Main
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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:
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ClinicalTrials.gov |
Last refreshed on:
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12 December 2020 |
Main ID: |
NCT04227080 |
Date of registration:
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10/01/2020 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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BH4 Responsiveness in PAH Deficiency PKU Patients
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Scientific title:
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To Evaluate BH4 Responsiveness in PAH Deficiency PKU Patients Who Failed to Achieve 30% Blood Phe Reduction Within 24-hour BH4 Loading Test by Extending the Period of BH4 Response Test: A Pilot Study in Taiwan |
Date of first enrolment:
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June 2020 |
Target sample size:
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40 |
Recruitment status: |
Not yet recruiting |
URL:
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https://clinicaltrials.gov/show/NCT04227080 |
Study type:
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Interventional |
Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 4
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Key inclusion & exclusion criteria
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Inclusion criteria
1. Subjects have a confirmed diagnosis of PAH deficiency PKU, defined as baseline blood
Phe levels of >360 umol/L
2. Subject and/or parent or guardian must be capable of understanding and providing
written informed consent
3. For PAH deficiency PKU newborns, they should be identified as non-BH4 responders by
24-hour BH4 loading test after birth
4. For PAH deficiency PKU older pediatric and adult patients, they should be identified
as non-BH4 responders by 24-hour BH4 loading test and currently use Phe-restricted
diet control alone
Exclusion criteria
1. Perceived to be unreliable or unavailable for study participation or, if under the age
of 18, had parents or legal guardians who the investigator perceived to be unreliable
or unavailable
2. Used any investigational agent other than Kuvan within 30 days of screening, or
required any investigational agent or investigational vaccine prior to completion of
all scheduled study assessments
3. Pregnant or breastfeeding, or considering pregnancy
4. Concurrent disease or condition that would interfere with study participation or
safety (eg, seizure disorder, asthma or other condition requiring oral or parenteral
corticosteroid administration, insulin-dependent diabetes, or organ transplantation
recipient)
5. Serious neuropsychiatric illness (eg, major depression) not currently under medical
management
6. Required concomitant treatment with any drug known to inhibit folate synthesis (eg,
methotrexate)
7. Clinical diagnosis of primary BH4 deficiency
8. Patients him/herself or his/her caregivers are unable or unwilling to sign informed
consent or to comply with the requirements of the study
9. Patient attend other clinical trials should not be included
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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PAH Deficiency
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Pku Phenylketonuria
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Intervention(s)
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Drug: BH4
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Primary Outcome(s)
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Changes of blood Phe level from baseline
[Time Frame: 7th, 14th, and 28th day]
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Secondary Outcome(s)
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Phe level decreases for >= 30%
[Time Frame: 7th, 14th, and 28th day]
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PAH gene mutation and Phe level
[Time Frame: 7th, 14th, and 28th day]
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Phe tolerance increase from baseline for >=50%
[Time Frame: 7th, 14th, and 28th day]
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Secondary ID(s)
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Version 1.0, 2018/03/21
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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