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 - NA
- Conditions
- Phenylketonuria (PKU)MedDRA version: 6.1 Classification code 10034872
- Registration Number
- EUCTR2004-002071-16-GB
- Lead Sponsor
- BioMarin Pharmaceutical Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 700
1. Age = or > 8 years.
2. Blood Phe level = or >450 µmol/L at screening· Clinical diagnosis of PKU with hyperphenylalaninemia documented by past medical history of at least one blood Phe measurement = or > 360 µmol/L (6 mg/dL).
3. 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.
4. Negative urine pregnancy test at screening (non-sterile females of child-bearing potential only).
5. Male and female subjects of childbearing potential (if sexually active and nonsterile) 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.
6. Willing and able to comply with study procedures.
7. Willing to continue current diet unchanged while participating in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
1.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
2. 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.
3. Pregnant or breastfeeding, or considering pregnancy.
4. ALT > 5 times the upper limit of normal (i.e., Grade 3 or higher based on WHO Toxicity Criteria) at screening.
5. 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.
6. Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate).
7. Concurrent use of levodopa.
8. Clinical diagnosis of primary BH4 deficiency.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of this study is to evaluate the degree and frequency of response to Phenoptin treatment, as demonstrated by a reduction in blood phenylalanine (Phe) level, among subjects with phenylketonuria (PKU) who have elevated blood Phe levels.;<br> Secondary Objective: The secondary objectives of this study are as follows:<br><br> To evaluate the safety of Phenoptin treatment in this subject population·<br><br> To identify individuals in this subject population who respond to Phenoptin treatment with a reduction in blood Phe level<br> ;Primary end point(s): The percentage of subjects who experience a response to Phenoptin on Day 8 will be calculated, with response defined as a reduction in blood Phe level of =30% compared with baseline (Day 1). In addition, the mean change in blood Phe levels at Day 8 will be estimated in two baseline strata (<1200 µmol/L and =1200 µmol/L).
- Secondary Outcome Measures
Name Time Method