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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. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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9 May 2022 |
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Main ID: |
NCT02677870 |
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Date of registration:
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26/01/2016 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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The Effectiveness of Kuvan in Amish PKU Patients
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Scientific title:
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The Effectiveness of High-Dose Synthetic BH4 (Saproterin Dihydrochloride or "Kuvan") in Amish PKU Patients |
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Date of first enrolment:
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January 22, 2018 |
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Target sample size:
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7 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02677870 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 4
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Countries of recruitment
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United States
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Contacts
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Name:
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Lori-Anne P Schillaci, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University Hospitals Cleveland Medical Center |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Current diagnosis of PKU with the following:
- Age of at least 2 years or older
- Baseline Phe level of > 360 umol/L
- Willing to maintain a stable diet
- Patient or guardian are willing and able to provide written, signed informed consent
after the nature of the study has been explained and prior to any research-related
procedures
- Are willing to comply with all study procedures
- Two identifiable mutations found on PAH gene sequencing
- Any patients already taking Saproterin (or have taken in the past), must have a
treatment end date at least 14 days prior to Day 1 of the study.
Exclusion Criteria:
- Any patient currently taking Saproterin who has taken the medication at any point in
the 14 days prior to Day 1 of the study
- Under 2 years of age
- Unwilling to maintain a stable diet
- Patients with baseline Phe levels < 360 umol/L
- Patients unable to comply with all study procedures
- Patients unable to provide written, signed informed consent
- One (or no) identifiable mutations found on PAH gene sequencing
Age minimum:
2 Years
Age maximum:
60 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Phenylketonuria
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Intervention(s)
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Other: Diet treatment
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Drug: saproterin dihydrochloride
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Primary Outcome(s)
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Number of Participants With 20% Decrease in Phenylalanine Levels From Baseline (Positive Response)
[Time Frame: Baseline, weekly for 4 weeks during diet arm, weekly for 4 weeks during both high and standard dose arms]
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Secondary ID(s)
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UHCMC IRB#
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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