Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations
Model scores of the 29,894 trials active now.
Model score 13% 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 14% of the time (121 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Terminated on 2024-06-24, after 2023-08-15 start.
Reason given by the sponsor: “business”
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 2024-06-24. 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
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Accepts healthy volunteers: No (raises risk) · Who can join
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Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
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Disease area, neurology: Yes (raises risk) · Disease area
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Phase: 1/2 (raises risk) · Design
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Has a data monitoring committee: Yes (raises risk) · Design
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Wording of the registration (title, summary, criteria) (lowers risk) · Wording
Phrases that raised it: eyeocularthe subjectacuityvisual
Phrases that lowered it: efficacydiseasesevaluateclinicalhepatitis -
Disease area, cancer: No (lowers risk) · Disease area
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Sponsor type: academic or other (raises risk) · Sponsor's track record
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Number of arms: 1 (raises risk) · Design
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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Design: sequential (lowers risk) · Design
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Minimum age: 18 (raises risk) · Who can join
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Primary purpose: treatment (raises risk) · Design
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Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
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Trials the sponsor started in the prior 2 years: 1 (raises risk) · Sponsor's track record
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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
- 1/2 (typical: 2)
- Number of arms
- 1 (typical: 2)
- Allocation
- not applicable (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
- 18
- Maximum age
- 75 (typical: 71)
- Sex
- all
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- 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
- 1 (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, metabolic
- 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
- Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation NCT02652767 · started 2016 · scored riskier than 53% of active trials completed
- Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE) NCT02652780 · started 2016 · scored riskier than 60% of active trials completed
- NT-101 Topical Ophthalmic Solution in Patients With Wet AMD NCT06704009 · started 2025 · scored riskier than 59% of active trials completed
- Study to Assess Safety and Tolerability of PMC-403 in Subjects With Neovascular Age-related Macular Degeneration NCT05953012 · started 2023 · scored riskier than 80% of active trials completed
- 12 Week Patient Study in Neovascular Age-related Macular Degeneration (AMD) NCT01362348 · started 2011 · scored riskier than 16% 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.