Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
Model scores of the 29,894 trials active now.
Model score 17% for any early termination, 3% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 10% of the time (139 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2023-04-18, after 2021-07-19 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 2023-04-18. 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: 14.9% (raises risk) · Sponsor's track record
-
Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: cellfailurepatientsdiseasetherapy
Phrases that lowered it: participantparticipantsand safetyefficacy andsingle -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Phase: 3 (lowers risk) · Design
-
Sponsor's past trials: 1394 (raises risk) · Sponsor's track record
-
Trials the sponsor started in the prior 2 years: 139 (lowers risk) · Sponsor's track record
-
Design: single group (raises risk) · Design
-
Has a data monitoring committee: Yes (raises risk) · Design
-
Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
-
Minimum age: 18 (raises risk) · Who can join
-
Sponsor type: industry (lowers risk) · Sponsor's track record
-
Primary purpose: treatment (raises risk) · Design
-
Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
-
Disease area, cancer: No (lowers risk) · Disease area
-
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
- 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
- 5 (typical: 1)
Who can join
- Minimum age
- 18
- Maximum age
- not given (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
- 1394 (typical: 32)
- Sponsor's past termination rate
- 14.9% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 139 (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
- hematology
- Drugs matched to FDA substances
- iptacopan
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
- Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. NCT05630001 · started 2023 · scored riskier than 40% of active trials completed
- A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy NCT06799546 · started 2025 · scored riskier than 10% of active trials completed
- Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT02598583 · started 2015 · scored riskier than 20% of active trials completed
- Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT03439839 · started 2018 · scored riskier than 82% of active trials completed
- BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy NCT05116787 · started 2021 · scored riskier than 70% 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.