A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
Model scores of the 29,894 trials active now.
Model score 17% 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 6% of the time (240 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2021-09-01, after 2020-10-15 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 2021-09-01. 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: of studystudy drugthe patientdose ofinvestigational
Phrases that lowered it: patientsopen labellabel studyconsent formphase open -
Phase: 2 (raises risk) · Design
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Accepts healthy volunteers: No (raises risk) · Who can join
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Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
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Drug's FAERS reports marked serious: 56.7% (lowers risk) · Drug's FDA history
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Drug's FAERS reports with a death: 1.6% (lowers risk) · Drug's FDA history
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Design: single group (raises risk) · Design
-
Disease area, cancer: No (lowers risk) · Disease area
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Trials the sponsor started in the prior 2 years: 1 (raises risk) · Sponsor's track record
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Disease area, dermatology: Yes (raises risk) · Disease area
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Number of arms: 1 (raises risk) · Design
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Primary purpose: treatment (raises risk) · Design
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Has a data monitoring committee: No (lowers risk) · Design
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Blinding: none (raises risk) · Design
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Maximum age: not given (raises risk) · Who can join
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Minimum age: 12 (raises risk) · Who can join
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
- 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
- 0
- Keywords listed
- 1
Who can join
- Minimum age
- 12 (typical: 18)
- Maximum age
- not given (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
- 0 (typical: 32)
- Sponsor's past termination rate
- no earlier finished trials (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
- 1
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 826 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 229 (typical: 412.5)
- Drug's FAERS reports marked serious
- 56.7% (typical: 85.6%)
- Drug's FAERS reports with a death
- 1.6% (typical: 15.9%)
Disease area and drugs
- Disease areas
- dermatology
- Drugs matched to FDA substances
- collagen
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
- Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01) NCT05143190 · started 2021 · scored riskier than 18% of active trials completed
- A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) NCT03752905 · started 2019 · scored riskier than 15% of active trials completed
- Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa NCT03529877 · started 2019 · scored riskier than 47% of active trials completed
- ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa NCT02384460 · started 2015 · scored riskier than 10% of active trials completed
- Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa NCT02090283 · started 2014 · scored riskier than 41% of active trials terminated
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.