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Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

Primary Purpose

Phenylketonurias

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
sapropterin dihydrochloride
Sponsored by
BioMarin Pharmaceutical
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Phenylketonurias

Eligibility Criteria

8 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Outcomes

Primary Outcome Measures

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 Outcome Measures

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

Full Information

First Posted
February 24, 2005
Last Updated
April 5, 2007
Sponsor
BioMarin Pharmaceutical
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1. Study Identification

Unique Protocol Identification Number
NCT00104260
Brief Title
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
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 Type
Interventional

2. Study Status

Record Verification Date
April 2007
Overall Recruitment Status
Completed
Study Start Date
December 2004 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
November 2005 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
BioMarin Pharmaceutical

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonurias

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
700 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
sapropterin dihydrochloride
Primary Outcome Measure Information:
Title
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 Outcome Measure Information:
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
City
Los Angeles
State/Province
California
Country
United States
City
Oakland
State/Province
California
Country
United States
City
New Haven
State/Province
Connecticut
Country
United States
City
Chicago
State/Province
Illinois
Country
United States
City
Boston
State/Province
Massachusetts
Country
United States
City
Minneapolis
State/Province
Minnesota
Country
United States
City
St. Louis
State/Province
Missouri
Country
United States
City
New York
State/Province
New York
Country
United States
City
Portland
State/Province
Oregon
Country
United States
City
Pittsburgh
State/Province
Pennsylvania
Country
United States
City
Dallas
State/Province
Texas
Country
United States
City
Salt Lake City
State/Province
Utah
Country
United States
City
Madison
State/Province
Wisconsin
Country
United States

12. IPD Sharing Statement

Links:
URL
http://www.biomarinpharm.com/
Description
Related Info

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Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

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