NCT07157254
Codes
What other systems call apazunersen sodium · ClinicalTrials.gg INT016072
- UNII (FDA) B5PZX5263HAPAZUNERSEN SODIUMOne source FDA
- NCI Thesaurus C226349Apazunersen SodiumOne source FDA
| System | Code | Name there | Checked by |
|---|---|---|---|
| UNII (FDA) | B5PZX5263H | APAZUNERSEN SODIUM | One source FDA |
| NCI Thesaurus | C226349 | Apazunersen Sodium | One source FDA |
“Confirmed” means two independent publishers link the code to this drug; “one source”, one does and its name agrees.