Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Model scores of the 29,894 trials active now.
Model score 21% 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 8% of the time (544 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 2017-03-31, after 2013-01-31 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 2017-03-31. 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: kidney diseaseor equalequal toml mincells
Phrases that lowered it: typesafetyclinicalparticipantshealth -
Accepts healthy volunteers: No (raises risk) · Who can join
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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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Has a data monitoring committee: not given (lowers risk) · Design
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Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
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Design: single group (raises risk) · Design
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Disease area, neurology: Yes (raises risk) · Disease area
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Primary purpose: treatment (raises risk) · Design
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Maximum age: 25 (raises risk) · Who can join
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Disease area, cancer: No (lowers risk) · Disease area
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Number of arms: 1 (raises risk) · Design
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Sponsor's past trials: 0 (raises risk) · Sponsor's track record
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Minimum age: 2 (raises risk) · Who can join
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Collaborators: 0 (raises risk) · Design
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Drug's FAERS reports with a death: no FAERS history (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
- not given (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 3 (typical: 1)
Who can join
- Minimum age
- 2 (typical: 18)
- Maximum age
- 25 (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
- 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
- 0 (typical: 1)
- Drug has FAERS history
- No (typical: 64% yes)
- Drug's FAERS reports before start
- 0 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 0 (typical: 412.5)
- Drug's FAERS reports marked serious
- no FAERS history (typical: 85.6%)
- Drug's FAERS reports with a death
- no FAERS history (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic, hematology
- Drugs matched to FDA substances
- none matched
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
- VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease NCT02534844 · started 2015 · scored riskier than 67% of active trials completed
- Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) NCT02939547 · started 2017 · scored riskier than 44% of active trials completed
- Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C NCT03471143 · started 2019 · scored riskier than 96% of active trials completed
- Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 NCT03879655 · started 2019 · scored riskier than 53% of active trials terminated
- Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients NCT02912793 · started 2017 · scored riskier than 58% 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.