Phenylketonurias Clinical Trial
Official title:
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
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.
Status | Recruiting |
Enrollment | 20 |
Est. completion date | July 31, 2024 |
Est. primary completion date | July 31, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 16 Years and older |
Eligibility | Inclusion Criteria: 1. 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 2. Male or female, aged =16 years. 3. Patients with a registered diagnosis of PKU 4. Ability and willingness to comply with all study procedures and availability for the duration of the study. 5. 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). 6. Patient taking free-AA and/or GMP as usual amino-acidssupplementation. Exclusion Criteria: 1. Known or suspected hypersensitivity to any excipients/components of PKU GOLIKE. 2. Treatment with any drug therapy for PKU 3. Patient taking PKU GOLIKE as usual amino-acids supplementation 4. Patient taking LNAA as usual amino-acids supplementation 5. Any moderate to severe medical condition, which in the opinion of the Investigator would interfere with the study procedures or study outcome (reason to be provided) 6. Any current participation in another clinical trial involving investigational or marketed products in the 3 months prior to the inclusion in this study. 7. Pregnancy or lactation. |
Country | Name | City | State |
---|---|---|---|
Italy | ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8 | Milan |
Lead Sponsor | Collaborator |
---|---|
APR Applied Pharma Research s.a. |
Italy,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Phe blood concentration with dried blood spots (umol/L) on the second test day with Standard of Care right before product self-administration and before any food intake | at 8:00 am(±15 min) on the second test day with SoC | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the second test day with GOLIKE PLUS right before product self-administration and before any food intake | at 8:00 am(±15 min) on the second test day with Golike Plus | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the second test day with Standard of Care right before product self-administration and before food intake | at 12:00 am (±15 min) on the second test day with SoC | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the second test day with GOLIKE PLUS right before product self-administration and before food intake | at 12:00 am (±15 min) on the second test day with Golike Plus | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the second test day with Standard of Care before food intake | At 4:00 pm (±15 min) on the second test day with SoC | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the second test day with GOLIKE PLUS before food intake | At 4:00 pm (±15 min) on the second test day with Golike Plus | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the second test day with Standard of Care right before product self-administration and before any food intake | at 8:00 pm (±15 min) on the second test day with SoC | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the second test day with GOLIKE PLUS right before product self-administration and before any food intake | at 8:00 pm (±15 min) on the second test day with Golike Plus | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the day following the second test day with Standard of Care on the following morning, before any food intake. | at 8:00 am (±15 min) on the day following the second test day with SoC | ||
Primary | Phe blood concentration with dried blood spots (umol/L) on the day following the second test day with GOLIKE PLUS on the following morning, before any food intake. | at 8:00 am (±15 min) on the day following the second test day with Golike Plus | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L) on the second test day with Standard of Care right before product self-administration and before any food intake | at 8:00 am(±15 min) on the second test day with SoC | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L)on the second test day with GOLIKE PLUS right before product self-administration and before any food intake | at 8:00 am(±15 min)on the second test day with Golike Plus | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L)on the second test day with Standard of Care right before product self-administration and before any food intake | at 12:00 am(±15 min) on the second test day with SoC | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L) on the second test day with GOLIKE PLUS right before product self-administration and before any food intake | at 12:00 am(±15 min) on the second test day with Golike Plus | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L)on the second test day with Standard of Care before food intake | at 4:00 pm(±15 min)on the second test day with SoC | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L) on the second test day with GOLIKE PLUS before food intake | at 4:00 pm(±15 min)on the second test day with Golike Plus | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L)on the second test day with Standard of Care right before product self-administration and before any food intake | at 8:00 pm(±15 min) on the second test day with SoC | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L)on the second test day with GOLIKE PLUS right before product self-administration and before any food intake | at 8:00 pm(±15 min) on the second test day with Golike Plus | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L)on the day following the second test day with Standard of Care on the following morning, before any food intake. | at 8:00 am(±15 min) on the day following the second test day with SoC | ||
Secondary | Tyr blood concentration with dried blood spots (umol/L) on the day following the second test day with GOLIKE PLUS on the following morning, before any food intake. | at 8:00 am(±15 min) on the day following the second test day with Golike Plus |
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