An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
Model scores of the 29,894 trials active now.
Model score 27% for any early termination, 3% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 15% of the time (95 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2022-12-29, after 2015-04-02 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 2022-12-29. 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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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: ngtypespinaladministrationsafety tolerability
Phrases that lowered it: tubeevaluate theto evaluateopen labelefficacy -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 13.7% (raises risk) · Sponsor's track record
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Phase: 1/2 (raises risk) · Design
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Has a data monitoring committee: Yes (raises risk) · Design
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Sponsor's past trials: 761 (raises risk) · Sponsor's track record
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Disease area, neurology: Yes (raises risk) · Disease area
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Trials the sponsor started in the prior 2 years: 242 (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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Design: single group (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Primary purpose: treatment (raises risk) · Design
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Maximum age: 0.5 (raises risk) · Who can join
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Minimum age: 0.08 (lowers risk) · Who can join
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Collaborators: 0 (raises risk) · Design
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Sponsor type: industry (lowers risk) · Sponsor's track record
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
- 1/2 (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
- 1
Who can join
- Minimum age
- 0.08 (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
- 761 (typical: 32)
- Sponsor's past termination rate
- 13.7% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 242 (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
- neurology
- Drugs matched to FDA substances
- branaplam
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
- A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). NCT02462759 · started 2015 · scored riskier than 84% of active trials terminated
- Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I NCT00439218 · started 2008 · scored riskier than 99% of active trials terminated
- A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) NCT01839656 · started 2013 · scored riskier than 90% of active trials completed
- An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy NCT03921528 · started 2019 · scored riskier than 59% of active trials completed
- Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients. NCT01302600 · started 2010 · scored riskier than 57% of active trials completed
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.