Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
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ClinicalTrials.gov Identifier: NCT00104247 |
Recruitment Status :
Completed
First Posted : February 25, 2005
Results First Posted : April 16, 2009
Last Update Posted : July 23, 2014
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Phenylketonurias | Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 89 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double |
Primary Purpose: | Treatment |
Official Title: | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels |
Study Start Date : | March 2005 |
Actual Study Completion Date : | February 2006 |

- Change in Blood Phenylalanine Levels From Baseline to Week 6. [ Time Frame: baseline to week 6 ]

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Ages Eligible for Study: | 8 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 8 years of age and older
- Received at least 7 out of 8 scheduled doses in Study PKU 001
- Responsive to Phenoptin™ in Study PKU-001, defined as a reduction in blood Phenylalanine level of >/=30% compared with baseline
- Blood Phenylalanine level >/=450 μmol/L at screening
- Willing and able to provide written informed consent or, in the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained
- Negative urine pregnancy test at screening (females of child-bearing potential)
- Male and Female subjects of childbearing potential (if sexually active) must be using acceptable birth control measures, as determined by the investigator, and willing to continue to use acceptable birth control measures while participating in the study
- Willing and able to comply with study procedures
- Willing to continue current diet unchanged while participating in the study
Exclusion Criteria:
- Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable
- Use of any investigational agent other than Phenoptin™ within 30 days prior to screening, or requirement for any investigational agent or investigational vaccine prior to completion of all scheduled study assessments
- Pregnant or breastfeeding, or considering pregnancy
- ALT >5 times the upper limit of normal (i.e., Grade 3 or higher based on World Health Organization Toxicity Criteria) at screening
- Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes, or organ transplantation recipient)
- Serious neuropsychiatric illness (e.g., major depression) not currently under medical management
- Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
- Concurrent use of levodopa
- Clinical diagnosis of primary BH4 deficiency

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00104247
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Dallas, Texas, United States | |
United States, Utah | |
Salt Lake City, Utah, United States | |
United States, Wisconsin | |
Madison, Wisconsin, United States |
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
ClinicalTrials.gov Identifier: | NCT00104247 History of Changes |
Other Study ID Numbers: |
PKU-003 |
First Posted: | February 25, 2005 Key Record Dates |
Results First Posted: | April 16, 2009 |
Last Update Posted: | July 23, 2014 |
Last Verified: | July 2014 |
Keywords provided by BioMarin Pharmaceutical:
Phenylalanine Hydroxylase |
Additional relevant MeSH terms:
Phenylketonurias Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Amino Acid Metabolism, Inborn Errors Metabolism, Inborn Errors |
Genetic Diseases, Inborn Metabolic Diseases Verapamil Anti-Arrhythmia Agents Calcium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Vasodilator Agents |