Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Model scores of the 29,894 trials active now.
Model score 22% 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 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 2015-08-20, after 2013-03-26 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 2015-08-20. 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: equal toor equaltypeterm safetylong term
Phrases that lowered it: safety ofand safetyefficacy and20affect -
Sponsor's past termination rate: 46.7% (raises risk) · Sponsor's track record
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Accepts healthy volunteers: No (raises risk) · Who can join
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Disease area, neurology: Yes (raises risk) · Disease area
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Phase: 3 (lowers risk) · Design
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Has a data monitoring committee: Yes (raises risk) · Design
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Design: parallel (raises risk) · Design
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Minimum age: 7 (raises risk) · Who can join
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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Blinding: quadruple (lowers risk) · Design
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Maximum age: 16 (raises risk) · Who can join
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Sponsor's past trials: 15 (lowers risk) · Sponsor's track record
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Disease area, cancer: No (lowers risk) · Disease area
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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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Sponsor type: industry (lowers risk) · Sponsor's track record
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Collaborators: 0 (raises risk) · Design
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
- 8 (typical: 1)
Who can join
- Minimum age
- 7 (typical: 18)
- Maximum age
- 16 (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
- 15 (typical: 32)
- Sponsor's past termination rate
- 46.7% (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
- ataluren
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
- Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) NCT00592553 · started 2008 · scored riskier than 62% of active trials completed
- Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) NCT01009294 · started 2010 · scored riskier than 91% of active trials terminated
- Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) NCT00759876 · started 2008 · scored riskier than 87% of active trials terminated
- A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD） NCT04587908 · started 2020 · scored riskier than 44% of active trials completed
- An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy NCT02090959 · started 2014 · scored riskier than 62% of active trials terminated
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.