A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
Model scores of the 29,894 trials active now.
Model score 12% 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 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 2011-03-31, after 2009-04-30 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 2011-03-31. 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
-
Accepts healthy volunteers: No (raises risk) · Who can join
-
Wording of the registration (title, summary, criteria) (lowers risk) · Wording
Phrases that raised it: celltherapystemprioriv
Phrases that lowered it: efficacysubjectsscreeningsafetyevaluate the -
Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
-
Phase: 1/2 (raises risk) · Design
-
Design: single group (raises risk) · Design
-
Disease area, cancer: No (lowers risk) · Disease area
-
Maximum age: 18 (raises risk) · Who can join
-
Trials the sponsor started in the prior 2 years: 6 (raises risk) · Sponsor's track record
-
Has a data monitoring committee: No (lowers risk) · Design
-
Minimum age: 5 (raises risk) · Who can join
-
Disease area, dermatology: Yes (raises risk) · Disease area
-
Number of arms: 1 (raises risk) · Design
-
Sponsor type: industry (lowers risk) · Sponsor's track record
-
Primary purpose: treatment (raises risk) · Design
-
Keywords listed: 13 (raises risk) · Design
-
Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
-
Disease area, neurology: No (lowers risk) · Disease area
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
- 1 (typical: 2)
- Allocation
- non randomized (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
- 13 (typical: 1)
Who can join
- Minimum age
- 5 (typical: 18)
- Maximum age
- 18 (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
- 11 (typical: 32)
- Sponsor's past termination rate
- 0% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 6 (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, dermatology
- 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 BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) NCT01515956 · started 2011 · scored riskier than 44% of active trials completed
- A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) NCT01275066 · started 2011 · scored riskier than 30% of active trials completed
- Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) NCT01415427 · started 2011 · scored riskier than 43% of active trials completed
- A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) NCT01242111 · started 2010 · scored riskier than 70% of active trials terminated
- BMN 110 Phase 3B in Australian Patients NCT01966029 · started 2013 · scored riskier than 51% 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.