A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Model scores of the 29,894 trials active now.
Model score 8% 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
Terminated on 2024-12-26, after 2020-06-29 start.
Reason given by the sponsor: “other”
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 2024-12-26. 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: Yes (lowers risk) · Who can join
-
Sponsor's past termination rate: 14.3% (raises risk) · Sponsor's track record
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Wording of the registration (title, summary, criteria) (lowers risk) · Wording
Phrases that raised it: onsetpatientsecondaryassessdisease
Phrases that lowered it: participantparticipantsperiodonce dailymale -
Has a data monitoring committee: Yes (raises risk) · Design
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Phase: 3 (lowers 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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Design: parallel (raises risk) · Design
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Disease area, neurology: Yes (raises risk) · Disease area
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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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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Blinding: quadruple (lowers risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Collaborators: 0 (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
- 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
- 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
- Yes (typical: 9% yes)
Sponsor's track record
- Sponsor type
- industry (typical: academic or other)
- Sponsor's past trials
- 231 (typical: 32)
- Sponsor's past termination rate
- 14.3% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 10 (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
- venglustat
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
- N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) NCT03759665 · started 2019 · scored riskier than 21% of active trials completed
- Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 NCT03485677 · started 2018 · scored riskier than 47% of active trials completed
- Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease NCT02228460 · started 2014 · scored riskier than 59% of active trials completed
- Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease NCT05758922 · started 2023 · scored riskier than 18% of active trials completed
- A 2-Part Trial in Subjects With Type 2 Diabetes and in Healthy Subjects to Evaluate GSK1614235, a New Glucose Lowering Drug to Treat Type 2 Diabetes NCT00976261 · started 2009 · scored riskier than 3% 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.