Trial · NCT01889862
Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT01889862, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2019-07-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU. Trials, record NCT01889862. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01889862
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
- NCT number
- NCT01889862
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 215 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Phenylketonuria (PKU)
- Interventions
- BMN165 20mg/day, BMN165 40mg/day, Placebo
- First posted
- 2013-07-01
- Last updated
- 2021-05-21
- Results first posted
- 2019-07-12
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | Blood phe concentration in subjects previously exposed to BMN165 who were administered BMN165 20 or 40 mg/day compared with those who were administered a matching placebo. |
| Secondary | Change From Baseline in the Cognitive and Mood Symptoms Measured by ADHD Rating Scale (RS)-IV Inattention Subscale at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | Attention deficit hyperactivity disorder rating scale IV (ADHD RS-IV) is investigator-rated, 9-item, inattention subscale score ranges from 0 to 27, with higher scores indicative of a greater degree of impairment and a baseline score \>9 indicative of a presence of symptoms. |
| Secondary | Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 4 | At Part 4 week 57, 105, 161, 209 and 249 | The long term effect of multiple dose levels of BMN165 on blood phe concentration in subjects who are administered BMN165 using pre-filled syringes. |
| Secondary | Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS TMD at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | Phenylketonuria (PKU) Total Mood Disturbance (TMD) Profile of Mood States (POMS) PKU POMS TMD is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely). |
| Secondary | Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS Confusion Subscale at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | PKU POMS confusion mood domain (referred to as the confusion subscale) is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely). |
| Secondary | Change From Baseline in the Cognitive and Mood Symptoms Measured by POMS TMD at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | Full-length POMS TMD is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely). |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | Modified Intent-to-Treat (mITT) population consists of all subjects who reached the randomized dose of 20mg/day or 40mg/day and were randomized into Part 2 with a mean blood Phe reduction of ≥20% (using the last two consecutive blood Phe assessments of Part 1) from the baseline of 165-301 (or the Phase 2 study in which they initiated BMN165). | µmol/L | MEAN |
| SECONDARY | Change From Baseline in the Cognitive and Mood Symptoms Measured by ADHD Rating Scale (RS)-IV Inattention Subscale at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | mITT population | Units on a scale | MEAN |
| SECONDARY | Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 4 | At Part 4 week 57, 105, 161, 209 and 249 | All Subjects Entered 165-302 Part 4 Data | µmol/L | MEAN |
| SECONDARY | Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS TMD at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | mITT population | Units on a scale | MEAN |
| SECONDARY | Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS Confusion Subscale at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | mITT population | Units on a scale | MEAN |
| SECONDARY | Change From Baseline in the Cognitive and Mood Symptoms Measured by POMS TMD at Part 2 | At Part 2 baseline and change from baseline at Part 2 week 8 | mITT population | Units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group63
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | BMN165 20mg/Day | Part 2 Baseline | 596.8 | Standard Deviation 582.75 | µmol/L |
| Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | Placebo 20mg/Day | Part 2 Baseline | 563.9 | Standard Deviation 504.62 | µmol/L |
| Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | BMN165 40mg/Day | Part 2 Baseline | 410.9 | Standard Deviation 439.95 | µmol/L |
| Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | Placebo 40mg/Day | Part 2 Baseline | 508.2 | Standard Deviation 363.68 | µmol/L |
| Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | BMN165 20mg/Day | Change from Baseline Part 2 Week 8 | -65.9 | Standard Deviation 192.02 | µmol/L |
| Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | Placebo 20mg/Day | Change from Baseline Part 2 Week 8 | 996.4 | Standard Deviation 555.00 | µmol/L |
| Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | BMN165 40mg/Day | Change from Baseline Part 2 Week 8 | 114.1 | Standard Deviation 332.40 | µmol/L |
| Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 | Placebo 40mg/Day | Change from Baseline Part 2 Week 8 | 599.0 | Standard Deviation 507.40 | µmol/L |
| Change From Baseline in the Cognitive and Mood Symptoms Measured by ADHD Rating Scale (RS)-IV Inattention Subscale at Part 2 | BMN165 20mg/Day | Baseline | 5.9 | Standard Deviation 4.70 | Units on a scale |
| Change From Baseline in the Cognitive and Mood Symptoms Measured by ADHD Rating Scale (RS)-IV Inattention Subscale at Part 2 | Placebo 20mg/Day | Baseline | 5.0 | Standard Deviation 4.26 | Units on a scale |
1 to 10 of 63Source ClinicalTrials.gov
See all 63 results by groupParticipant flow70
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 (Up to 13 Weeks) | STARTED | BMN165 20mg/Day | 86 | |
| Part 1 (Up to 13 Weeks) | STARTED | Placebo 20mg/Day | 0 | |
| Part 1 (Up to 13 Weeks) | STARTED | BMN165 40mg/Day | 78 | |
| Part 1 (Up to 13 Weeks) | STARTED | Placebo 40mg/Day | 0 | |
| Part 1 (Up to 13 Weeks) | STARTED | BMN165 | 0 | |
| Part 1 (Up to 13 Weeks) | COMPLETED | BMN165 20mg/Day | 74 | |
| Part 1 (Up to 13 Weeks) | COMPLETED | Placebo 20mg/Day | 0 | |
| Part 1 (Up to 13 Weeks) | COMPLETED | BMN165 40mg/Day | 78 | |
| Part 1 (Up to 13 Weeks) | COMPLETED | Placebo 40mg/Day | 0 | |
| Part 1 (Up to 13 Weeks) | COMPLETED | BMN165 | 0 |
1 to 10 of 70Source ClinicalTrials.gov
See all 70 participant flowAdverse events2,296
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nasopharyngitis | Infections and infestations | Part 1 - 20 mg/Day | 7 | 86 | |
| other | Nasopharyngitis | Infections and infestations | Part 1 - 40 mg/Day | 11 | 78 | |
| other | Nasopharyngitis | Infections and infestations | Part 2 - 20 mg/Day Active | 4 | 34 | |
| other | Nasopharyngitis | Infections and infestations | Part 2 - 40 mg/Day Active | 1 | 32 | |
| other | Nasopharyngitis | Infections and infestations | Part 2 - 20 mg/Day Placebo | 0 | 15 | |
| other | Nasopharyngitis | Infections and infestations | Part 2 - 40 mg/Day Placebo | 1 | 14 | |
| other | Nasopharyngitis | Infections and infestations | Part 3 - Stay on Active 20 mg/Day | 2 | 23 | |
| other | Nasopharyngitis | Infections and infestations | Part 3 - Stay on Active 40 mg/Day | 0 | 18 | |
| other | Nasopharyngitis | Infections and infestations | Part 3 - Switch From Placebo to Active 20 mg/Day | 0 | 11 | |
| other | Nasopharyngitis | Infections and infestations | Part 3 - Switch From Placebo to Active 40 mg/Day | 0 | 8 |
1 to 10 of 2,296Source ClinicalTrials.gov
See all 2,296 adverse eventsAdverse event totals42
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1 - 20 mg/Day | 0 | 86 |
| Total, all-cause mortality | Part 1 - 40 mg/Day | 0 | 78 |
| Total, all-cause mortality | Part 2 - 20 mg/Day Active | 0 | 34 |
| Total, all-cause mortality | Part 2 - 40 mg/Day Active | 0 | 32 |
| Total, all-cause mortality | Part 2 - 20 mg/Day Placebo | 0 | 15 |
| Total, all-cause mortality | Part 2 - 40 mg/Day Placebo | 0 | 14 |
| Total, all-cause mortality | Part 3 - Stay on Active 20 mg/Day | 0 | 23 |
| Total, all-cause mortality | Part 3 - Stay on Active 40 mg/Day | 0 | 18 |
| Total, all-cause mortality | Part 3 - Switch From Placebo to Active 20 mg/Day | 0 | 11 |
| Total, all-cause mortality | Part 3 - Switch From Placebo to Active 40 mg/Day | 0 | 8 |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
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
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01889862
GET https://conductscience.org/api/trials/v1/NCT01889862/results?page=2