Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
Model scores of the 29,894 trials active now.
Model score 16% for any early termination, 6% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 30% of the time (37 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2024-12-01, after 2015-10-29 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 2024-12-01. 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: cellschronicpatientpatientshepatitis
Phrases that lowered it: effectclinicalefficacyevaluatethe upper -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Collaborators: 4 (lowers risk) · Design
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Phase: 1/2 (raises risk) · Design
-
Sponsor's past termination rate: 7.8% (raises risk) · Sponsor's track record
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Design: single group (raises risk) · Design
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Trials the sponsor started in the prior 2 years: 75 (lowers risk) · Sponsor's track record
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Disease area, cancer: No (lowers risk) · Disease area
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Sponsor type: academic or other (raises risk) · Sponsor's track record
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Sponsor's past trials: 245 (lowers risk) · Sponsor's track record
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Minimum age: 1.92 (lowers risk) · Who can join
-
Has a data monitoring committee: Yes (raises risk) · Design
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Primary purpose: treatment (raises risk) · Design
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Responsible party: principal investigator (lowers risk) · Design
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Maximum age: not given (raises risk) · Who can join
-
Number of arms: 2 (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
- 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
- principal investigator (typical: sponsor)
- Collaborators
- 4 (typical: 0)
- Keywords listed
- 3 (typical: 1)
Who can join
- Minimum age
- 1.92 (typical: 18)
- Maximum age
- not given (typical: 71)
- Sex
- male (typical: all)
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- academic or other
- Sponsor's past trials
- 245 (typical: 32)
- Sponsor's past termination rate
- 7.8% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 75 (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
- immunology, 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
- Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID NCT02999984 · started 2016 · scored riskier than 82% of active trials completed
- Calcineurin Inhibitor-Free Interventions BMT CTN 1301 for Prevention of Graft-versus-Host Disease (BMT CTN 1301) NCT02345850 · started 2015 · scored riskier than 74% of active trials completed
- Phase I Study of GNKG168 in Acute Lymphoblastic Leukemia and Acute Myelogenous Leukemia NCT01743807 · started 2014 · scored riskier than 96% of active trials terminated
- Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID NCT03765632 · started 2018 · scored riskier than 88% of active trials completed
- Phase II Trial of Efprezimod Alfa (CD24Fc, MK-7110) for the Prevention of Acute Graft-Versus-Host Disease (GVHD) Following Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) (MK-7110-002) NCT02663622 · started 2016 · scored riskier than 48% 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.