Trial · NCT00332189
Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
NCT00332189, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2012-11-16
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006. Trials, record NCT00332189. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00332189
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
- NCT number
- NCT00332189
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 111 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Phenylketonuria
- Interventions
- sapropterin dihydrochloride
- First posted
- 2006-06-01
- Last updated
- 2012-11-16
- Results first posted
- 2012-11-16
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study. | Baseline through Final Visit (a Maximum of 30 months) with AEs collected at month 3 and 6 then at 6 month intervals | Safety was assessed with regards to the rate and type of AEs, clinically significant changes to vital signs and/or physical examination findings, and clinically significant changes in laboratory test results. |
| Secondary | Following Blood Phe Levels. | Baseline through Final Visit (a Maximum of 30 months) with blood phe samples taken at months 3 and 6 then at 6 month intervals | There were no pre-specified efficacy analysis, blood Phe samples were taken at each visit. Blood Phe concentrations remained within levels consistent with local clinical site recommendations for blood Phe control. |
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 | Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study. | Baseline through Final Visit (a Maximum of 30 months) with AEs collected at month 3 and 6 then at 6 month intervals | Percentage of total population who experienced an AE or SAE presented here. For full list of SAEs, and AEs experienced with a frequency of greater than 5%, see the Reported Adverse Event section. | percentage of subjects reporting events | NUMBER |
| SECONDARY | Following Blood Phe Levels. | Baseline through Final Visit (a Maximum of 30 months) with blood phe samples taken at months 3 and 6 then at 6 month intervals | The population includes all subjects who received at least one dose of study drug during the study and had at least one measurement of blood Phe level. | micromoles per liter | MEAN |
Source ClinicalTrials.gov
Results by group17
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study. | Total Patient Population | % of Subjects Reporting Any Adverse Event | 83.8 | percentage of subjects reporting events | |
| Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study. | Total Patient Population | % of Subjects Reporting Related Adverse Events | 33.3 | percentage of subjects reporting events | |
| Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study. | Total Patient Population | % of Subjects Reporting Serious Adverse Events | 6.3 | percentage of subjects reporting events | |
| Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study. | Total Patient Population | % of Subjects Reporting Related Serious AEs | 0.9 | percentage of subjects reporting events | |
| Following Blood Phe Levels. | Total Patient Population | Baseline Blood Phe Level (N=111) | 607.4 | Standard Deviation 328.44 | micromoles per liter |
| Following Blood Phe Levels. | Total Patient Population | Month 3 Blood Phe Level (N=109) | 504.6 | Standard Deviation 316.33 | micromoles per liter |
| Following Blood Phe Levels. | Total Patient Population | Month 3 Change from Baseline (N=109) | -105.7 | Standard Deviation 287.20 | micromoles per liter |
| Following Blood Phe Levels. | Total Patient Population | Month 6 Blood Phe Level (N=104) | 529.9 | Standard Deviation 332.45 | micromoles per liter |
| Following Blood Phe Levels. | Total Patient Population | Month 6 Change from Baseline (N=104) | -68.0 | Standard Deviation 310.76 | micromoles per liter |
| Following Blood Phe Levels. | Total Patient Population | Month 12 Blood Phe Level (N=97) | 494.4 | Standard Deviation 330.57 | micromoles per liter |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Total Patient Population | 111 | |
| Overall Study | COMPLETED | Total Patient Population | 90 | |
| Overall Study | NOT COMPLETED | Total Patient Population | 21 |
Source ClinicalTrials.gov
Adverse events25
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 | Bronchitis | Infections and infestations | Total Patient Population | 6 | 111 | 7 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Total Patient Population | 21 | 111 | 28 |
| other | Diarrhoea | Gastrointestinal disorders | Total Patient Population | 10 | 111 | 16 |
| other | Gastroenteritis | Infections and infestations | Total Patient Population | 7 | 111 | 7 |
| other | Gastroenteritis viral | Infections and infestations | Total Patient Population | 8 | 111 | 9 |
| other | Headache | Nervous system disorders | Total Patient Population | 13 | 111 | 48 |
| other | Influenza | Infections and infestations | Total Patient Population | 9 | 111 | 15 |
| other | Nasal congestion | Respiratory, thoracic and mediastinal disorders | Total Patient Population | 9 | 111 | 13 |
| other | Nasopharyngitis | Infections and infestations | Total Patient Population | 20 | 111 | 30 |
| other | Pharyngitis | Infections and infestations | Total Patient Population | 7 | 111 | 13 |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Total Patient Population | ||
| Total, other adverse events | Total Patient Population | 93 | 111 |
| Total, serious adverse events | Total Patient Population | 7 | 111 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 21646032 | 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 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/NCT00332189
GET https://conductscience.org/api/trials/v1/NCT00332189/events?page=2