Comparison · NCT02122952-cc53cadc
Cohort 1 vs Cohort 2: Number of Participants Who Experienced Permanent Ventilation or Death
Calculated by ConductScience from the counts the trial posted to ClinicalTrials.gov.
2 sourcestrial-comparisons-2026-03-23-0.1.0
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ConductScience Library. Cohort 1 vs Cohort 2: Number of Participants Who Experienced Permanent Ventilation or Death. Trial comparisons, record NCT02122952-cc53cadc. Release trial-comparisons-2026-03-23-0.1.0, 23 Mar 2026. https://conductscience.org/library/comparisons/NCT02122952-cc53cadc
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Measures6
Calculated by ConductScience from the counts the trial posted to ClinicalTrials.gov.
| Measure | Value | 95% interval, lower | 95% interval, upper | Method | Note |
|---|---|---|---|---|---|
| Risk in group 1 | 0 | 0.0000000000000000555 | 0.561 | wilson | |
| Risk in group 2 | 0 | 0.0000000000000000278 | 0.242 | wilson | |
| Risk difference, group 1 minus group 2 | 0 | -0.242 | 0.561 | newcombe hybrid score | |
| Risk ratio, group 1 over group 2 | log | a group has no participants with the outcome | |||
| Odds ratio, group 1 over group 2 | log | a cell of the two-by-two table is zero | |||
| Number needed to treat | from the risk difference interval | the two groups' risks are equal |
Source ClinicalTrials.gov
Groups2
Neither group, or both, names a comparator, so the groups keep ClinicalTrials.gov's order.
| Group | Title | Description | With the outcome | Analysed | ClinicalTrials.gov code |
|---|---|---|---|---|---|
| Group 1 | Cohort 1 | 6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)~AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter | 0 | 3 | OG000 |
| Group 2 | Cohort 2 | 2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)~AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter | 0 | 12 | OG001 |
Source ClinicalTrials.gov
Outcome
- Type
- SECONDARY
- Title
- Number of Participants Who Experienced Permanent Ventilation or Death
- Description
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.
- Time frame
- Up to 13.6 months of age
- Population
Source ClinicalTrials.gov
Trial
- Title
- Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
- NCT number
- NCT02122952
- Phases
- PHASE1
- Conditions
- Spinal Muscular Atrophy 1
- Interventions
- AVXS-101
- Lead sponsor
- Novartis Gene Therapies
- Results first posted
- 2019-05-10
- Results on ClinicalTrials.gov
- https://clinicaltrials.gov/study/NCT02122952?tab=results
Source ClinicalTrials.gov
Source
- Release
- trial-comparisons-2026-03-23-0.1.0
- Release manifest SHA-256
- f66225897fbf258b57ea10bdacfd0347d3cdfda93f7f5685234b7e06ea36fa57
- AACT copy
- 2026-03-23
- Tables read
- outcome_counts.parquet, outcome_measurements.parquet, outcomes.parquet, reported_event_totals.parquet, reported_events.parquet, result_groups.parquet, studies.parquet, ctgov_trial.parquet
Source ClinicalTrials.gov
Sources 2
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
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
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions. The measures are ConductScience's calculation from them.
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