Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
Model scores of the 29,894 trials active now.
Model score 25% for any early termination, 14% 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 in 2015 or later: the model never saw it during training.
How it ended
Completed on 2022-09-28, after 2018-01-03 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 2022-09-28. 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: donorcellsmarrowtherapycell
Phrases that lowered it: and safetyefficacy andsubjectssinglesafety of -
Accepts healthy volunteers: No (raises risk) · Who can join
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Sponsor's past termination rate: 12.5% (raises risk) · Sponsor's track record
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Drug's FAERS reports with a death: 39.8% (raises risk) · Drug's FDA history
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Phase: 1/2 (raises risk) · Design
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Design: single group (raises risk) · Design
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Drug's FAERS reports marked serious: 96.8% (raises risk) · Drug's FDA history
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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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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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Trials the sponsor started in the prior 2 years: 3 (raises risk) · Sponsor's track record
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Primary purpose: treatment (raises risk) · Design
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Number of arms: 1 (raises risk) · Design
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Collaborators: 1 (raises risk) · Design
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Drug's FAERS reports before start: 5066 (raises risk) · Drug's FDA history
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Maximum age: 17 (raises risk) · Who can join
-
Blinding: none (raises risk) · Design
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Responsible party: sponsor (lowers 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
- 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
- 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
- 16 (typical: 32)
- Sponsor's past termination rate
- 12.5% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 3 (typical: 19)
Drug's FDA history
- Drugs with FDA adverse-event history
- 2 (typical: 1)
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 5066 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 929 (typical: 412.5)
- Drug's FAERS reports marked serious
- 96.8% (typical: 85.6%)
- Drug's FAERS reports with a death
- 39.8% (typical: 15.9%)
Disease area and drugs
- Disease areas
- none of the 14 tracked areas
- Drugs matched to FDA substances
- busulfan, pegademase bovine
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
- 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
- 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
- Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease NCT02234934 · started 2015 · scored riskier than 46% 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.