Trial · NCT00104247
Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
NCT00104247, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2009-04-16
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels. Trials, record NCT00104247. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00104247
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
- NCT number
- NCT00104247
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 89 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Phenylketonurias
- Interventions
- sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
- First posted
- 2005-02-25
- Last updated
- 2014-07-23
- Results first posted
- 2009-04-16
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Blood Phenylalanine Levels From Baseline to Week 6. | baseline to week 6 |
Source ClinicalTrials.gov
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Blood Phenylalanine Levels From Baseline to Week 6. | baseline to week 6 | micromole per liter | MEAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Blood Phenylalanine Levels From Baseline to Week 6. | Sapropterin Dihydrochloride | Baseline Blood Phe Level | 843 | Standard Deviation 300 | micromole per liter |
| Change in Blood Phenylalanine Levels From Baseline to Week 6. | Placebo | Baseline Blood Phe Level | 888 | Standard Deviation 323 | micromole per liter |
| Change in Blood Phenylalanine Levels From Baseline to Week 6. | Sapropterin Dihydrochloride | Week 6 Blood Phe Level | 607 | Standard Deviation 377 | micromole per liter |
| Change in Blood Phenylalanine Levels From Baseline to Week 6. | Placebo | Week 6 Blood Phe Level | 891 | Standard Deviation 348 | micromole per liter |
| Change in Blood Phenylalanine Levels From Baseline to Week 6. | Sapropterin Dihydrochloride | Mean Change in Blood Phe from Baseline to Week 6 | -239 | Standard Deviation 38 | micromole per liter |
| Change in Blood Phenylalanine Levels From Baseline to Week 6. | Placebo | Mean Change in Blood Phe from Baseline to Week 6 | 6 | Standard Deviation 36 | micromole per liter |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sapropterin Dihydrochloride | 42 | |
| Overall Study | STARTED | Placebo | 47 | |
| Overall Study | COMPLETED | Sapropterin Dihydrochloride | 41 | |
| Overall Study | COMPLETED | Placebo | 46 | |
| Overall Study | NOT COMPLETED | Sapropterin Dihydrochloride | 1 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events22
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 | Upper Respiratory Tract Infection | Infections and infestations | Sapropterin Dihydrochloride | 7 | 41 | |
| other | Upper Respiratory Tract Infection | Infections and infestations | Placebo | 13 | 47 | |
| other | Pharyngitis | Infections and infestations | Sapropterin Dihydrochloride | 2 | 41 | |
| other | Pharyngitis | Infections and infestations | Placebo | 1 | 47 | |
| other | Infection | Infections and infestations | Sapropterin Dihydrochloride | 2 | 41 | |
| other | Infection | Infections and infestations | Placebo | 0 | 47 | |
| other | Vomiting | Gastrointestinal disorders | Sapropterin Dihydrochloride | 2 | 41 | |
| other | Vomiting | Gastrointestinal disorders | Placebo | 4 | 47 | |
| other | Abdominal Pain | Gastrointestinal disorders | Sapropterin Dihydrochloride | 1 | 41 | |
| other | Abdominal Pain | Gastrointestinal disorders | Placebo | 4 | 47 |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 adverse eventsAdverse 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 | 21 | |
| Total, other adverse events | Placebo | 34 | |
| Total, serious adverse events | Sapropterin Dihydrochloride | 0 | 41 |
| Total, serious adverse events | Placebo | 0 | 47 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 17693179 | 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.
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/NCT00104247
GET https://conductscience.org/api/trials/v1/NCT00104247/events?page=2