Anaplerotic Therapy in Propionic Acidemia
Model scores of the 29,894 trials active now.
Model score 13% for any early termination, 5% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 14% of the time (201 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 2010-02-28, after 2008-07-31 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 2010-02-28. 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) (raises risk) · Wording
Phrases that raised it: alphadiagnosismuscleimproveinability
Phrases that lowered it: in patientspatients withillnesssevereobjective -
Phase: 1 (lowers risk) · Design
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Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
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Responsible party: sponsor investigator (raises risk) · Design
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Minimum age: 5 (lowers risk) · Who can join
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Design: single group (raises risk) · Design
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Has a data monitoring committee: Yes (raises risk) · Design
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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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Sponsor type: academic or other (raises risk) · Sponsor's track record
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Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
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Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
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Number of arms: 1 (raises risk) · Design
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Drug's FAERS reports with a death: 21.2% (raises risk) · Drug's FDA history
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Drug's FAERS reports before start: 132 (lowers risk) · Drug's FDA history
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Drug's FAERS reports marked serious: 84.1% (raises risk) · Drug's FDA history
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
- 1 (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
- Yes (typical: 56% yes)
- Responsible party
- sponsor investigator (typical: sponsor)
- Collaborators
- 2 (typical: 0)
- Keywords listed
- 6 (typical: 1)
Who can join
- Minimum age
- 5 (typical: 18)
- Maximum age
- 12 (typical: 71)
- Sex
- all
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- academic or other
- Sponsor's past trials
- 0 (typical: 32)
- Sponsor's past termination rate
- no earlier finished trials (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 0 (typical: 19)
Drug's FDA history
- Drugs with FDA adverse-event history
- 4 (typical: 1)
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 132 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 33 (typical: 412.5)
- Drug's FAERS reports marked serious
- 84.1% (typical: 85.6%)
- Drug's FAERS reports with a death
- 21.2% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic
- Drugs matched to FDA substances
- citric acid, glutamic acid, glutamine, ornithine
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
- Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia NCT01597440 · started 2012 · scored riskier than 85% of active trials terminated
- Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia NCT01599286 · started 2012 · scored riskier than 57% of active trials completed
- Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder NCT00345605 · started 2008 · scored riskier than 46% of active trials completed
- Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. NCT03734263 · started 2018 · scored riskier than 66% of active trials completed
- Phase 2B Efficacy/Safety of Ornithine Phenylacetate in Hospitalized Cirrhotic Patients With Hepatic Encephalopathy (STOP-HE) NCT01966419 · started 2014 · scored riskier than 47% 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.