Trial · NCT03381729
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
NCT03381729, Phase 1, Terminated, Novartis Gene Therapies; results first posted 2023-02-16
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy. Trials, record NCT03381729. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03381729
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
- NCT number
- NCT03381729
- Status
- Terminated
- Phases
- Phase 1
- Enrollment
- 32 participants, actual
- Lead sponsor
- Novartis Gene Therapies
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Onasemnogene Abeparvovec-xioi
- First posted
- 2017-12-22
- Last updated
- 2026-01-26
- Other registries' IDs
- eudract:2020-003678-28
- Results first posted
- 2023-02-16
- 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=17933
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone | From Day 1 up to Month 12 | Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported. |
| Primary | Age 24 to <60 Months Only: Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score at Month 12 | Baseline and Month 12 | The HFMSE contained 33 items which were scored on a scale of 0-2 with a total achievable score ranging from 0, if all activities are failed, to 66, if all the activities are achieved. A positive change from baseline indicates a better outcome. |
| Primary | Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE) | Adverse events were collected from the single dose of study treatment until the end of study visit (12 months for Cohort 1 and 2 and 15 months for Cohort 3) | A TEAE was defined as any event that began or worsened in severity on or after the administration of AVXS-101 through the last study visit. Evaluation of TEAEs included the number of participants with at least one: * TEAE * Serious TEAE * TEAE related to AVXS-101 * TEAE with Common Terminology Criteria for Adverse Events (CTCAE) grade ≥ 3 (grade 3 = severe or medically significant to grade 5 = death related to TEAE) |
| Secondary | Number of Participants Who Achieved the Ability to Walk Alone | From Day 1 up to Month 12 | Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps. |
| Secondary | Average Number of Hours Per Day of Non-invasive Ventilatory Support | Months 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, and 12 | Participants were assessed by a pulmonologist and may have been fitted with a non-invasive positive pressure ventilatory (e.g., Bilevel Positive Airway Pressure BiPAP) at the discretion of the pulmonologist and/or investigator. The number of hours per day of non-invasive ventilatory support was captured continuously by the device. |
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 | Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone | From Day 1 up to Month 12 | Intent-to-Treat (ITT) Set: All enrolled participants who were given an AVXS-101 intrathecal injection. Participants were analyzed according to the assigned dose. Data were only collected in participants aged 6 to \<24 months at time of dosing. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Age 24 to <60 Months Only: Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score at Month 12 | Baseline and Month 12 | ITT Set: All enrolled participants who were given an AVXS-101 intrathecal injection. Participants were analyzed according to the assigned dose. Data were only collected in participants aged 24 to \<60 months at time of dosing. | score on a scale | LEAST_SQUARES_MEAN |
| PRIMARY | Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE) | Adverse events were collected from the single dose of study treatment until the end of study visit (12 months for Cohort 1 and 2 and 15 months for Cohort 3) | Safety Analysis Set: All participants given an AVXS-101 intrathecal injection. Participants were analyzed according to actual dose received. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Achieved the Ability to Walk Alone | From Day 1 up to Month 12 | ITT Set: All enrolled participants who were given an AVXS-101 intrathecal injection. Participants were analyzed according to the assigned dose. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Average Number of Hours Per Day of Non-invasive Ventilatory Support | Months 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, and 12 | Data were only collected in participants requiring BiPAP support. | hours/day | MEAN |
Source ClinicalTrials.gov
Results by group51
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Able to Stand Alone | 1 | Participants | |
| Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Able to Stand Alone | 1 | Participants | |
| Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | Able to Stand Alone | 0 | Participants | |
| Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Unable to Stand Alone | 2 | Participants | |
| Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Unable to Stand Alone | 12 | Participants | |
| Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | Unable to Stand Alone | 4 | Participants | |
| Age 24 to <60 Months Only: Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score at Month 12 | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | 6.0 | 95% Confidence Interval 3.7 to 8.3 | score on a scale | |
| Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE) | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | TEAE | 3 | Participants | |
| Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE) | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | TEAE | 13 | Participants | |
| Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE) | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | TEAE | 12 | Participants |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 results by groupParticipant flow20
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 3 | |
| Overall Study | STARTED | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 13 | |
| Overall Study | STARTED | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | 12 | |
| Overall Study | STARTED | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | 4 | |
| Overall Study | STARTED | Cohort 3: 2.4E14 vg - Age 24 to <60 Months | 0 | |
| Overall Study | Received AVXS-101 | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 3 | |
| Overall Study | Received AVXS-101 | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 13 | |
| Overall Study | Received AVXS-101 | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | 12 | |
| Overall Study | Received AVXS-101 | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | 4 | |
| Overall Study | Received AVXS-101 | Cohort 3: 2.4E14 vg - Age 24 to <60 Months | 0 |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 participant flowAdverse events572
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: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 0 | 3 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 2 | 13 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | 1 | 12 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | 0 | 4 | |
| other | Leukopenia | Blood and lymphatic system disorders | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 0 | 3 | |
| other | Leukopenia | Blood and lymphatic system disorders | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 1 | 13 | |
| other | Leukopenia | Blood and lymphatic system disorders | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | 0 | 12 | |
| other | Leukopenia | Blood and lymphatic system disorders | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | 0 | 4 | |
| other | Tachycardia | Cardiac disorders | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 0 | 3 | |
| other | Tachycardia | Cardiac disorders | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 2 | 13 |
1 to 10 of 572Source ClinicalTrials.gov
See all 572 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 0 | 3 |
| Total, all-cause mortality | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 0 | 13 |
| Total, all-cause mortality | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | 0 | 12 |
| Total, all-cause mortality | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | 0 | 4 |
| Total, other adverse events | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 3 | 3 |
| Total, other adverse events | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 13 | 13 |
| Total, other adverse events | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | 12 | 12 |
| Total, other adverse events | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | 4 | 4 |
| Total, serious adverse events | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 1 | 3 |
| Total, serious adverse events | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 2 | 13 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers3
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/NCT03381729
GET https://conductscience.org/api/trials/v1/NCT03381729/results?page=2