Trial · NCT00272792

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

NCT00272792, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2009-07-09

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet. Trials, record NCT00272792. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00272792

On this page

Trial

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

Title: Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
NCT number: NCT00272792
Status: Completed
Phases: Phase 3
Enrollment: 45 participants, actual
Lead sponsor: BioMarin Pharmaceutical
Conditions: Phenylketonurias
Interventions: Sapropterin Dihydrochloride, Placebo
First posted: 2006-01-09
Last updated: 2015-08-13
Results first posted: 2009-07-09

Source ClinicalTrials.gov

Registered outcomes2

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

List	Outcome	Time frame	Description
Primary	Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria	at Week 10
Secondary	Change in Phenylalanine Levels From Baseline to Week 3	Baseline to Week 3

Source ClinicalTrials.gov

Outcomes2

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria	at Week 10		mg/kg/day	MEAN
SECONDARY	Change in Phenylalanine Levels From Baseline to Week 3	Baseline to Week 3		umol/L	MEAN

Source ClinicalTrials.gov

Results by group4

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

Outcome	Group	Value	Spread	Units
Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria	Sapropterin Dihydrochloride	20.9	Standard Deviation 15.4	mg/kg/day
Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria	Placebo	2.9	Standard Deviation 4.0	mg/kg/day
Change in Phenylalanine Levels From Baseline to Week 3	Sapropterin Dihydrochloride	-147.4	Standard Error 29.0	umol/L
Change in Phenylalanine Levels From Baseline to Week 3	Placebo	-90.3	Standard Error 48.3	umol/L

Source ClinicalTrials.gov

Participant flow12

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

Period	Milestone	Group	Participants
Part 1: Responsiveness	STARTED	Sapropterin Dihydrochloride	90
Part 1: Responsiveness	STARTED	Placebo	0
Part 1: Responsiveness	COMPLETED	Sapropterin Dihydrochloride	89
Part 1: Responsiveness	COMPLETED	Placebo	0
Part 1: Responsiveness	NOT COMPLETED	Sapropterin Dihydrochloride	1
Part 1: Responsiveness	NOT COMPLETED	Placebo	0
Part 2: Blinded, Placebo Control Period	STARTED	Sapropterin Dihydrochloride	33
Part 2: Blinded, Placebo Control Period	STARTED	Placebo	12
Part 2: Blinded, Placebo Control Period	COMPLETED	Sapropterin Dihydrochloride	32
Part 2: Blinded, Placebo Control Period	COMPLETED	Placebo	9

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events60

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	Contusion	Injury, poisoning and procedural complications	Sapropterin Dihydrochloride	3	33	3
other	Contusion	Injury, poisoning and procedural complications	Placebo	1	12	1
other	Excoriation	Injury, poisoning and procedural complications	Sapropterin Dihydrochloride	2	33	3
other	Excoriation	Injury, poisoning and procedural complications	Placebo	0	12	0
other	Thermal Burn	Injury, poisoning and procedural complications	Sapropterin Dihydrochloride	0	33	0
other	Thermal Burn	Injury, poisoning and procedural complications	Placebo	1	12	1
other	Streptococcal Infection	Infections and infestations	Sapropterin Dihydrochloride	2	33	2
other	Streptococcal Infection	Infections and infestations	Placebo	2	12	2
other	Upper Respiratory Tract Infection	Infections and infestations	Sapropterin Dihydrochloride	2	33	2
other	Upper Respiratory Tract Infection	Infections and infestations	Placebo	1	12	1

1 to 10 of 60Source ClinicalTrials.gov

See all 60 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Sapropterin Dihydrochloride
Total, all-cause mortality	Placebo
Total, other adverse events	Sapropterin Dihydrochloride	26
Total, other adverse events	Placebo	8
Total, serious adverse events	Sapropterin Dihydrochloride	0	33
Total, serious adverse events	Placebo	0	12

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
19261295	DERIVED

Source 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 comparisons1

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

Comparison
Sapropterin Dihydrochloride vs Placebo: serious adverse events

Source 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/NCT00272792

GET https://conductscience.org/api/trials/v1/NCT00272792/events?page=2

API reference For AI assistants