A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Model scores of the 29,894 trials active now.
Model score 25% for any early termination, 3% for termination due to slow enrollment. Past trials like this one (phase and sponsor type, started 2008–2016) ended terminated 16% of the time (370 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2025-07-07, after 2018-11-27 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 2025-07-07. 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: stemthe safetyand efficacydonorsubjects
Phrases that lowered it: participantssicklepriorto enrollmentcell disease -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 11.6% (raises risk) · Sponsor's track record
-
Has a data monitoring committee: Yes (raises risk) · Design
-
Design: single group (raises risk) · Design
-
Phase: 2/3 (raises risk) · Design
-
Trials the sponsor started in the prior 2 years: 27 (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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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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Disease area, cancer: No (lowers risk) · Disease area
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Collaborators: 1 (raises risk) · Design
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Sponsor's past trials: 95 (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
- 2/3 (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
- 1 (typical: 0)
- Keywords listed
- 0 (typical: 1)
Who can join
- Minimum age
- 12 (typical: 18)
- Maximum age
- 35 (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
- 95 (typical: 32)
- Sponsor's past termination rate
- 11.6% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 27 (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
- exagamglogene autotemcel
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 Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia NCT03655678 · started 2018 · scored riskier than 60% of active trials completed
- A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia NCT04925206 · started 2021 · scored riskier than 70% of active trials terminated
- Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) NCT04443907 · started 2020 · scored riskier than 95% of active trials terminated
- CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease NCT04091737 · started 2019 · scored riskier than 89% of active trials terminated
- A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease NCT01279616 · started 2010 · scored riskier than 99% 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.