Trial · NCT03461289
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
NCT03461289, Phase 3, Completed, Novartis Gene Therapies; results first posted 2021-04-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1. Trials, record NCT03461289. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03461289
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
- NCT number
- NCT03461289
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 33 participants, actual
- Lead sponsor
- Novartis Gene Therapies
- Conditions
- SMA
- Interventions
- Onasemnogene Abeparvovec-xioi
- First posted
- 2018-03-12
- Last updated
- 2026-01-26
- Other registries' IDs
- eudract:2017-000266-29
- Results first posted
- 2021-04-02
- Limitations and caveats
A comparison of the results from this study to the results from the natural history observational study (Pediatric Neuromuscular Clinical Research Network (PNCR), Finkel et al, 2014) are included in the Novartis Clinical Trial Results, as a historical control. These full results are available via this link: https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17805.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds | From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months) | Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight with head erect for at least 10 seconds; participant does not use arms or hands to balance body or support position. |
| Secondary | Event-free Survival at 14 Months of Age | Up to 14 months of age | Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds | From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months) | The analysis population was the Intent-to-Treat (ITT) population which consisted of symptomatic participants with bi-allelic deletion of SMN1 (exon 7/8 common homozygous deletions) and 2 copies of SMN2 without the known gene modifier mutation (c.859G\>C) who received an IV infusion of onasemnogene abeparvovec-xioi at less than 180 days of age. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Event-free Survival at 14 Months of Age | Up to 14 months of age | The analysis population was the ITT population which consisted of symptomatic participants with bi-allelic deletion of SMN1 (exon 7/8 common homozygous deletions) and 2 copies of SMN2 without the known gene modifier mutation (c.859G\>C) who received an IV infusion of onasemnogene abeparvovec-xioi at less than 180 days of age. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds | Onasemnogene Abeparvovec-xioi | 14 | Participants | ||
| Event-free Survival at 14 Months of Age | Onasemnogene Abeparvovec-xioi | 31 | Participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Onasemnogene Abeparvovec-xioi | 33 | |
| Overall Study | COMPLETED | Onasemnogene Abeparvovec-xioi | 32 | |
| Overall Study | NOT COMPLETED | Onasemnogene Abeparvovec-xioi | 1 |
Source ClinicalTrials.gov
Adverse events148
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hypertension | Vascular disorders | Onasemnogene Abeparvovec-xioi | 4 | 33 | 4 |
| other | Hypotension | Vascular disorders | Onasemnogene Abeparvovec-xioi | 1 | 33 | 2 |
| other | Drug hypersensitivity | Immune system disorders | Onasemnogene Abeparvovec-xioi | 1 | 33 | 1 |
| other | Pyrexia | General disorders | Onasemnogene Abeparvovec-xioi | 20 | 33 | 29 |
| other | Crying | General disorders | Onasemnogene Abeparvovec-xioi | 1 | 33 | 2 |
| other | Extravasation | General disorders | Onasemnogene Abeparvovec-xioi | 1 | 33 | 1 |
| other | Generalised oedema | General disorders | Onasemnogene Abeparvovec-xioi | 1 | 33 | 1 |
| other | Hypothermia | General disorders | Onasemnogene Abeparvovec-xioi | 1 | 33 | 1 |
| other | Necrosis | General disorders | Onasemnogene Abeparvovec-xioi | 1 | 33 | 1 |
| other | Irritability | Psychiatric disorders | Onasemnogene Abeparvovec-xioi | 2 | 33 | 2 |
1 to 10 of 148Source ClinicalTrials.gov
See all 148 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Onasemnogene Abeparvovec-xioi | 1 | 33 |
| Total, other adverse events | Onasemnogene Abeparvovec-xioi | 32 | 33 |
| Total, serious adverse events | Onasemnogene Abeparvovec-xioi | 19 | 33 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03461289
GET https://conductscience.org/api/trials/v1/NCT03461289/events?page=2