Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients
Model scores of the 29,894 trials active now.
Model score 6% 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 9% of the time (803 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2023-11-18, after 2022-09-10 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 2023-11-18. 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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Design: crossover (lowers risk) · Design
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Wording of the registration (title, summary, criteria) (lowers risk) · Wording
Phrases that raised it: acutetransplantationto assessassess theenrollment
Phrases that lowered it: and safetyefficacy andpatientssafety ofthe efficacy -
Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
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Accepts healthy volunteers: No (raises risk) · Who can join
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Phase: 3 (lowers risk) · Design
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Drug's FAERS reports marked serious: 60.2% (lowers risk) · Drug's FDA history
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Has a data monitoring committee: not given (lowers risk) · Design
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Drug's FAERS reports with a death: 8.0% (lowers risk) · Drug's FDA history
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Disease area, cancer: No (lowers risk) · Disease area
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Trials the sponsor started in the prior 2 years: 6 (raises risk) · Sponsor's track record
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Maximum age: 18 (raises risk) · Who can join
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Primary purpose: treatment (raises risk) · Design
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Disease area, neurology: No (lowers risk) · Disease area
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Sponsor's past trials: 13 (lowers risk) · Sponsor's track record
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Number of arms: 2 (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
- non randomized (typical: randomized)
- Design
- crossover (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- not given (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 4 (typical: 1)
Who can join
- Minimum age
- 5 (typical: 18)
- Maximum age
- 18 (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
- 13 (typical: 32)
- Sponsor's past termination rate
- 0% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 6 (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
- 6055 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 520 (typical: 412.5)
- Drug's FAERS reports marked serious
- 60.2% (typical: 85.6%)
- Drug's FAERS reports with a death
- 8.0% (typical: 15.9%)
Disease area and drugs
- Disease areas
- metabolic, dermatology
- Drugs matched to FDA substances
- histamine, laronidase
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 JR-171 in Patients With Mucopolysaccharidosis I NCT04227600 · started 2020 · scored riskier than 16% of active trials completed
- Immune Tolerance Study With Aldurazyme® (Laronidase) NCT00741338 · started 2008 · scored riskier than 56% of active trials completed
- An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) NCT01856218 · started 2013 · scored riskier than 76% of active trials completed
- A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) NCT02230566 · started 2014 · scored riskier than 19% of active trials completed
- A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) NCT02432144 · started 2015 · scored riskier than 79% 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.