Trial · NCT03505099
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
NCT03505099, Phase 3, Completed, Novartis Gene Therapies; results first posted 2022-01-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2. Trials, record NCT03505099. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03505099
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
- NCT number
- NCT03505099
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 30 participants, actual
- Lead sponsor
- Novartis Gene Therapies
- Conditions
- Spinal Muscular Atrophy
- Interventions
- onasemnogene abeparvovec-xioi
- First posted
- 2018-04-23
- Last updated
- 2026-01-26
- Other registries' IDs
- eudract:2017-004087-35
- Results first posted
- 2022-01-11
- Limitations and caveats
- Link to the full study results: https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17902
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds | From Day 1 up to 18 months of age visit | Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 26, confirmed by video recording, as a participant who sits for at least 30 seconds without assistance from another person or object. The participant was allowed to use their upper extremities. |
| Primary | Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds | From Day 1 up to 24 months of age visit | Defined by the BSID GM subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported. |
| Secondary | Cohort 1: Event-free Survival at 14 Months of Age | From Day 1 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. |
| Secondary | Cohort 1: Number of Participants Who Achieved the Ability to Maintain Weight at or Above the Third Percentile Without the Need for Non-Oral or Mechanical Feeding Support | From Day 1 up to 18 months of age | The ability to maintain weight at or above the third percentile without the need for non-oral or mechanical feeding support was defined by meeting the following criteria at each visit up to 18 months of age: * Did not receive nutrition through mechanical support (i.e., feeding tube) * Maintained weight (≥ third percentile for age and sex as defined by World Health Organization \[WHO\] guidelines) consistent with the participant's age at the assessment. |
| Secondary | Cohort 2: Number of Participants Who Achieved the Ability to Walk Alone | From Day 1 up to 24 months of age visit | Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds | From Day 1 up to 18 months of age visit | Intent-to-Treat (ITT) population (cohort 1) - All enrolled participants with bi-allelic SMN1 deletions and 2 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\>C) who received AVXS-101. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds | From Day 1 up to 24 months of age visit | ITT population (cohort 2) - All enrolled participants with bi-allelic SMN1 deletions and 3 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\>C) who received AVXS-101. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Cohort 1: Event-free Survival at 14 Months of Age | From Day 1 up to 14 months of age | ITT population (cohort 1) - All enrolled participants with bi-allelic SMN1 deletions and 2 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\>C) who received AVXS-101. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Cohort 1: Number of Participants Who Achieved the Ability to Maintain Weight at or Above the Third Percentile Without the Need for Non-Oral or Mechanical Feeding Support | From Day 1 up to 18 months of age | ITT population (cohort 1) - All enrolled participants with bi-allelic SMN1 deletions and 2 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\>C) who received AVXS-101. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Cohort 2: Number of Participants Who Achieved the Ability to Walk Alone | From Day 1 up to 24 months of age visit | ITT population (cohort 2) - All enrolled participants with bi-allelic SMN1 deletions and 3 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\>C) who received AVXS-101. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group5
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 14 | Participants | ||
| Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 15 | Participants | ||
| Cohort 1: Event-free Survival at 14 Months of Age | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 14 | Participants | ||
| Cohort 1: Number of Participants Who Achieved the Ability to Maintain Weight at or Above the Third Percentile Without the Need for Non-Oral or Mechanical Feeding Support | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 13 | Participants | ||
| Cohort 2: Number of Participants Who Achieved the Ability to Walk Alone | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 14 | Participants |
Source ClinicalTrials.gov
Participant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 14 | |
| Overall Study | STARTED | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 15 | |
| Overall Study | Received AVXS-101 | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 14 | |
| Overall Study | Received AVXS-101 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 15 | |
| Overall Study | COMPLETED | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 14 | |
| Overall Study | COMPLETED | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 15 | |
| Overall Study | NOT COMPLETED | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 0 | |
| Overall Study | NOT COMPLETED | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 0 |
Source ClinicalTrials.gov
Adverse events276
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 | Lymphadenopathy | Blood and lymphatic system disorders | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 1 | 14 | 1 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 0 | 15 | 0 |
| other | Microcytic anaemia | Blood and lymphatic system disorders | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 1 | 14 | 1 |
| other | Microcytic anaemia | Blood and lymphatic system disorders | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 2 | 15 | 3 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 1 | 14 | 1 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 0 | 15 | 0 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 0 | 14 | 0 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 1 | 15 | 1 |
| other | Dacryostenosis congenital | Congenital, familial and genetic disorders | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 1 | 14 | 1 |
| other | Dacryostenosis congenital | Congenital, familial and genetic disorders | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 1 | 15 | 1 |
1 to 10 of 276Source ClinicalTrials.gov
See all 276 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 0 | 14 |
| Total, all-cause mortality | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 0 | 15 |
| Total, other adverse events | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 14 | 14 |
| Total, other adverse events | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 15 | 15 |
| Total, serious adverse events | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 5 | 14 |
| Total, serious adverse events | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 3 | 15 |
Source ClinicalTrials.gov
Papers3
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 vs Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2: deaths from any cause | Trial comparisons |
| Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 vs Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2: serious adverse events | Trial comparisons |
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/NCT03505099
GET https://conductscience.org/api/trials/v1/NCT03505099/events?page=2