Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia
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 8% of the time (60 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Terminated on 2023-01-10, after 2021-05-29 start.
Reason given by the sponsor: “other”
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 2023-01-10. 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: no earlier finished trials (lowers risk) · Sponsor's track record
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Phase: 1/2 (raises risk) · Design
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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: insulinmonthskidneydialysisto assess
Phrases that lowered it: participantparticipantspatients12 yearssafety -
Number of arms: 5 (lowers 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: no FAERS history (raises risk) · Drug's FDA history
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Minimum age: 0.5 (lowers risk) · Who can join
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Maximum age: 12 (raises risk) · Who can join
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Drug's FAERS reports marked serious: no FAERS history (raises 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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Design: sequential (lowers risk) · Design
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Primary purpose: treatment (raises risk) · Design
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Sponsor's past trials: 0 (lowers 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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Collaborators: 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
- 1/2 (typical: 2)
- Number of arms
- 5 (typical: 2)
- Allocation
- non randomized (typical: randomized)
- Design
- sequential (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- Yes (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 1 (typical: 0)
- Keywords listed
- 6 (typical: 1)
Who can join
- Minimum age
- 0.5 (typical: 18)
- Maximum age
- 12 (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
- 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
- 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
- Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia NCT04732429 · started 2021 · scored riskier than 17% of active trials terminated
- A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia NCT04899310 · started 2021 · scored riskier than 16% of active trials terminated
- Ataluren for Nonsense Mutation Methylmalonic Acidemia NCT01141075 · started 2010 · scored riskier than 64% of active trials terminated
- Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD NCT00681811 · started 2008 · scored riskier than 61% of active trials terminated
- Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency NCT05222178 · started 2022 · scored riskier than 22% 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.