A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
Model scores of the 29,894 trials active now.
Model score 18% 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
Terminated on 2017-03-06, after 2015-05-19 start.
Reason given by the sponsor: “efficacy”
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 2017-03-06. 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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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: long termextensionassessfailurepatient
Phrases that lowered it: diseasesvisitsuspectedchildrendisorders -
Phase: 2 (raises risk) · Design
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Sponsor's past termination rate: 9.3% (raises risk) · Sponsor's track record
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Disease area, neurology: Yes (raises risk) · Disease area
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Design: single group (raises risk) · Design
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Disease area, cancer: No (lowers risk) · Disease area
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Has a data monitoring committee: No (lowers risk) · Design
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Drug's FAERS reports with a death: 14.2% (lowers risk) · Drug's FDA history
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Trials the sponsor started in the prior 2 years: 81 (lowers risk) · Sponsor's track record
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Primary purpose: treatment (raises risk) · Design
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Maximum age: 17 (raises risk) · Who can join
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Drug's FAERS reports marked serious: 96.4% (raises risk) · Drug's FDA history
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Sponsor's past trials: 366 (lowers risk) · Sponsor's track record
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Number of arms: 1 (raises risk) · Design
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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
- 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
- No (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 8 (typical: 1)
Who can join
- Minimum age
- 6 (typical: 18)
- Maximum age
- 17 (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
- 366 (typical: 32)
- Sponsor's past termination rate
- 9.3% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 81 (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
- 197 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 114 (typical: 412.5)
- Drug's FAERS reports marked serious
- 96.4% (typical: 85.6%)
- Drug's FAERS reports with a death
- 14.2% (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic, musculoskeletal
- Drugs matched to FDA substances
- cysteamine
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
- Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease NCT02023866 · started 2014 · scored riskier than 71% of active trials completed
- A Study to Assess TTI-0102 vs Placebo in MELAS Patients NCT06644534 · started 2025 · scored riskier than 15% of active trials completed
- A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease NCT03888716 · started 2019 · scored riskier than 6% of active trials completed
- A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy NCT04378075 · started 2020 · scored riskier than 56% of active trials terminated
- An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead) NCT05267574 · started 2022 · scored riskier than 98% 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.