Trial · NCT01819727

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

NCT01819727, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2018-09-10

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165. Trials, record NCT01819727. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01819727

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT number: NCT01819727
Status: Completed
Phases: Phase 3
Enrollment: 261 participants, actual
Lead sponsor: BioMarin Pharmaceutical
Conditions: Phenylketonuria
Interventions: BMN 165
First posted: 2013-03-28
Last updated: 2019-02-26
Results first posted: 2018-09-10

Source ClinicalTrials.gov

Registered outcomes3

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Hypersensitivity Adverse Reaction	baseline and 36 weeks	Hypersensitivity AEs will be identified in two ways: * Broad Algorithmic anaphylactic reaction Standardized MedDRA Queries (SMQ) * Modified Hypersensitivity SMQ to include above additional preferred terms
Secondary	Blood Phenylalanine Concentration	baseline and 36 weeks	Plasma phenylalanine (Phe) concentration
Other	Dietary Phenylalanine	baseline and 36 weeks	All patients will complete a 3-day diet diary in order to assess dietary phenylalanine intake.

Source ClinicalTrials.gov

Outcomes3

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Hypersensitivity Adverse Reaction	baseline and 36 weeks	The safety population will consist of all subjects who receive any pegvaliase throughout the study duration. The safety population will be analyzed according to the treatment assignment actually received.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Blood Phenylalanine Concentration	baseline and 36 weeks	The intent-to-treat (ITT) population will consist of all subjects who are randomized to study treatment whether or not treatment was received.	umol/L	MEAN
OTHER_PRE_SPECIFIED	Dietary Phenylalanine	baseline and 36 weeks	The intent-to-treat (ITT) population will consist of all subjects who are randomized to study treatment whether or not treatment was received.	mg	MEAN

Source ClinicalTrials.gov

Results by group10

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Number of Participants With Hypersensitivity Adverse Reaction	BMN 165, 20mg/Day		111		Participants
Number of Participants With Hypersensitivity Adverse Reaction	BMN 165, 40mg/Day		119		Participants
Blood Phenylalanine Concentration	BMN 165, 20mg/Day	Baseline	1241.0	Standard Deviation 389.70	umol/L
Blood Phenylalanine Concentration	BMN 165, 40mg/Day	Baseline	1224.4	Standard Deviation 384.28	umol/L
Blood Phenylalanine Concentration	BMN 165, 20mg/Day	Week 36	868.4	Standard Deviation 501.78	umol/L
Blood Phenylalanine Concentration	BMN 165, 40mg/Day	Week 36	624.4	Standard Deviation 530.58	umol/L
Dietary Phenylalanine	BMN 165, 20mg/Day	Baseline	1750.8	Standard Deviation 1168.91	mg
Dietary Phenylalanine	BMN 165, 40mg/Day	Baseline	1647.9	Standard Deviation 1222.68	mg
Dietary Phenylalanine	BMN 165, 20mg/Day	Week 36	1851.8	Standard Deviation 1145.06	mg
Dietary Phenylalanine	BMN 165, 40mg/Day	Week 36	2057.9	Standard Deviation 1448.21	mg

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	BMN 165, 20mg/Day	131
Overall Study	STARTED	BMN 165, 40mg/Day	130
Overall Study	COMPLETED	BMN 165, 20mg/Day	111
Overall Study	COMPLETED	BMN 165, 40mg/Day	102
Overall Study	NOT COMPLETED	BMN 165, 20mg/Day	20
Overall Study	NOT COMPLETED	BMN 165, 40mg/Day	28

Source ClinicalTrials.gov

Adverse events142

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	Lymphadenopathy	Blood and lymphatic system disorders	BMN 165, 20mg/Day	12	131	21
other	Lymphadenopathy	Blood and lymphatic system disorders	BMN 165, 40mg/Day	10	130	18
other	Abdominal discomfort	Gastrointestinal disorders	BMN 165, 20mg/Day	11	131	13
other	Abdominal discomfort	Gastrointestinal disorders	BMN 165, 40mg/Day	5	130	5
other	Abdominal pain	Gastrointestinal disorders	BMN 165, 20mg/Day	9	131	10
other	Abdominal pain	Gastrointestinal disorders	BMN 165, 40mg/Day	5	130	8
other	Abdominal pain upper	Gastrointestinal disorders	BMN 165, 20mg/Day	10	131	13
other	Abdominal pain upper	Gastrointestinal disorders	BMN 165, 40mg/Day	5	130	6
other	Diarrhoea	Gastrointestinal disorders	BMN 165, 20mg/Day	13	131	16
other	Diarrhoea	Gastrointestinal disorders	BMN 165, 40mg/Day	11	130	13

1 to 10 of 142Source ClinicalTrials.gov

See all 142 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	BMN 165, 20mg/Day	1	131
Total, all-cause mortality	BMN 165, 40mg/Day	0	130
Total, other adverse events	BMN 165, 20mg/Day	130	131
Total, other adverse events	BMN 165, 40mg/Day	129	130
Total, serious adverse events	BMN 165, 20mg/Day	7	131
Total, serious adverse events	BMN 165, 40mg/Day	19	130

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
34826353	DERIVED	PubMed
40411344	DERIVED	PubMed

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 comparisons3

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
BMN 165, 20mg/Day vs BMN 165, 40mg/Day: Number of Participants With Hypersensitivity Adverse Reaction	Trial comparisons
BMN 165, 20mg/Day vs BMN 165, 40mg/Day: deaths from any cause	Trial comparisons
BMN 165, 20mg/Day vs BMN 165, 40mg/Day: serious adverse events	Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT01819727

GET https://conductscience.org/api/trials/v1/NCT01819727/events?page=2

API reference For AI assistants