NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Model scores of the 29,894 trials active now.
Model score 11% 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 2023-07-12, after 2019-12-11 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 2023-07-12. 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
-
Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: placebodiabeteskeytransplantwithin months
Phrases that lowered it: labelparticipantopenand safetyto evaluate -
Design: crossover (lowers risk) · Design
-
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 4.3% (lowers risk) · Sponsor's track record
-
Disease area, neurology: Yes (raises risk) · Disease area
-
Phase: 3 (lowers risk) · Design
-
Drug's FAERS reports with a death: 16.7% (lowers risk) · Drug's FDA history
-
Disease area, cancer: No (lowers risk) · Disease area
-
Sponsor type: industry (lowers risk) · Sponsor's track record
-
Drug's FAERS reports marked serious: 98.1% (raises risk) · Drug's FDA history
-
Minimum age: 18 (raises risk) · Who can join
-
Has a data monitoring committee: No (lowers risk) · Design
-
Primary purpose: treatment (raises risk) · Design
-
Sponsor's past trials: 47 (lowers risk) · Sponsor's track record
-
Maximum age: 82 (raises risk) · Who can join
-
Number of arms: 2 (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
- crossover (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- No (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 4 (typical: 1)
Who can join
- Minimum age
- 18
- Maximum age
- 82 (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
- 47 (typical: 32)
- Sponsor's past termination rate
- 4.3% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 12 (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
- 108 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 106 (typical: 412.5)
- Drug's FAERS reports marked serious
- 98.1% (typical: 85.6%)
- Drug's FAERS reports with a death
- 16.7% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic
- Drugs matched to FDA substances
- eplontersen, inotersen
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 to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM) NCT04601051 · started 2020 · scored riskier than 44% of active trials completed
- Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy NCT01737398 · started 2013 · scored riskier than 37% of active trials completed
- The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis NCT00630864 · started 2008 · scored riskier than 82% of active trials completed
- APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis NCT01960348 · started 2013 · scored riskier than 85% of active trials completed
- Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ). NCT03728634 · started 2018 · scored riskier than 7% 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.