Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

NCT01557400 on ClinicalTrials.gov Phase 3 PTC Therapeutics (industry) Started 2012-05-20 Completed

Riskier than 69% of active trials

Model scores of the 29,894 trials active now.

Model score 20% 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 (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 2018-01-19, after 2012-05-20 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 2018-01-19. 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

Sponsor's past termination rate: 46.7% (raises risk) · Sponsor's track record
Accepts healthy volunteers: No (raises risk) · Who can join
Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: administrationinvestigational drugprovideabilitynote
Phrases that lowered it: participantsparticipanttrialreceivedsafety of
Phase: 3 (lowers risk) · Design
Disease area, neurology: Yes (raises risk) · Disease area
Design: single group (raises risk) · Design
Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
Disease area, cancer: No (lowers risk) · Disease area
Has a data monitoring committee: No (lowers risk) · Design
Primary purpose: treatment (raises risk) · Design
Minimum age: not given (raises risk) · Who can join
Sponsor type: industry (lowers risk) · Sponsor's track record
Blinding: none (raises risk) · Design
Maximum age: not given (raises risk) · Who can join
Collaborators: 0 (raises risk) · Design
Sponsor's past trials: 15 (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: 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: No (typical: 56% yes)
Responsible party: sponsor
Collaborators: 0
Keywords listed: 10 (typical: 1)

Who can join

Minimum age: not given (typical: 18)
Maximum age: not given (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

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) NCT01247207 · started 2010 · scored riskier than 76% 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
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) NCT00759876 · started 2008 · scored riskier than 87% of active trials terminated
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 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) NCT00847379 · started 2009 · scored riskier than 99% 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.