An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
Model scores of the 29,894 trials active now.
Model score 19% 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 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
Terminated on 2018-06-12, after 2014-03-20 start.
Reason given by the sponsor: “business”
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 2018-06-12. 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: conditionparentthe safetydystrophydmd
Phrases that lowered it: participantsparticipantsafety ofclinical trialmale -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 43.8% (raises risk) · Sponsor's track record
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Phase: 3 (lowers risk) · Design
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Disease area, neurology: Yes (raises risk) · Disease area
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Has a data monitoring committee: Yes (raises risk) · Design
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Design: single group (raises risk) · Design
-
Trials the sponsor started in the prior 2 years: 5 (lowers risk) · Sponsor's track record
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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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Disease area, cancer: No (lowers risk) · Disease area
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Sponsor's past trials: 16 (lowers risk) · Sponsor's track record
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Primary purpose: treatment (raises risk) · Design
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Blinding: none (raises risk) · Design
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Disease area, musculoskeletal: Yes (raises risk) · Disease area
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Responsible party: sponsor (lowers 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
- 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
- Collaborators
- 0
- Keywords listed
- 8 (typical: 1)
Who can join
- Minimum age
- 7 (typical: 18)
- Maximum age
- 15 (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
- 16 (typical: 32)
- Sponsor's past termination rate
- 43.8% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 5 (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 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) NCT00759876 · started 2008 · scored riskier than 87% of active trials terminated
- Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) NCT00847379 · started 2009 · scored riskier than 99% of active trials terminated
- Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada NCT01557400 · started 2012 · scored riskier than 69% of active trials completed
- Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) NCT01247207 · started 2010 · scored riskier than 76% of active trials completed
- A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren NCT03796637 · started 2019 · scored riskier than 49% 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.