A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
Model scores of the 29,894 trials active now.
Model score 18% for any early termination, 2% 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 2021-01-15, after 2018-12-04 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 2021-01-15. 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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Sponsor's past termination rate: 20% (raises risk) · Sponsor's track record
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Accepts healthy volunteers: No (raises risk) · Who can join
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Has a data monitoring committee: Yes (raises risk) · Design
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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: parallel (raises risk) · Design
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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: diseasesubjectsubjects withthe followingdefined
Phrases that lowered it: healthyand safetyefficacy andmalesafety of -
Sponsor type: industry (lowers risk) · Sponsor's track record
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Primary purpose: treatment (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Drug's FAERS reports with a death: 18.9% (lowers risk) · Drug's FDA history
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Drug's FAERS reports marked serious: 72.6% (lowers risk) · Drug's FDA history
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Minimum age: 18 (raises risk) · Who can join
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Blinding: double (raises risk) · Design
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Sponsor's past trials: 20 (lowers risk) · Sponsor's track record
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Responsible party: sponsor (lowers risk) · Design
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Maximum age: not given (lowers 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
- 3 (typical: 2)
- Number of arms
- 2
- Allocation
- randomized
- Design
- parallel
- Primary purpose
- treatment
- Blinding
- double (typical: none)
- Has a data monitoring committee
- Yes (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 2 (typical: 1)
Who can join
- Minimum age
- 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
- 20 (typical: 32)
- Sponsor's past termination rate
- 20% (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
- 2 (typical: 1)
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 2294 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 321 (typical: 412.5)
- Drug's FAERS reports marked serious
- 72.6% (typical: 85.6%)
- Drug's FAERS reports with a death
- 18.9% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic
- Drugs matched to FDA substances
- alglucosidase alfa, cipaglucosidase alfa, miglustat
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
- First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 NCT02675465 · started 2016 · scored riskier than 19% of active trials completed
- A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) NCT04138277 · started 2019 · scored riskier than 78% of active trials completed
- BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) NCT01924845 · started 2014 · scored riskier than 40% of active trials terminated
- Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease NCT00688597 · started 2008 · scored riskier than 8% of active trials terminated
- Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease NCT02782741 · started 2016 · scored riskier than 34% 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.