Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations
Model scores of the 29,894 trials active now.
Model score 10% 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 5% of the time (4,470 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 2012-08-31, after 2009-11-30 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 2012-08-31. 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) (lowers risk) · Wording
Phrases that raised it: functionof agecirrhosisyears oftherapy
Phrases that lowered it: screeningsubjectsdayoralcontaining -
Accepts healthy volunteers: No (raises risk) · Who can join
-
Sponsor's past termination rate: 55.6% (raises risk) · Sponsor's track record
-
Phase: 1 (lowers risk) · Design
-
Drug's FAERS reports with a death: 5.5% (lowers risk) · Drug's FDA history
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Design: single group (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
-
Sponsor type: industry (lowers risk) · Sponsor's track record
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Has a data monitoring committee: No (lowers risk) · Design
-
Number of arms: not given (lowers risk) · Design
-
Minimum age: 5 (raises risk) · Who can join
-
Sponsor's past trials: 9 (lowers risk) · Sponsor's track record
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Drug's FAERS reports marked serious: 70.3% (lowers risk) · Drug's FDA history
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Blinding: none (lowers risk) · Design
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Primary purpose: treatment (raises risk) · Design
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Disease area, neurology: No (lowers risk) · Disease area
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Maximum age: 65 (lowers risk) · Who can join
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
- not given (typical: 2)
- Allocation
- not applicable (typical: randomized)
- Design
- single group (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- No (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 0 (typical: 1)
Who can join
- Minimum age
- 5 (typical: 18)
- Maximum age
- 65 (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
- 9 (typical: 32)
- Sponsor's past termination rate
- 55.6% (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
- 1
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 91 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 24 (typical: 412.5)
- Drug's FAERS reports marked serious
- 70.3% (typical: 85.6%)
- Drug's FAERS reports with a death
- 5.5% (typical: 15.9%)
Disease area and drugs
- Disease areas
- none of the 14 tracked areas
- Drugs matched to FDA substances
- retinol
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
- Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01) NCT01521793 · started 2012 · scored riskier than 35% of active trials completed
- Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65) NCT01543906 · started 2012 · scored riskier than 15% of active trials completed
- Evaluation of the Effects of Oral QLT091001 in Adults With Impaired Dark Adaptation NCT01999764 · started 2013 · scored riskier than 31% of active trials completed
- Extension Study to Study PQ-110-001 (NCT03140969) NCT03913130 · started 2019 · scored riskier than 98% of active trials terminated
- Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene NCT03140969 · started 2017 · scored riskier than 24% 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.