Open Label Study in Adolescents and Children With Myotonic Disorders
Model scores of the 29,894 trials active now.
Model score 12% 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 in 2015 or later: the model never saw it during training.
How it ended
Completed on 2024-08-08, after 2021-09-03 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 2024-08-08. 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
-
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
-
Phase: 3 (lowers risk) · Design
-
Disease area, neurology: Yes (raises risk) · Disease area
-
Has a data monitoring committee: Yes (raises risk) · Design
-
Design: single group (raises risk) · Design
-
Drug's FAERS reports with a death: 16.9% (lowers risk) · Drug's FDA history
-
Disease area, cancer: No (lowers risk) · Disease area
-
Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: heartblockconcurrentintervalms
Phrases that lowered it: open labelan openpharmacokineticspatientssteady state -
Primary purpose: treatment (raises risk) · Design
-
Sponsor type: industry (lowers risk) · Sponsor's track record
-
Number of arms: 1 (raises risk) · Design
-
Trials the sponsor started in the prior 2 years: 2 (raises risk) · Sponsor's track record
-
Maximum age: 18 (raises 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
- 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
- 0 (typical: 1)
Who can join
- Minimum age
- 6 (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
- 5 (typical: 32)
- Sponsor's past termination rate
- 0% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 2 (typical: 19)
Drug's FDA history
- Drugs with FDA adverse-event history
- 1
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 1452 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 242 (typical: 412.5)
- Drug's FAERS reports marked serious
- 85.8% (typical: 85.6%)
- Drug's FAERS reports with a death
- 16.9% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, musculoskeletal
- Drugs matched to FDA substances
- mexiletine
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
- A Study to Investigate the Effect of Single Dose of AZD6094 (600 mg) on Cardiac Repolarization in Healthy Volunteers NCT03258515 · started 2017 · scored riskier than 2% of active trials completed
- Mexiletine in Sporadic Amyotrophic Lateral Sclerosis NCT02781454 · started 2016 · scored riskier than 53% of active trials completed
- Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia NCT00832000 · started 2008 · scored riskier than 59% of active trials completed
- Study to Assess the Effect of Macitentan on the Electrocardiogram (ECG) in Healthy Male and Female Subjects NCT02050802 · started 2011 · scored riskier than 3% of active trials completed
- A Study of Milademetan Administration on Cardiac Repolarization in Healthy Subjects NCT05758818 · started 2023 · scored riskier than 9% 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.