Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
Model scores of the 29,894 trials active now.
Model score 18% 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 25% of the time (96 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Terminated on 2023-10-02, after 2016-07-05 start.
Reason given by the sponsor: “safety”
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 2023-10-02. 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: 16.1% (raises 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) (lowers risk) · Wording
Phrases that raised it: cellmultiplesafety tolerabilitytolerability andsickle
Phrases that lowered it: participantsparticipanteffectthe pharmacokineticsvisit -
Sponsor's past trials: 1601 (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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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Disease area, cancer: No (lowers risk) · Disease area
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Primary purpose: treatment (raises risk) · Design
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Maximum age: 17 (raises risk) · Who can join
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Trials the sponsor started in the prior 2 years: 175 (lowers risk) · Sponsor's track record
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Minimum age: 0.5 (lowers risk) · Who can join
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Collaborators: 0 (raises risk) · Design
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Drug's FAERS reports before start: 0 (raises risk) · Drug's FDA history
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Disease area, neurology: No (lowers risk) · Disease area
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
- 1 (typical: 2)
- Allocation
- not applicable (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
- 0.5 (typical: 18)
- Maximum age
- 17 (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
- 1601 (typical: 32)
- Sponsor's past termination rate
- 16.1% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 175 (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
- hematology
- Drugs matched to FDA substances
- voxelotor
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 Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD) NCT04983264 · started 2021 · scored riskier than 64% of active trials completed
- Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease NCT04218084 · started 2020 · scored riskier than 43% of active trials terminated
- An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001 NCT03041909 · started 2016 · scored riskier than 31% of active trials completed
- Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease NCT04285827 · started 2021 · scored riskier than 44% of active trials completed
- A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease NCT02285088 · started 2014 · scored riskier than 5% 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.