Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Model scores of the 29,894 trials active now.
Model score 15% for any early termination, 2% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 19% of the time (731 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2023-12-22, after 2016-12-23 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 2023-12-22. 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) (lowers risk) · Wording
Phrases that raised it: typepriortime ofrecoveredcentral
Phrases that lowered it: participantsscreeningopen labeleffectpharmacokinetics -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 9.1% (raises risk) · Sponsor's track record
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Phase: 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: 926 (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: 140 (lowers 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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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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Maximum age: 0.58 (raises risk) · Who can join
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Design: sequential (raises risk) · Design
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Minimum age: 0.08 (lowers risk) · Who can join
-
Sponsor type: industry (lowers risk) · Sponsor's track record
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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
- 2
- Number of arms
- 2
- Allocation
- non randomized (typical: randomized)
- Design
- sequential (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
- 0.08 (typical: 18)
- Maximum age
- 0.58 (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
- 926 (typical: 32)
- Sponsor's past termination rate
- 9.1% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 140 (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
- risdiplam
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 of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy NCT03032172 · started 2017 · scored riskier than 39% of active trials completed
- A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy NCT05808764 · started 2024 · scored riskier than 17% of active trials completed
- A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants NCT02908685 · started 2016 · scored riskier than 47% of active trials completed
- An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) NCT02268552 · started 2015 · scored riskier than 92% of active trials completed
- A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) NCT02240355 · started 2014 · scored riskier than 18% 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.