Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
Model scores of the 29,894 trials active now.
Model score 11% 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 14% of the time (493 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2025-01-21, after 2018-09-16 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 2025-01-21. 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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Accepts healthy volunteers: No (raises risk) · Who can join
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Sponsor's past termination rate: 2.2% (lowers risk) · Sponsor's track record
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Phase: 3 (lowers risk) · Design
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Disease area, neurology: Yes (raises risk) · Disease area
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Design: single group (raises risk) · Design
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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Wording of the registration (title, summary, criteria) (lowers risk) · Wording
Phrases that raised it: long termterm safetythe longand efficacyextension study
Phrases that lowered it: participantsparticipantmaleoralevaluate the -
Trials the sponsor started in the prior 2 years: 19 (lowers risk) · Sponsor's track record
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Has a data monitoring committee: No (lowers 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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Sponsor's past trials: 93 (lowers risk) · Sponsor's track record
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Disease area, cancer: No (lowers risk) · Disease area
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Primary purpose: treatment (raises risk) · Design
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Minimum age: 18 (raises risk) · Who can join
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Disease area, cardiovascular: Yes (raises risk) · Disease area
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Number of arms: 1 (raises 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
- 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
- 5 (typical: 1)
Who can join
- Minimum age
- 18
- Maximum age
- 60 (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
- 93 (typical: 32)
- Sponsor's past termination rate
- 2.2% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 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
- cardiovascular, neurology, metabolic, urogenital
- Drugs matched to FDA substances
- pegunigalsidase 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
- Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients NCT03614234 · started 2018 · scored riskier than 39% of active trials completed
- Extension Study of PRX-102 for up to 60 Months NCT01981720 · started 2014 · scored riskier than 39% of active trials completed
- Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks NCT03180840 · started 2017 · scored riskier than 42% of active trials completed
- Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) NCT03018730 · started 2017 · scored riskier than 66% of active trials completed
- Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients NCT01678898 · started 2012 · scored riskier than 48% 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.