Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00104260 |
Recruitment Status :
Completed
First Posted : February 25, 2005
Last Update Posted : April 9, 2007
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Phenylketonurias | Drug: sapropterin dihydrochloride | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Enrollment : | 700 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels |
Study Start Date : | December 2004 |
Actual Study Completion Date : | November 2005 |

- Evaluate the degree and frequency of response to Phenoptin™, as demonstrated by a reduction in blood Phe level among subjects with PKU who have elevated Phe levels
- Evaluate the safety of Phenoptin™ treatment in this subject population, and identify individuals in this subject population who respond to Phenoptin™ treatment with a reduction in blood Phe level

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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:
- Age >/= 8 years
- Blood Phe level >/= 450 umol/L at screening
- Clinical diagnosis of PKU with hyperphenylalaninemia documented by past medical history of at least one blood Phe measurement >/= 360 umol/L (6 mg/dL)
- 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 (non-sterile females of child-bearing potential only)
- Male and Female subjects of childbearing potential childbearing potential (if sexually active and non-sterile) 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 within 30 days prior to screening, or requirement for any investigational agent or 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)
- Serious neuropsychiatric illness (e.g., major depression) not currently under medical control
- 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): NCT00104260
United States, California | |
Los Angeles, California, United States | |
Oakland, California, United States | |
United States, Connecticut | |
New Haven, Connecticut, United States | |
United States, Illinois | |
Chicago, Illinois, United States | |
United States, Massachusetts | |
Boston, Massachusetts, United States | |
United States, Minnesota | |
Minneapolis, Minnesota, United States | |
United States, Missouri | |
St. Louis, Missouri, United States | |
United States, New York | |
New York, New York, United States | |
United States, Oregon | |
Portland, Oregon, United States | |
United States, Pennsylvania | |
Pittsburgh, Pennsylvania, United States | |
United States, Texas | |
Dallas, Texas, United States | |
United States, Utah | |
Salt Lake City, Utah, United States | |
United States, Wisconsin | |
Madison, Wisconsin, United States |
ClinicalTrials.gov Identifier: | NCT00104260 |
Other Study ID Numbers: |
PKU-001 |
First Posted: | February 25, 2005 Key Record Dates |
Last Update Posted: | April 9, 2007 |
Last Verified: | April 2007 |
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 Calcium-Regulating Hormones and Agents Physiological Effects of Drugs Vasodilator Agents |