Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Model scores of the 29,894 trials active now.
Model score 13% for any early termination, 4% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 10% of the time (1,463 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2020-09-11, after 2018-08-16 start.
The score above never used this outcome: finished trial that started in 2015 or later: the model never saw it during training.
Termination over time, as the model saw it at the start
Cumulative chance of ending terminated (red) or completed (green) by each year from its start; it actually ended on 2020-09-11. The rest of the way to 100% is still running.
From the competing-risks survival model (termination vs completion, still-running trials censored), fit to trials that started 2008–2014 and recalibrated to termination rates over 2022–2024, since termination has become more common. Checked on past years, it still ran a little low when the rise continued.
What moved the score
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Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
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Accepts healthy volunteers: No (raises risk) · Who can join
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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: intravenoustime ofinfusionadministrationhours
Phrases that lowered it: patientstrialcopiesgrowtharm -
Phase: 3 (lowers risk) · Design
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Design: single group (raises risk) · Design
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Has a data monitoring committee: Yes (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Trials the sponsor started in the prior 2 years: 3 (raises risk) · Sponsor's track record
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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Primary purpose: treatment (raises risk) · Design
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Minimum age: not given (lowers risk) · Who can join
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Maximum age: 0.5 (raises risk) · Who can join
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Number of arms: 1 (raises risk) · Design
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Collaborators: 0 (raises risk) · Design
Every input that moved this score, largest first: SHAP contributions in log-odds, red raising risk and green lowering it. They describe the model, not causes of termination. Phrases are traced from the text features back to this registration's words, approximately.
The trial
Design
- Phase
- 3 (typical: 2)
- Number of arms
- 1 (typical: 2)
- Allocation
- not applicable (typical: randomized)
- Design
- single group (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- Yes (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 0 (typical: 1)
Who can join
- Minimum age
- not given (typical: 18)
- Maximum age
- 0.5 (typical: 71)
- Sex
- all
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- industry (typical: academic or other)
- Sponsor's past trials
- 1 (typical: 32)
- Sponsor's past termination rate
- 0% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 3 (typical: 19)
Drug's FDA history
- Drugs with FDA adverse-event history
- 0 (typical: 1)
- Drug has FAERS history
- No (typical: 64% yes)
- Drug's FAERS reports before start
- 0 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 0 (typical: 412.5)
- Drug's FAERS reports marked serious
- no FAERS history (typical: 85.6%)
- Drug's FAERS reports with a death
- no FAERS history (typical: 15.9%)
Disease area and drugs
- Disease areas
- none of the 14 tracked areas
- Drugs matched to FDA substances
- onasemnogene abeparvovec xioi
Typical values are among the trials active now. Sponsor and drug history are counted only up to this trial's start date.
Similar finished trials
- Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 NCT03306277 · started 2017 · scored riskier than 31% of active trials completed
- Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies NCT03837184 · started 2019 · scored riskier than 29% of active trials completed
- Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 NCT02122952 · started 2014 · scored riskier than 61% of active trials completed
- Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 NCT03505099 · started 2018 · scored riskier than 44% of active trials completed
- Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy NCT03381729 · started 2017 · scored riskier than 71% of active trials terminated
Closest by meaning of the registration text (PubMedBERT embeddings), among finished trials. How they ended is context, not an input to this trial's score.