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A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

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: NCT00225615
Recruitment Status : Completed
First Posted : September 26, 2005
Last Update Posted : August 20, 2009
Sponsor:
Information provided by:
BioMarin Pharmaceutical

Brief Summary:
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.

Condition or disease Intervention/treatment Phase
Phenylketonurias Drug: sapropterin dihydrochloride Phase 3

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Study Type : Interventional  (Clinical Trial)
Enrollment : 100 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Study Start Date : November 2005
Actual Study Completion Date : June 2006





Primary Outcome Measures :
  1. Primary objective:
  2. - To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.

Secondary Outcome Measures :
  1. Secondary objectives:
  2. To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.
  3. To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.
  4. To evaluate the population pharmacokinetics of Phenoptin.
  5. To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period.
  6. To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels.


Information from the National Library of Medicine

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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
Criteria

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

Information from the National Library of Medicine

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): NCT00225615


Locations
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United States, California
Los Angeles, California, United States
Oakland, California, United States
Sacramento, California, United States
San Jose, California, United States
United States, Connecticut
New Haven, Connecticut, United States
United States, Georgia
Atlanta, Georgia, United States
United States, Illinois
Chicago, Illinois, 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, Texas
Dallas, Texas, United States
United States, Utah
Salt Lake City, Utah, United States
United States, Wisconsin
Madison, Wisconsin, United States
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
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Study Chair: Alex Dorenbaum, MD BioMarin Pharmaceutical

Additional Information:
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ClinicalTrials.gov Identifier: NCT00225615    
Other Study ID Numbers: PKU-004
First Posted: September 26, 2005    Key Record Dates
Last Update Posted: August 20, 2009
Last Verified: August 2009
Keywords provided by BioMarin Pharmaceutical:
Phenylalanine Hydroxylase
Additional relevant MeSH terms:
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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