Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
Model scores of the 29,894 trials active now.
Model score 13% 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 21% of the time (63 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2024-10-31, after 2016-06-14 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 2024-10-31. 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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Accepts healthy volunteers: No (raises risk) · Who can join
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Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
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Blinding: quadruple (lowers risk) · Design
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Has a data monitoring committee: Yes (raises risk) · Design
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Disease area, neurology: Yes (raises risk) · Disease area
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Design: parallel (raises risk) · Design
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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Disease area, cancer: No (lowers risk) · Disease area
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Trials the sponsor started in the prior 2 years: 0 (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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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Phase: 2/3 (raises risk) · Design
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Minimum age: 2 (lowers risk) · Who can join
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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: prior tomonths priorctcentralimaging
Phrases that lowered it: and safetyagedparticipantsefficacy andparticipant -
Number of arms: 3 (lowers risk) · Design
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Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
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Sponsor type: industry (lowers risk) · Sponsor's track record
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
- 2/3 (typical: 2)
- Number of arms
- 3 (typical: 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
- 8 (typical: 1)
Who can join
- Minimum age
- 2 (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
- 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
- arimoclomol
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
- N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) NCT03759639 · started 2019 · scored riskier than 20% of active trials completed
- Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System NCT03643562 · started 2018 · scored riskier than 31% 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
- VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease NCT02534844 · started 2015 · scored riskier than 67% of active trials completed
- Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder NCT04745026 · started 2021 · scored riskier than 18% 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.