|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
12 December 2020 |
|
Main ID: |
NCT00355264 |
|
Date of registration:
|
19/07/2006 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
|
|
Scientific title:
|
Phase 2, Multicenter, Open Label Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to Primary BH4 Deficiency |
|
Date of first enrolment:
|
August 2006 |
|
Target sample size:
|
12 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT00355264 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
Germany
|
United States
| | | | | | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Documented history of blood Phe level > 180 µmol/L on at least one occasion
- Established diagnosis of hyperphenylalaninemia (HPA) due to primary BH4 deficiency
with a documented defect in biopterin metabolism with blood or urine tests
- Willing and able to provide written informed consent or, in the case of subjects under
the age of 18 years, provide written assent (if required) and written informed consent
by a parent or legal guardian
- Negative urine pregnancy test at screening for females of child-bearing potential
- Male and female subjects of childbearing potential (if sexually active and
non-sterile) must be using acceptable birth control measures and be willing to
continue to use acceptable birth control measures, as determined by the Investigator,
and be willing to continue to use acceptable birth control measures while
participating in the study
- Willing and able to comply with all study procedures
- Able to take medication orally
Exclusion Criteria:
- Perceived to be unreliable or unavailable for study participation or, if under the age
of 18 years, have parents or legal guardians who are perceived to be unreliable or
unavailable
- Use of any investigational agent (other than BH4) within 30 days prior to screening,
or requirement for any investigational agent or vaccine prior to completion of all
scheduled study assessments
- Positive urine pregnancy test at screening (non-sterile females of child bearing
potential only), already known to be pregnant or breastfeeding or planning a pregnancy
in self or partner during the study
- Female subjects of childbearing potential not using an effective method of birth
control, as determined by the PI, or unwilling to continue to use acceptable birth
control measures.
- Alanine aminotransferase (ALT) > 2 times the upper limit of normal (i.e., Grade 1 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, 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)
- Clinical diagnosis of phenylketonuria (PKU) due to phenylalanine hydroxylase
deficiency
- Any condition that, in the view of the PI, renders the subject at high risk from
treatment compliance and/or completing the study
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Hyperphenylalaninemia, Non-Phenylketonuric
|
|
Tetrahydrobiopterin Deficiencies
|
|
Intervention(s)
|
|
Drug: Phenoptin
|
|
Primary Outcome(s)
|
|
Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints
[Time Frame: At Baseline, Week 4 through Extension Week 130]
|
|
Percentage of Subjects With Blood Phenylalanine (Last Observation Carried Forward) < 360 µmol/L
[Time Frame: At Baseline, Week 4 through Extension Week 130]
|
|
Secondary Outcome(s)
|
|
Subjects Experiencing Adverse Events(AEs)
[Time Frame: Up to 35 Months]
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|