A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
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 19% of the time (731 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
Terminated on 2016-09-30, after 2008-03-31 start.
Reason given by the sponsor: “other”
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 2016-09-30. 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: 0% (lowers risk) · Sponsor's track record
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Has a data monitoring committee: Yes (raises risk) · Design
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Design: single group (raises risk) · Design
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Phase: 2 (raises risk) · Design
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Primary purpose: treatment (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Sponsor's past trials: 8 (lowers risk) · Sponsor's track record
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Disease area, neurology: Yes (raises risk) · Disease area
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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Trials the sponsor started in the prior 2 years: 7 (raises risk) · Sponsor's track record
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Blinding: none (raises risk) · Design
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Number of arms: 1 (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
- 1 (typical: 2)
- Allocation
- not applicable (typical: randomized)
- Design
- single group (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- Yes (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 1
Who can join
- Minimum age
- 5 (typical: 18)
- Maximum age
- 16 (typical: 71)
- Sex
- male (typical: all)
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- industry (typical: academic or other)
- Sponsor's past trials
- 8 (typical: 32)
- Sponsor's past termination rate
- 0% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 7 (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, musculoskeletal
- 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
- Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) NCT01037309 · started 2009 · scored riskier than 76% of active trials completed
- Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) NCT02329769 · started 2014 · scored riskier than 75% of active trials terminated
- Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy NCT01826474 · started 2013 · scored riskier than 42% of active trials terminated
- A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy NCT02752048 · started 2016 · scored riskier than 50% of active trials completed
- Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy NCT02286947 · started 2014 · scored riskier than 78% 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.