Onasemnogene abeparvovec clinical trials
Also written Zolgensma and AVXS-101.
How each study uses onasemnogene abeparvovec 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
- 24 May 2019 · Zolgensma · BLA 125694 (FDA, Purple Book)
- European Union
- 18 May 2020 · Zolgensma · EMEA/H/C/004750 (EMA)
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
How studies write it
Studies that test it under each name
Studies by the year they started
8 studies that test it; most in 2017 (2)
| Year | Studies |
|---|---|
| 2014 | 1 |
| 2017 | 2 |
| 2018 | 2 |
| 2019 | 1 |
| 2020 | 1 |
| 2022 | 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 8 · Other 0
Studies
Open first, then newest update. How each study uses onasemnogene abeparvovec 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 · 8
- Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioiActive, not recruitingPhase 3Novartis Gene TherapiesNCT04042025Written as Onasemnogene Abeparvovec-xioi
- Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical TrialsActive, not recruitingPhase 3Novartis PharmaceuticalsNCT05335876Written as onasemnogene abeparvovec
- Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1CompletedPhase 3Novartis Gene TherapiesNCT03461289Written as Onasemnogene Abeparvovec-xioi
- Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2CompletedPhase 3Novartis Gene TherapiesNCT03505099Written as onasemnogene abeparvovec-xioi
- Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1CompletedPhase 3Novartis Gene TherapiesNCT03306277Written as Onasemnogene Abeparvovec-xioi
- Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 CopiesCompletedPhase 3Novartis Gene TherapiesNCT03837184Written as Onasemnogene Abeparvovec-xioi
- Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular AtrophyTerminatedPhase 1Novartis Gene TherapiesNCT03381729Written as Onasemnogene Abeparvovec-xioi
- Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1CompletedPhase 1Novartis Gene TherapiesNCT02122952Written as AVXS-101
Codes
What other systems call onasemnogene abeparvovec · ClinicalTrials.gg INT001434
- MeSH (NLM) C000710948onasemnogene abeparvovecConfirmed ClinicalTrials.gov, NLM
- UNII (FDA) MLU3LU3EVVONASEMNOGENE ABEPARVOVECConfirmed FDA, NLM
- RxNorm 2170226One source FDA
- NCI Thesaurus C174715Onasemnogene AbeparvovecOne source FDA
| System | Code | Name there | Checked by |
|---|---|---|---|
| MeSH (NLM) | C000710948 | onasemnogene abeparvovec | Confirmed ClinicalTrials.gov, NLM |
| UNII (FDA) | MLU3LU3EVV | ONASEMNOGENE ABEPARVOVEC | Confirmed FDA, NLM |
| RxNorm | 2170226 | — | One source FDA |
| NCI Thesaurus | C174715 | Onasemnogene Abeparvovec | One source FDA |
“Confirmed” means two independent publishers link the code to this drug; “one source”, one does and its name agrees.