Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
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
1 other identifier
interventional
700
1 country
13
Brief Summary
The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe) level among subjects with phenylketonuria (PKU) who have elevated Phe levels. A secondary objective of this study is to 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Dec 2004
Shorter than P25 for phase_2
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 1, 2004
CompletedFirst Submitted
Initial submission to the registry
February 24, 2005
CompletedFirst Posted
Study publicly available on registry
February 25, 2005
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2005
CompletedApril 9, 2007
April 1, 2007
February 24, 2005
April 5, 2007
Conditions
Outcome Measures
Primary Outcomes (1)
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
Secondary Outcomes (1)
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
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (13)
Unknown Facility
Los Angeles, California, United States
Unknown Facility
Oakland, California, United States
Unknown Facility
New Haven, Connecticut, United States
Unknown Facility
Chicago, Illinois, United States
Unknown Facility
Boston, Massachusetts, United States
Unknown Facility
Minneapolis, Minnesota, United States
Unknown Facility
St Louis, Missouri, United States
Unknown Facility
New York, New York, United States
Unknown Facility
Portland, Oregon, United States
Unknown Facility
Pittsburgh, Pennsylvania, United States
Unknown Facility
Dallas, Texas, United States
Unknown Facility
Salt Lake City, Utah, United States
Unknown Facility
Madison, Wisconsin, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
February 24, 2005
First Posted
February 25, 2005
Study Start
December 1, 2004
Study Completion
November 1, 2005
Last Updated
April 9, 2007
Record last verified: 2007-04