Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Model scores of the 29,894 trials active now.
Model score 21% for any early termination, 4% 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 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 2013-05-25, after 2012-03-02 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 2013-05-25. 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: patientpatientsbonetherapydisease
Phrases that lowered it: japanesescreeningsafety ofhepatitisopen label -
Accepts healthy volunteers: No (raises risk) · Who can join
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Phase: 3 (lowers risk) · Design
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Design: single group (raises risk) · Design
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Drug's FAERS reports with a death: 23.1% (raises risk) · Drug's FDA history
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Trials the sponsor started in the prior 2 years: 57 (lowers risk) · Sponsor's track record
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Sponsor's past termination rate: 7.4% (lowers risk) · Sponsor's track record
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Disease area, neurology: Yes (raises risk) · Disease area
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Disease area, cancer: No (lowers risk) · Disease area
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Drug's FAERS reports marked serious: 97.4% (raises risk) · Drug's FDA history
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Has a data monitoring committee: No (lowers risk) · Design
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Primary purpose: treatment (raises risk) · Design
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Sponsor's past trials: 122 (lowers risk) · Sponsor's track record
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Responsible party: sponsor (lowers risk) · Design
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Maximum age: not given (raises risk) · Who can join
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Collaborators: 0 (raises risk) · Design
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Disease area, metabolic: Yes (lowers risk) · Disease area
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
- 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
- 0 (typical: 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
- 122 (typical: 32)
- Sponsor's past termination rate
- 7.4% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 57 (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
- 39 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 24 (typical: 412.5)
- Drug's FAERS reports marked serious
- 97.4% (typical: 85.6%)
- Drug's FAERS reports with a death
- 23.1% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic
- Drugs matched to FDA substances
- velaglucerase alfa
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
- Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease NCT01842841 · started 2013 · scored riskier than 60% of active trials completed
- Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease NCT01685216 · started 2012 · scored riskier than 84% of active trials completed
- A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease NCT05529992 · started 2023 · scored riskier than 57% of active trials completed
- Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease NCT00553631 · started 2008 · scored riskier than 17% of active trials completed
- An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease NCT00635427 · started 2008 · scored riskier than 40% 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.