Comparison · NCT01819727-sae
BMN 165, 20mg/Day vs BMN 165, 40mg/Day: serious adverse events
Calculated by ConductScience from the counts the trial posted to ClinicalTrials.gov.
2 sourcestrial-comparisons-2026-03-23-0.1.0
Cite this record
ConductScience Library. BMN 165, 20mg/Day vs BMN 165, 40mg/Day: serious adverse events. Trial comparisons, record NCT01819727-sae. Release trial-comparisons-2026-03-23-0.1.0, 23 Mar 2026. https://conductscience.org/library/comparisons/NCT01819727-sae
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Measures6
Calculated by ConductScience from the counts the trial posted to ClinicalTrials.gov.
| Measure | Value | 95% interval, lower | 95% interval, upper | Method | Note |
|---|---|---|---|---|---|
| Risk in group 1 | 0.0534 | 0.0261 | 0.106 | wilson | |
| Risk in group 2 | 0.146 | 0.0956 | 0.217 | wilson | |
| Risk difference, group 1 minus group 2 | -0.0927 | -0.169 | -0.0197 | newcombe hybrid score | |
| Risk ratio, group 1 over group 2 | 0.366 | 0.159 | 0.84 | log | |
| Odds ratio, group 1 over group 2 | 0.33 | 0.134 | 0.814 | log | |
| Number needed to treat, for one fewer participant with a serious adverse event on BMN 165, 20mg/Day than on BMN 165, 40mg/Day | 10.8 | 5.93 | 50.9 | from the risk difference interval |
Source ClinicalTrials.gov
Groups2
Neither group, or both, names a comparator, so the groups keep ClinicalTrials.gov's order.
| Group | Title | Description | With the outcome | Analysed | ClinicalTrials.gov code |
|---|---|---|---|---|---|
| Group 1 | BMN 165, 20mg/Day | Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and \> 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study. | 7 | 131 | EG000 |
| Group 2 | BMN 165, 40mg/Day | Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and \> 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study. | 19 | 130 | EG001 |
Source ClinicalTrials.gov
Harm
- What it counts
- Total, serious adverse events
- Time frame
- Week 0- Week 36
Source ClinicalTrials.gov
Trial
- Title
- An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
- NCT number
- NCT01819727
- Phases
- PHASE3
- Conditions
- Phenylketonuria
- Interventions
- BMN 165
- Lead sponsor
- BioMarin Pharmaceutical
- Results first posted
- 2018-09-10
- Results on ClinicalTrials.gov
- https://clinicaltrials.gov/study/NCT01819727?tab=results
Source ClinicalTrials.gov
Source
- Release
- trial-comparisons-2026-03-23-0.1.0
- Release manifest SHA-256
- f66225897fbf258b57ea10bdacfd0347d3cdfda93f7f5685234b7e06ea36fa57
- AACT copy
- 2026-03-23
- Tables read
- outcome_counts.parquet, outcome_measurements.parquet, outcomes.parquet, reported_event_totals.parquet, reported_events.parquet, result_groups.parquet, studies.parquet, ctgov_trial.parquet
Source ClinicalTrials.gov
Sources 2
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions. The measures are ConductScience's calculation from them.
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