N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
Model scores of the 29,894 trials active now.
Model score 11% 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 19% of the time (731 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Completed on 2023-01-09, after 2019-06-07 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 2023-01-09. 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: extensiondlpatientarthritistherapy
Phrases that lowered it: maleefficacyclinicalparentvisit -
Accepts healthy volunteers: No (raises risk) · Who can join
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Phase: 2 (raises 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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Blinding: single (lowers risk) · Design
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Disease area, neurology: Yes (raises risk) · Disease area
-
Design: single group (raises risk) · Design
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Has a data monitoring committee: Yes (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Drug's FAERS reports with a death: 14.8% (lowers risk) · Drug's FDA history
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Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
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Primary purpose: treatment (raises risk) · Design
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
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Number of arms: 2 (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
- 2
- Number of arms
- 2
- Allocation
- non randomized (typical: randomized)
- Design
- single group (typical: parallel)
- Primary purpose
- treatment
- Blinding
- single (typical: 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
- 6 (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
- 2 (typical: 1)
- Drug has FAERS history
- Yes (typical: 64% yes)
- Drug's FAERS reports before start
- 757 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 125 (typical: 412.5)
- Drug's FAERS reports marked serious
- 90.4% (typical: 85.6%)
- Drug's FAERS reports with a death
- 14.8% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic
- Drugs matched to FDA substances
- acetylleucine, leucine
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
- N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) NCT03759639 · started 2019 · scored riskier than 20% of active trials completed
- Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease NCT05758922 · started 2023 · scored riskier than 18% of active trials completed
- N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) NCT03759678 · started 2020 · scored riskier than 21% of active trials terminated
- A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 NCT04221451 · started 2020 · scored riskier than 11% of active trials terminated
- Safety, Pharmacokinetics and Pharmacodynamics Study With 2B3-201 in Healthy Subjects and Multiple Sclerosis(MS) Patients NCT02048358 · started 2013 · scored riskier than 2% 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.