Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
Model scores of the 29,894 trials active now.
Model score 37% for any early termination, 15% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 14% of the time (121 trials).
Finished trial that started 2008–2014: scored by a model trained without any trial from its start year, so its own outcome was never used.
How it ended
Terminated on 2009-05-31, after 2008-01-31 start.
Reason given by the sponsor: “enrollment”
The score above never used this outcome: finished trial that started 2008–2014: scored by a model trained without any trial from its start year, so its own outcome was never used.
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 2009-05-31. 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: ulntherapygradedlallowed
Phrases that lowered it: subjectsclinicaleffectjudgeddrug -
Drug's FAERS reports with a death: 60% (raises risk) · Drug's FDA history
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Accepts healthy volunteers: No (raises risk) · Who can join
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Phase: 1/2 (raises risk) · Design
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Design: single group (raises risk) · Design
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Responsible party: not given (raises risk) · Design
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Has a data monitoring committee: Yes (raises risk) · Design
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Primary purpose: treatment (raises risk) · Design
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Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
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Minimum age: 0.17 (lowers risk) · Who can join
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Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
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Disease area, cancer: No (lowers risk) · Disease area
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Number of arms: 1 (raises risk) · Design
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Drug's FAERS reports before start: 5 (lowers risk) · Drug's FDA history
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Drug's FAERS reports marked serious: 100% (raises risk) · Drug's FDA history
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Maximum age: 4 (raises risk) · Who can join
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Sponsor type: academic or other (raises risk) · Sponsor's track record
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Collaborators: 1 (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
- 1/2 (typical: 2)
- Number of arms
- 1 (typical: 2)
- Allocation
- non randomized (typical: randomized)
- Design
- single group (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- Yes (typical: 56% yes)
- Responsible party
- not given (typical: sponsor)
- Collaborators
- 1 (typical: 0)
- Keywords listed
- 10 (typical: 1)
Who can join
- Minimum age
- 0.17 (typical: 18)
- Maximum age
- 4 (typical: 71)
- Sex
- all
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- academic or other
- Sponsor's past trials
- 0 (typical: 32)
- Sponsor's past termination rate
- no earlier finished trials (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 0 (typical: 19)
Drug's FDA history
- Drugs with FDA adverse-event history
- 1
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 5 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 2 (typical: 412.5)
- Drug's FAERS reports marked serious
- 100% (typical: 85.6%)
- Drug's FAERS reports with a death
- 60% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology
- Drugs matched to FDA substances
- phenylbutyrate, phenylbutyric acid
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
- Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III NCT00439569 · started 2008 · scored riskier than 99% of active trials terminated
- 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
- 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
- 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
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.