Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency
Model scores of the 29,894 trials active now.
Model score 17% for any early termination, 1% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 15% of the time (734 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2023-02-01, after 2019-05-01 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 2023-02-01. 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
-
Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: subjectssubjectrandomizationlong termextension
Phrases that lowered it: malehas historyand safetyefficacy andperiod -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
-
Disease area, neurology: Yes (raises risk) · Disease area
-
Has a data monitoring committee: Yes (raises risk) · Design
-
Phase: 3 (lowers risk) · Design
-
Design: parallel (raises risk) · Design
-
Disease area, cancer: No (lowers risk) · Disease area
-
Blinding: quadruple (lowers risk) · Design
-
Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
-
Trials the sponsor started in the prior 2 years: 1 (raises risk) · Sponsor's track record
-
Primary purpose: treatment (raises risk) · Design
-
Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
-
Number of arms: 3 (lowers risk) · Design
-
Minimum age: 2 (lowers risk) · Who can join
-
Sponsor's past trials: 1 (lowers risk) · Sponsor's track record
-
Sponsor type: industry (lowers risk) · Sponsor's track record
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
- 3 (typical: 2)
- Number of arms
- 3 (typical: 2)
- Allocation
- randomized
- Design
- parallel
- Primary purpose
- treatment
- Blinding
- quadruple (typical: none)
- Has a data monitoring committee
- Yes (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 1 (typical: 0)
- Keywords listed
- 1
Who can join
- Minimum age
- 2 (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
- 1 (typical: 32)
- Sponsor's past termination rate
- 0% (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
- neurology, metabolic
- Drugs matched to FDA substances
- pegzilarginase
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
- A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency NCT05676853 · started 2023 · scored riskier than 14% of active trials terminated
- Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency NCT06582524 · started 2024 · scored riskier than 33% of active trials completed
- A Study of AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency NCT03378531 · started 2017 · scored riskier than 99% of active trials completed
- A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency NCT05154890 · started 2021 · scored riskier than 54% of active trials terminated
- A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency NCT02488044 · started 2016 · 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.