Open-label, Pivotal Clinical Trial to Confirm Efficacy and Safety of Autologous Grafts Containing Stem Cells Genetically Modified for Epidermis Restoration in Patients With Junctional Epidermolysis Bullosa
Model scores of the 29,894 trials active now.
Model score 16% for any early termination, 7% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 11% of the time (75 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Terminated on 2024-11-22, after 2022-07-15 start.
Reason given by the sponsor: “business”
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 2024-11-22. 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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Sponsor's past termination rate: 66.7% (raises risk) · Sponsor's track record
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Accepts healthy volunteers: No (raises risk) · Who can join
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Wording of the registration (title, summary, criteria) (lowers risk) · Wording
Phrases that raised it: transplantationto assessassess thesubjectwomen
Phrases that lowered it: efficacypatientscontainingand safetyclinical -
Design: single group (raises risk) · Design
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Collaborators: 2 (lowers risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Minimum age: 0.5 (lowers risk) · Who can join
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Phase: 2/3 (raises risk) · Design
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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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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Sponsor's past trials: 3 (lowers risk) · Sponsor's track record
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Number of arms: 1 (raises risk) · Design
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Responsible party: sponsor (lowers risk) · Design
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Trials the sponsor started in the prior 2 years: 1 (raises risk) · Sponsor's track record
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Has a data monitoring committee: No (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
- 2/3 (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
- No (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 2 (typical: 0)
- Keywords listed
- 0 (typical: 1)
Who can join
- Minimum age
- 0.5 (typical: 18)
- Maximum age
- 65 (typical: 71)
- Sex
- all
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- industry (typical: academic or other)
- Sponsor's past trials
- 3 (typical: 32)
- Sponsor's past termination rate
- 66.7% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 1 (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
- none of the 14 tracked areas
- 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
- Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. NCT02984085 · started 2017 · scored riskier than 29% of active trials terminated
- Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa NCT01263379 · started 2010 · scored riskier than 55% of active trials completed
- Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) NCT04227106 · started 2020 · scored riskier than 34% of active trials completed
- Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa NCT02493816 · started 2015 · scored riskier than 34% of active trials completed
- Biochemical Correction of Severe EB by Allo HSCT and Off-the-shelf MSCs NCT01033552 · started 2010 · scored riskier than 86% 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.