Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
Model scores of the 29,894 trials active now.
Model score 14% 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 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 2013-03-31, after 2010-08-31 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 2013-03-31. 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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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: subjectpotentialrandomizationeffectsprior
Phrases that lowered it: to evaluateevaluate thesafetyscreeningsubjects -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
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Disease area, neurology: Yes (raises risk) · Disease area
-
Drug's FAERS reports with a death: 0.4% (lowers risk) · Drug's FDA history
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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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Design: parallel (raises risk) · Design
-
Blinding: quadruple (lowers risk) · Design
-
Disease area, cancer: No (lowers risk) · Disease area
-
Drug's FAERS reports marked serious: 12.2% (lowers risk) · Drug's FDA history
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Disease area, psychiatry: Yes (raises risk) · Disease area
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Maximum age: 65 (raises risk) · Who can join
-
Sponsor's past trials: 17 (lowers risk) · Sponsor's track record
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Minimum age: 8 (raises 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
- quadruple (typical: none)
- Has a data monitoring committee
- Yes (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 6 (typical: 1)
Who can join
- Minimum age
- 8 (typical: 18)
- Maximum age
- 65 (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
- 17 (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
- 1
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 278 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 176 (typical: 412.5)
- Drug's FAERS reports marked serious
- 12.2% (typical: 85.6%)
- Drug's FAERS reports with a death
- 0.4% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, psychiatry, 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
- Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria NCT01977820 · started 2014 · scored riskier than 12% of active trials terminated
- Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects NCT06481709 · started 2024 · scored riskier than 1% of active trials completed
- Kuvan® in Phenylketonuria Patients Less Than 4 Years Old NCT01376908 · started 2011 · scored riskier than 15% of active trials completed
- 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
- A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension NCT04257929 · started 2020 · scored riskier than 40% 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.