Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Model scores of the 29,894 trials active now.
Model score 22% for any early termination, 5% 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 (56 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 2023-03-31, after 2010-04-30 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 2023-03-31. 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: leukemiachemotherapycelltransplantdonor
Phrases that lowered it: efficacypatients whotrials20clinical -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Collaborators: 3 (lowers risk) · Design
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Phase: 1/2 (raises risk) · Design
-
Responsible party: sponsor investigator (raises risk) · Design
-
Design: single group (raises risk) · Design
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Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
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Trials the sponsor started in the prior 2 years: 0 (raises 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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Has a data monitoring committee: Yes (raises risk) · Design
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Maximum age: not given (raises risk) · Who can join
-
Minimum age: not given (raises risk) · Who can join
-
Sponsor's past trials: 0 (raises risk) · Sponsor's track record
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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 (raises risk) · Drug's FDA history
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Primary purpose: treatment (raises risk) · Design
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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, dermatology: 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
- 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
- 3 (typical: 0)
- Keywords listed
- 4 (typical: 1)
Who can join
- Minimum age
- not given (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
- 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
- metabolic, immunology
- 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
- Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene NCT01852071 · started 2013 · scored riskier than 62% of active trials completed
- MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID NCT00794508 · started 2008 · scored riskier than 78% of active trials completed
- 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
- 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
- Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome NCT01410825 · started 2011 · scored riskier than 87% 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.