Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Model scores of the 29,894 trials active now.
Model score 14% 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 16% of the time (257 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2018-12-31, after 2016-04-30 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 2018-12-31. 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: no earlier finished trials (lowers risk) · Sponsor's track record
-
Phase: 1/2 (raises risk) · Design
-
Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: retinalassociatedtrial ofonsettherapy
Phrases that lowered it: trialpatients withopen labelclinicalparticipated -
Number of arms: 3 (lowers risk) · Design
-
Disease area, cancer: No (lowers risk) · Disease area
-
Has a data monitoring committee: Yes (raises risk) · Design
-
Minimum age: 3 (lowers risk) · Who can join
-
Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
-
Trials the sponsor started in the prior 2 years: 0 (raises 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
-
Maximum age: not given (raises risk) · Who can join
-
Sponsor type: industry (lowers risk) · Sponsor's track record
-
Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
-
Blinding: none (raises risk) · Design
-
Responsible party: sponsor (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
- 1/2 (typical: 2)
- Number of arms
- 3 (typical: 2)
- Allocation
- non randomized (typical: randomized)
- Design
- sequential (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
- 3 (typical: 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
- 0 (typical: 32)
- Sponsor's past termination rate
- no earlier finished trials (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 0 (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
- none of the 14 tracked areas
- Drugs matched to FDA substances
- none matched
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
- Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 NCT01496040 · started 2011 · scored riskier than 73% of active trials completed
- Gene Therapy for Achromatopsia (CNGB3) NCT03001310 · started 2017 · scored riskier than 37% of active trials completed
- Gene Therapy for Achromatopsia (CNGA3) NCT03758404 · started 2019 · scored riskier than 35% of active trials completed
- Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene NCT03328130 · started 2017 · scored riskier than 33% of active trials terminated
- Gene Therapy for Blindness Caused by Choroideremia NCT01461213 · started 2011 · scored riskier than 12% 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.