Trial · NCT00355264
Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
NCT00355264, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2020-09-25
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency. Trials, record NCT00355264. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00355264
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
- NCT number
- NCT00355264
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Tetrahydrobiopterin Deficiencies, Hyperphenylalaninemia, Non-Phenylketonuric
- Interventions
- Phenoptin
- First posted
- 2006-07-21
- Last updated
- 2020-09-25
- Results first posted
- 2020-09-25
- Limitations and caveats
Due to the small sample size in this study, a single patient AE exceeded the 5% maximum AE reporting threshold. Many of these manifestations are due to primary BH4 deficiency disease and the associated inadequate neurotransmitters.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | At Baseline, Week 4 through Extension Week 130 | Baseline blood phenylalanine(Phe) value is the latest measurement taken prior to initiation of Phenoptin treatment. The ideal range for blood Phe levels is approximately 120-360 µmol/L. |
| Primary | Percentage of Subjects With Blood Phenylalanine (Last Observation Carried Forward) < 360 μmol/L | At Baseline, Week 4 through Extension Week 130 | Baseline blood phenylalanine(Phe) value is the latest measurement taken prior to initiation of Phenoptin treatment. The objective of this outcome was to compare to Phe levels achieved using previous treatment regimens. |
| Secondary | Subjects Experiencing Adverse Events(AEs) | Up to 35 Months | Intensity was determined by the Investigator. For symptomatic AEs the following definitions were applied. Mild = AE did not limit usual activities. Moderate = AE resulted in some limitation of usual activities. Severe = AE resulted in an inability to carry out usual activities. A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. Serious AE (SAE) resulted in death, was life-threatening, required in patient hospitalization or prolongation of an existing hospitalization, resulted in persistent or significant disability or incapacity, is a congenital anomaly or birth defect; or was an important medical event. |
| Other | AEs Consistent With Signs and/or Symptoms of BH4 Deficiency | Up to 35 months | Attention was paid to AEs that may be consistent with signs and/or symptoms associated with primary BH4 deficiency to determine if such signs and symptoms arose or increased in severity or frequency during the study. These included prematurity and low birth weight, inability to control body temperature, low muscle tone, decreased spontaneous movements, poor sucking, movement disorders, difficulty swallowing, hypersalivation, seizures or convulsions, behavioral problems, developmental delay, and mental retardation. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | At Baseline, Week 4 through Extension Week 130 | Efficacy analysis population are the subjects received at least 1 dose of Phenoptin and has at least 1 post-treatment measurement of blood Phe. | μmol/L | MEAN |
| PRIMARY | Percentage of Subjects With Blood Phenylalanine (Last Observation Carried Forward) < 360 μmol/L | At Baseline, Week 4 through Extension Week 130 | Efficacy analysis population are the subjects received at least 1 dose of Phenoptin and has at least 1 post-treatment measurement of blood Phe. | percentage of participants | NUMBER |
| SECONDARY | Subjects Experiencing Adverse Events(AEs) | Up to 35 Months | participants | NUMBER | |
| OTHER_PRE_SPECIFIED | AEs Consistent With Signs and/or Symptoms of BH4 Deficiency | Up to 35 months | AEs that are signs and symptoms of BH4 deficiency were reported for 3 subjects: One SAE of mild dyskinesia (required hospitalization) in one subject; events of severe dystonia and moderate Dyskinesia in one subject; and one event of mild vertigo in one subject. All events resolved and were considered unrelated to Phenoptin except mild vertigo. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group72
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | All Subjects | Baseline | 132.9 | Standard Deviation 134.7 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | Subgroup: Subjects With Synthesis Enzymatic Defect | Baseline | 72.2 | Standard Deviation 13.4 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | Subgroup: Subjects With Recycling Enzyme Defect | Baseline | 315.0 | Standard Deviation 180.9 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | All Subjects | Part 2: Week 4 | 104.1 | Standard Deviation 66.7 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | Subgroup: Subjects With Synthesis Enzymatic Defect | Part 2: Week 4 | 78.2 | Standard Deviation 28.4 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | Subgroup: Subjects With Recycling Enzyme Defect | Part 2: Week 4 | 181.7 | Standard Deviation 96.1 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | All Subjects | Part 2: Week 6 | 120.8 | Standard Deviation 119.0 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | Subgroup: Subjects With Synthesis Enzymatic Defect | Part 2: Week 6 | 70.2 | Standard Deviation 17.2 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | Subgroup: Subjects With Recycling Enzyme Defect | Part 2: Week 6 | 272.7 | Standard Deviation 175.1 | μmol/L |
| Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints | All Subjects | Part 2: Week 8 | 112.3 | Standard Deviation 106.8 | μmol/L |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 (2 Weeks) | STARTED | Phenoptin | 12 | |
| Part 1 (2 Weeks) | COMPLETED | Phenoptin | 12 | |
| Part 1 (2 Weeks) | NOT COMPLETED | Phenoptin | 0 | |
| Part 2 (8 Weeks) | STARTED | Phenoptin | 12 | |
| Part 2 (8 Weeks) | COMPLETED | Phenoptin | 12 | |
| Part 2 (8 Weeks) | NOT COMPLETED | Phenoptin | 0 | |
| Part 3 (7 Weeks of Extension) | STARTED | Phenoptin | 2 | |
| Part 3 (7 Weeks of Extension) | COMPLETED | Phenoptin | 2 | |
| Part 3 (7 Weeks of Extension) | NOT COMPLETED | Phenoptin | 0 | |
| Extension (Variable Duration) | STARTED | Phenoptin | 12 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events77
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 | Neutropenia | Blood and lymphatic system disorders | Phenoptin | 1 | 12 | |
| other | Vertigo | Ear and labyrinth disorders | Phenoptin | 1 | 12 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Phenoptin | 2 | 12 | |
| other | Constipation | Gastrointestinal disorders | Phenoptin | 1 | 12 | |
| other | Diarrhoea | Gastrointestinal disorders | Phenoptin | 7 | 12 | |
| other | Dyspepsia | Gastrointestinal disorders | Phenoptin | 1 | 12 | |
| other | Nausea | Gastrointestinal disorders | Phenoptin | 1 | 12 | |
| other | Vomiting | Gastrointestinal disorders | Phenoptin | 4 | 12 | |
| other | Fatigue | General disorders | Phenoptin | 2 | 12 | |
| other | Irritability | General disorders | Phenoptin | 1 | 12 |
1 to 10 of 77Source ClinicalTrials.gov
See all 77 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Phenoptin | 0 | 12 |
| Total, other adverse events | Phenoptin | 12 | 12 |
| Total, serious adverse events | Phenoptin | 2 | 12 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT00355264
GET https://conductscience.org/api/trials/v1/NCT00355264/results?page=2