Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
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 2021-06-08, after 2020-01-09 start.
Reason given by the sponsor: “other”
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 2021-06-08. 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: 15.5% (raises risk) · Sponsor's track record
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Accepts healthy volunteers: No (raises risk) · Who can join
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Sponsor's past trials: 1873 (raises risk) · Sponsor's track record
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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: sickletransfusion30pulmonarywhichever
Phrases that lowered it: participantparticipantsthe investigatorevaluate theopinion -
Phase: 2 (raises risk) · Design
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Has a data monitoring committee: not given (lowers risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Number of arms: 4 (lowers risk) · Design
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Primary purpose: treatment (raises risk) · Design
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Minimum age: 18 (raises risk) · Who can join
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Trials the sponsor started in the prior 2 years: 158 (lowers risk) · Sponsor's track record
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Drug's FAERS reports with a death: 0% (lowers risk) · Drug's FDA history
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Design: sequential (raises risk) · Design
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Maximum age: 60 (lowers risk) · Who can join
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Drug's FAERS reports marked serious: 100% (raises risk) · Drug's FDA history
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
- 4 (typical: 2)
- Allocation
- non randomized (typical: randomized)
- Design
- sequential (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- not given (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 1
Who can join
- Minimum age
- 18
- Maximum age
- 60 (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
- 1873 (typical: 32)
- Sponsor's past termination rate
- 15.5% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 158 (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
- 3 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 3 (typical: 412.5)
- Drug's FAERS reports marked serious
- 100% (typical: 85.6%)
- Drug's FAERS reports with a death
- 0% (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
- Safety, Tolerability and Pharmacokinetics of FTX-6058 NCT04586985 · started 2020 · scored riskier than 8% of active trials completed
- Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease NCT04255875 · started 2020 · scored riskier than 13% of active trials completed
- Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients NCT03474965 · started 2018 · scored riskier than 86% of active trials completed
- The Effect of Voxelotor on Cerebral Hemodynamic Response in Children With Sickle Cell Anemia NCT05018728 · started 2022 · scored riskier than 89% of active trials terminated
- Adenosine 2A Agonist Lexiscan in Children and Adults With Sickle Cell Disease NCT01085201 · started 2010 · scored riskier than 32% 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.