Trial · NCT03306277
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
NCT03306277, Phase 3, Completed, Novartis Gene Therapies; results first posted 2020-07-16
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1. Trials, record NCT03306277. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03306277
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
- NCT number
- NCT03306277
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 22 participants, actual
- Lead sponsor
- Novartis Gene Therapies
- Conditions
- SMA - Spinal Muscular Atrophy, Gene Therapy
- Interventions
- Onasemnogene Abeparvovec-xioi
- First posted
- 2017-10-11
- Last updated
- 2026-01-26
- Other registries' IDs
- eudract:2020-000095-38
- Results first posted
- 2020-07-16
- Limitations and caveats
- The complete results are available via this link: https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17657
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Achievement of Independent Sitting for at Least 30 Seconds | Up to 18 months | Independent sitting is defined as sitting up straight with head erect for at least 30 seconds. This endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance, was the endpoint of event-free survival assessed. |
| Primary | Event-free Survival | 14 months | Survival is defined by the avoidance of combined endpoint of either death or permanent ventilation, which is defined by tracheostomy or by the requirement of ≥ 16 hours of respiratory assistance per day for ≥ 14 consecutive days in the absence of an acute reversible illness, excluding perioperative ventilation. Permanent ventilation is considered a surrogate for death. An acute reversible illness is defined as any condition other than SMA that results in increased medical intervention. The endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance was the survival endpoint assessed. |
| Secondary | Ability to Thrive | 18 months | Ability to thrive is defined as achieving all of the following at 18 months of age: * does not receive nutrition through mechanical support or other non-oral method * ability to tolerate thin liquids as demonstrated through a formal swallowing test * maintains weight This is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed. |
| Secondary | Ventilatory Support Independence | Up to 18 months | Ventilatory support independence is defined as requiring no daily ventilator support/usage at 18 months of age, excluding acute reversible illness and perioperative ventilation, through assessment of actual usage data captured from the device (Phillips Trilogy BiPAP device). This endpoint is derived solely from the Phillips Trilogy BiPAP device. This is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Achievement of Independent Sitting for at Least 30 Seconds | Up to 18 months | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Event-free Survival | 14 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Ability to Thrive | 18 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Ventilatory Support Independence | Up to 18 months | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Achievement of Independent Sitting for at Least 30 Seconds | Onasemnogene Abeparvovec-xioi | 13 | Participants | ||
| Event-free Survival | Onasemnogene Abeparvovec-xioi | 20 | Participants | ||
| Ability to Thrive | Onasemnogene Abeparvovec-xioi | 9 | Participants | ||
| Ventilatory Support Independence | Onasemnogene Abeparvovec-xioi | 18 | 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 | 22 | |
| Overall Study | COMPLETED | Onasemnogene Abeparvovec-xioi | 19 | |
| Overall Study | NOT COMPLETED | Onasemnogene Abeparvovec-xioi | 3 |
Source ClinicalTrials.gov
Adverse events80
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 | Diastolic hypertension | Vascular disorders | Onasemnogene Abeparvovec-xioi | 2 | 22 | 2 |
| other | Pyrexia | General disorders | Onasemnogene Abeparvovec-xioi | 12 | 22 | 26 |
| other | Use of accessory respiratory muscles | Respiratory, thoracic and mediastinal disorders | Onasemnogene Abeparvovec-xioi | 5 | 22 | 7 |
| other | Contusion | Injury, poisoning and procedural complications | Onasemnogene Abeparvovec-xioi | 4 | 22 | 4 |
| other | Arthropod bite | Injury, poisoning and procedural complications | Onasemnogene Abeparvovec-xioi | 3 | 22 | 7 |
| other | Aspartate aminotransferase increased | Investigations | Onasemnogene Abeparvovec-xioi | 6 | 22 | 9 |
| other | Alanine aminotransferase increased | Investigations | Onasemnogene Abeparvovec-xioi | 5 | 22 | 8 |
| other | Blood creatine phosphokinase MB increased | Investigations | Onasemnogene Abeparvovec-xioi | 2 | 22 | 2 |
| other | Gamma-glutamyltransferase increased | Investigations | Onasemnogene Abeparvovec-xioi | 2 | 22 | 2 |
| other | Lymphocyte count decreased | Investigations | Onasemnogene Abeparvovec-xioi | 2 | 22 | 2 |
1 to 10 of 80Source ClinicalTrials.gov
See all 80 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 | 22 |
| Total, other adverse events | Onasemnogene Abeparvovec-xioi | 22 | 22 |
| Total, serious adverse events | Onasemnogene Abeparvovec-xioi | 10 | 22 |
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/NCT03306277
GET https://conductscience.org/api/trials/v1/NCT03306277/events?page=2