Status:
TERMINATED
Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria
Lead Sponsor:
APR Applied Pharma Research s.a.
Conditions:
Phenylketonurias
Eligibility:
All Genders
3-20 years
Phase:
NA
Brief Summary
This is an open-label, randomized, 4-way crossover, monocentric, controlled study in patients (3 ≤ years of age ≤ 20 )) with phenylketonuria (PKU). The primary comparison will be between the test pro...
Detailed Description
This is an open-label, randomized, 4-way crossover, monocentric, controlled study in patients (3 ≤ years of age ≤ 20 ) with phenylketonuria (PKU). The primary comparison will be between the test prod...
Eligibility Criteria
Inclusion
- Signed, informed consent obtained prior to being enrolled into the study and prior to starting any data collection. Consent of a legally authorized guardian is required for legally minor patients as defined by local regulation. If the patient is legally a minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from the patient, if applicable.
- Male or female, aged 3-20 (limits included).
- Patients with diagnosis of PKU at newborn screening.
- Patients starting low Phe diet before 1 month of age.
- Ability and willingness to comply with all study procedures and availability for the duration of the study.
Exclusion
- Known or suspected hypersensitivity to any excipients/components of the investigational products.
- Treatment with therapy for PKU other than amino-acids supplementation.
- Patients aged \>12 years old with 4 repeated average values of blood Phe \>600 µM in the previous 6 months.
- Patients aged \<12 years old with 4 repeated average values of blood Phe \>360 µM in the previous 6 months.
- Any moderate to severe medical condition, which in the opinion of the Investigator would interfere with the study procedures or study outcome (reasoning to be provided), such as history of clinically significant diseases or malfunctions including, but not limited to, gastrointestinal, renal, hepatic, pulmonary, cardiovascular or endocrine disease, hemophilia, hyperlipidemia, impaired glucose tolerance or diabetes (type 1 or 2), or anemia.
- History of poor co-operation, non-compliance with medical treatments and diet, or poor adherence to investigational procedures.
- Any current participation in another clinical trial involving investigational or marketed products in the 3 months prior to the inclusion in this study.
- Pregnancy or lactation.
Key Trial Info
Start Date :
May 6 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 7 2023
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT05229549
Start Date
May 6 2022
End Date
February 7 2023
Last Update
July 7 2023
Active Locations (1)
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1
San Paolo Hospital
Milan, Italy