A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
Model scores of the 29,894 trials active now.
Model score 24% for any early termination, 6% 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 (95 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 2017-11-30, after 2013-04-30 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 2017-11-30. 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: heartfailureventricularthe effectsrisk
Phrases that lowered it: safetydailypharmacokineticsdrugaffect -
Sponsor's past termination rate: 55.6% (raises risk) · Sponsor's track record
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Accepts healthy volunteers: No (raises risk) · Who can join
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Phase: 1/2 (raises risk) · Design
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Disease area, neurology: Yes (raises risk) · Disease area
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Disease area, cancer: No (lowers risk) · Disease area
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Design: single group (raises risk) · Design
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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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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Sponsor's past trials: 9 (lowers risk) · Sponsor's track record
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Maximum age: 11 (raises risk) · Who can join
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Has a data monitoring committee: No (lowers risk) · Design
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Number of arms: 1 (raises risk) · Design
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Collaborators: 0 (raises risk) · Design
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Minimum age: 7 (raises risk) · Who can join
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Blinding: none (raises risk) · Design
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Sex: male (lowers 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
- 1/2 (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
- 1
Who can join
- Minimum age
- 7 (typical: 18)
- Maximum age
- 11 (typical: 71)
- Sex
- male (typical: all)
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- industry (typical: academic or other)
- Sponsor's past trials
- 9 (typical: 32)
- Sponsor's past termination rate
- 55.6% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 3 (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, musculoskeletal
- Drugs matched to FDA substances
- givinostat
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
- Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy NCT02851797 · started 2017 · scored riskier than 70% of active trials completed
- A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) NCT01462292 · started 2011 · scored riskier than 89% of active trials completed
- A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) NCT05185622 · started 2022 · scored riskier than 38% of active trials completed
- A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy NCT01254019 · started 2010 · scored riskier than 90% of active trials completed
- A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) NCT03439670 · started 2018 · 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.