- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00225615
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
August 18, 2009 updated by: BioMarin Pharmaceutical
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment
100
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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California
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Los Angeles, California, United States
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Oakland, California, United States
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Sacramento, California, United States
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San Jose, California, United States
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Connecticut
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New Haven, Connecticut, United States
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Georgia
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Atlanta, Georgia, United States
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Illinois
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Chicago, Illinois, United States
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Minnesota
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Minneapolis, Minnesota, United States
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Missouri
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St. Louis, Missouri, United States
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New York
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New York, New York, United States
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Oregon
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Portland, Oregon, United States
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Texas
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Dallas, Texas, United States
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Utah
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Salt Lake City, Utah, United States
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Wisconsin
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Madison, Wisconsin, United States
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
8 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- 8 years of age and older
- Prior successful participation in Study PKU-003
- Willing and able to provide written informed consent or assent and written informed consent (if required) by a parent or legal guardian
- For females of child-bearing potential only: Negative urine pregnancy test within 24 hours prior to enrollment. Women using acceptable birth control measures must agree to continue to use those 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
- Withdrew from, or otherwise did not successfully complete, study PKU-003, except for subjects who were removed from the study because their blood Phe exceeded the alert level
- Expected to require any investigational agent or vaccine prior to completion of all scheduled study assessments
- Pregnant or breastfeeding, or planning pregnancy
- 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)
- Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
- Concurrent use of levodopa
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
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Primary objective:
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- To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
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Secondary Outcome Measures
Outcome Measure |
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Secondary objectives:
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To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.
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To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.
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To evaluate the population pharmacokinetics of Phenoptin.
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To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period.
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To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels.
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Chair: Alex Dorenbaum, MD, BioMarin Pharmaceutical
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
November 1, 2005
Study Completion (Actual)
June 1, 2006
Study Registration Dates
First Submitted
September 22, 2005
First Submitted That Met QC Criteria
September 22, 2005
First Posted (Estimate)
September 26, 2005
Study Record Updates
Last Update Posted (Estimate)
August 20, 2009
Last Update Submitted That Met QC Criteria
August 18, 2009
Last Verified
August 1, 2009
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Amino Acid Metabolism, Inborn Errors
- Phenylketonurias
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Arrhythmia Agents
- Vasodilator Agents
- Membrane Transport Modulators
- Calcium-Regulating Hormones and Agents
- Calcium Channel Blockers
- Verapamil
Other Study ID Numbers
- PKU-004
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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