Trial · NCT02122952
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
NCT02122952, Phase 1, Completed, Novartis Gene Therapies; results first posted 2019-05-10
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1. Trials, record NCT02122952. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02122952
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
- NCT number
- NCT02122952
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 15 participants, actual
- Lead sponsor
- Novartis Gene Therapies
- Conditions
- Spinal Muscular Atrophy 1
- Interventions
- AVXS-101
- First posted
- 2014-04-25
- Last updated
- 2022-09-15
- Results first posted
- 2019-05-10
- Limitations and caveats
- Link to the full study results: https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17849
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment | 2 years | |
| Secondary | Number of Participants Who Experienced Permanent Ventilation or Death | Up to 13.6 months of age | Permanent ventilation was defined as the requirement of ≥ 16-hour respiratory assistance, including non-invasive ventilatory support, per day continuously for ≥ 2 weeks in the absence of an acute reversible illness, excluding perioperative ventilation. |
| Secondary | Percent Change From Baseline in Mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score | Baseline to 24 months post-dose | Score ranges from 0 to 64, where 64 is the maximum possible score. A higher score is indicative of higher/better motor function. CHOP-INTEND assessments were discontinued once patients achieved higher functioning status, so the number of available data points decreased over time. |
| Secondary | Number of Participants With Assessed Improvement in Motor Function | 24 months post-dose | Improvement in motor function was determined by achievement of developmental milestones, specifically achievement of ability to sit unassisted for at least 30 seconds, determined by physical therapist and confirmed by an independent central video reviewer. Achievement of functional independent sitting was defined as the ability to maintain a sitting position independently for at least 30 seconds as confirmed per video evaluation by an expert central reviewer based on videos taken either at scheduled visits or provided by the parent/legal guardian. |
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 | Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment | 2 years | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants Who Experienced Permanent Ventilation or Death | Up to 13.6 months of age | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Percent Change From Baseline in Mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score | Baseline to 24 months post-dose | Includes all treated participants with non-missing data. For a major, systemic, and externally imposed limitation of movement that prevented accurate assessment of multiple items, the total score was regarded as missing. Also, CHOP-INTEND assessments were discontinued once participants achieved higher functioning status (2 consecutive scores ≥62). | Percentage Change | MEAN |
| SECONDARY | Number of Participants With Assessed Improvement in Motor Function | 24 months post-dose | full analysis set- all treated patients | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group7
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment | Cohort 1 | 1 | Participants | ||
| Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment | Cohort 2 | 3 | Participants | ||
| Number of Participants Who Experienced Permanent Ventilation or Death | Cohort 1 | 0 | Participants | ||
| Number of Participants Who Experienced Permanent Ventilation or Death | Cohort 2 | 0 | Participants | ||
| Percent Change From Baseline in Mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score | Cohort 2 | 30.7 | Standard Deviation 145.61 | Percentage Change | |
| Number of Participants With Assessed Improvement in Motor Function | Cohort 1 | 0 | Participants | ||
| Number of Participants With Assessed Improvement in Motor Function | Cohort 2 | 9 | Participants |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1 | 3 | |
| Overall Study | STARTED | Cohort 2 | 12 | |
| Overall Study | COMPLETED | Cohort 1 | 3 | |
| Overall Study | COMPLETED | Cohort 2 | 12 | |
| Overall Study | NOT COMPLETED | Cohort 1 | 0 | |
| Overall Study | NOT COMPLETED | Cohort 2 | 0 |
Source ClinicalTrials.gov
Adverse events282
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 | constipation | Gastrointestinal disorders | Cohort 1 | 1 | 3 | 1 |
| other | constipation | Gastrointestinal disorders | Cohort 2 | 6 | 12 | 8 |
| other | diarrhea | Gastrointestinal disorders | Cohort 1 | 0 | 3 | 0 |
| other | diarrhea | Gastrointestinal disorders | Cohort 2 | 3 | 12 | 3 |
| other | gastroesophageal reflux disease | Gastrointestinal disorders | Cohort 1 | 1 | 3 | 1 |
| other | gastroesophageal reflux disease | Gastrointestinal disorders | Cohort 2 | 5 | 12 | 6 |
| other | vomiting | Gastrointestinal disorders | Cohort 1 | 0 | 3 | 0 |
| other | vomiting | Gastrointestinal disorders | Cohort 2 | 8 | 12 | 11 |
| other | pyrexia | General disorders | Cohort 1 | 1 | 3 | 1 |
| other | pyrexia | General disorders | Cohort 2 | 7 | 12 | 12 |
1 to 10 of 282Source ClinicalTrials.gov
See all 282 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 | 0 | 3 |
| Total, all-cause mortality | Cohort 2 | 0 | 12 |
| Total, other adverse events | Cohort 1 | 3 | 3 |
| Total, other adverse events | Cohort 2 | 12 | 12 |
| Total, serious adverse events | Cohort 1 | 3 | 3 |
| Total, serious adverse events | Cohort 2 | 10 | 12 |
Source ClinicalTrials.gov
Papers10
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 comparisons5
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cohort 1 vs Cohort 2: Number of Participants With Assessed Improvement in Motor Function | Trial comparisons |
| Cohort 1 vs Cohort 2: Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment | Trial comparisons |
| Cohort 1 vs Cohort 2: Number of Participants Who Experienced Permanent Ventilation or Death | Trial comparisons |
| Cohort 1 vs Cohort 2: deaths from any cause | Trial comparisons |
| Cohort 1 vs Cohort 2: 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/NCT02122952
GET https://conductscience.org/api/trials/v1/NCT02122952/events?page=2