Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus Standard of Care in Patients With Phenylketonuria

Status: Recruiting
Location: See location...
Intervention Type: Dietary supplement
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY

This is an open-label, randomized, 2-way crossover, monocentric controlled study in patients (≥ 16 years old) with phenylketonuria (PKU). The comparison will be between the test product (PKU GOLIKE, a prolonged-release amino-acids (AAs) mixture) and standard of care.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 16
Healthy Volunteers: f
View:

• Signed, informed consent obtained by the patient prior to beingenrolled into the study and prior to starting any data collection. For legally minor patients, signed written consent shall be obtained also by the parents/legal guardian

• Male or female, aged ≥16 years.

• Patients with a registered diagnosis of PKU

• Ability and willingness to comply with all study procedures and availability for the duration of the study.

• Patients with mean value of blood Phe \>360 µM in the previous 12 months (calculated on at least 3 samples during the previous 12 months; the last sample should be preferably obtained in the 30 days preceding inclusion in the study).

• Patient taking free-AA and/or GMP as usual amino-acidssupplementation.

Locations
Other Locations
Italy
ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8
RECRUITING
Milan
Contact Information
Primary
Giorgio Reiner
giorgio.reiner@apr.ch
+41.91.6957020
Time Frame
Start Date: 2023-10-30
Estimated Completion Date: 2024-12-31
Participants
Target number of participants: 20
Treatments
Other: AB where A=standard of care and B=PKU GOLIKE
Patients will receive 3 daily self-administrations of their usual standard of care for 2 consecutive days in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period.
Other: BA where B=PKU GOLIKE and A=standard of care
Patients will receive 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the first period followed by 3 daily self-administrations of their usual standard of care for 2 consecutive days in the second period.
Related Therapeutic Areas
Sponsors
Leads: APR Applied Pharma Research s.a.

This content was sourced from clinicaltrials.gov