Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
Model scores of the 29,894 trials active now.
Model score 10% 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 2018-09-14, after 2009-02-28 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 2018-09-14. 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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Wording of the registration (title, summary, criteria) (lowers risk) · Wording
Phrases that raised it: prioragentconcurrentstandardgrowth
Phrases that lowered it: subjectsevaluate theto evaluatescreeningsafety -
Sponsor's past termination rate: 0% (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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Drug's FAERS reports with a death: 2.5% (lowers risk) · Drug's FDA history
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Design: single group (raises risk) · Design
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Minimum age: not given (raises risk) · Who can join
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Trials the sponsor started in the prior 2 years: 5 (raises risk) · Sponsor's track record
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Drug's FAERS reports marked serious: 7.5% (lowers risk) · Drug's FDA history
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Disease area, cancer: No (lowers risk) · Disease area
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Has a data monitoring committee: No (lowers risk) · Design
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Maximum age: 6 (raises risk) · Who can join
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Primary purpose: treatment (raises risk) · Design
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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
- 7 (typical: 1)
Who can join
- Minimum age
- not given (typical: 18)
- Maximum age
- 6 (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
- 10 (typical: 32)
- Sponsor's past termination rate
- 0% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 5 (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
- 40 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 40 (typical: 412.5)
- Drug's FAERS reports marked serious
- 7.5% (typical: 85.6%)
- Drug's FAERS reports with a death
- 2.5% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic
- Drugs matched to FDA substances
- sapropterin
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
- Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria NCT01732471 · started 2012 · scored riskier than 40% of active trials completed
- Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response NCT00841100 · started 2008 · scored riskier than 28% of active trials completed
- Kuvan® in Phenylketonuria Patients Less Than 4 Years Old NCT01376908 · started 2011 · scored riskier than 15% of active trials completed
- Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU NCT03694353 · started 2018 · scored riskier than 73% of active trials completed
- Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) NCT01212744 · started 2011 · scored riskier than 21% 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.