Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
Model scores of the 29,894 trials active now.
Model score 18% for any early termination, 8% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 18% of the time (283 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 2018-08-27, after 2013-08-02 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 2018-08-27. 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: dlcardiacserumboneevidence of
Phrases that lowered it: severeefficacysafetyhivviral load -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Collaborators: 4 (lowers risk) · Design
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Drug's FAERS reports with a death: 46.6% (raises risk) · Drug's FDA history
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Sponsor's past termination rate: 5.7% (lowers risk) · Sponsor's track record
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Trials the sponsor started in the prior 2 years: 75 (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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Sponsor's past trials: 176 (lowers risk) · Sponsor's track record
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Maximum age: 17 (raises risk) · Who can join
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Number of arms: 1 (raises risk) · Design
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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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Minimum age: 0.08 (lowers risk) · Who can join
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Disease area, cancer: No (lowers risk) · Disease area
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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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Drug's FAERS reports marked serious: 98.3% (raises risk) · Drug's FDA history
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Drug's FAERS reports before start: 2298 (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
- 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
- principal investigator (typical: sponsor)
- Collaborators
- 4 (typical: 0)
- Keywords listed
- 4 (typical: 1)
Who can join
- Minimum age
- 0.08 (typical: 18)
- Maximum age
- 17 (typical: 71)
- Sex
- all
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- academic or other
- Sponsor's past trials
- 176 (typical: 32)
- Sponsor's past termination rate
- 5.7% (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
- 1
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 2298 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 397 (typical: 412.5)
- Drug's FAERS reports marked serious
- 98.3% (typical: 85.6%)
- Drug's FAERS reports with a death
- 46.6% (typical: 15.9%)
Disease area and drugs
- Disease areas
- none of the 14 tracked areas
- Drugs matched to FDA substances
- busulfan
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 NCT03765632 · started 2018 · scored riskier than 88% 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 NCT02999984 · started 2016 · scored riskier than 82% of active trials completed
- Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome NCT00604201 · started 2008 · scored riskier than 58% of active trials completed
- Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector NCT01129544 · started 2010 · scored riskier than 79% 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.