Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
Model scores of the 29,894 trials active now.
Model score 15% 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 (201 trials).
Finished trial that started 2008–2014: scored by a model trained without any trial from its start year, so its own outcome was never used.
How it ended
Completed on 2025-03-31, after 2014-07-14 start.
The score above never used this outcome: finished trial that started 2008–2014: scored by a model trained without any trial from its start year, so its own outcome was never used.
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 2025-03-31. 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
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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: patientscancertherapymultiple sclerosiscarcinoma
Phrases that lowered it: type diabetesexaminationdiseasesclinical trialscreening -
Accepts healthy volunteers: No (raises risk) · Who can join
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Responsible party: sponsor investigator (raises risk) · Design
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Phase: 1 (lowers risk) · Design
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Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
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Design: parallel (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Disease area, neurology: Yes (raises risk) · Disease area
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Has a data monitoring committee: Yes (raises risk) · Design
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Number of arms: 4 (lowers risk) · Design
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Sponsor type: academic or other (raises risk) · Sponsor's track record
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Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
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Minimum age: 15 (raises risk) · Who can join
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Primary purpose: treatment (raises risk) · Design
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Collaborators: 1 (raises risk) · Design
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Blinding: none (raises risk) · Design
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Keywords listed: 4 (lowers 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 (typical: 2)
- Number of arms
- 4 (typical: 2)
- Allocation
- non randomized (typical: randomized)
- Design
- parallel
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- Yes (typical: 56% yes)
- Responsible party
- sponsor investigator (typical: sponsor)
- Collaborators
- 1 (typical: 0)
- Keywords listed
- 4 (typical: 1)
Who can join
- Minimum age
- 15 (typical: 18)
- Maximum age
- not given (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
- 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
- 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 & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year NCT03293524 · started 2018 · scored riskier than 47% of active trials completed
- Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations NCT05820152 · started 2023 · scored riskier than 32% of active trials terminated
- 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
- 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
- A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy NCT02693119 · started 2016 · scored riskier than 20% 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.