Stem Cell Gene Therapy for Sickle Cell Disease
Model scores of the 29,894 trials active now.
Model score 23% for any early termination, 11% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 12% of the time (32 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
Completed on 2025-09-24, after 2014-12-31 start.
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 2025-09-24. 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: donorcellhlamriallogeneic
Phrases that lowered it: painparticipantupperdayslimit of -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
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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: sponsor investigator (raises risk) · Design
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Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
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Sponsor type: academic or other (raises risk) · Sponsor's track record
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Has a data monitoring committee: Yes (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Minimum age: 18 (raises risk) · Who can join
-
Sponsor's past trials: 0 (raises risk) · Sponsor's track record
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Collaborators: 1 (raises risk) · Design
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Primary purpose: treatment (raises risk) · Design
-
Number of arms: 1 (raises risk) · Design
-
Blinding: none (raises risk) · Design
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Drug's FAERS reports with a death: no FAERS history (lowers 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
- 1/2 (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 investigator (typical: sponsor)
- Collaborators
- 1 (typical: 0)
- Keywords listed
- 4 (typical: 1)
Who can join
- Minimum age
- 18
- Maximum age
- not given (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
- 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
- none matched
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
- Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601) NCT00745420 · started 2008 · scored riskier than 71% of active trials completed
- Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders NCT00489281 · started 2008 · scored riskier than 60% of active trials terminated
- Stem Cell Transplantation for Sickle Cell Anemia NCT01877837 · started 2011 · scored riskier than 92% of active trials completed
- 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
- Sickle Cell Disease Conditioning for Bone Marrow Transplant NCT00968162 · started 2009 · scored riskier than 99% 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.