TerminatedNot Applicable

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

Stopped: Due to recruitment challenges, and despite an extension of the study period, the decision to terminate the trial early was made in agreement with the Principal Investigator

Italy21 participantsStarted 2023-10-30

Plain-language 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.

Who can participate

Age range16 Years

SexALL

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Inclusion criteria

✓. 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.

Exclusion criteria

✕. Known or suspected hypersensitivity to any excipients/components of PKU GOLIKE.
✕. Treatment with any drug therapy for PKU
✕

What they're measuring

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

Timeframe: at 8:00 am(±15 min) on the second test day (T2; T4) with SoC

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

Timeframe: at 8:00 am(±15 min) on the second test day (T2; T4) with Golike Plus

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

Timeframe: at 12:00 am (±15 min) on the second test day (T2; T4)with SoC

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

Timeframe: at 12:00 am (±15 min) on the second test day (T2; T4) with Golike Plus

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake

Timeframe: At 4:00 pm (±15 min) on the second test (T2;T4) day with SoC

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake

Timeframe: At 4:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus

Trial details

NCT IDNCT05827536

SponsorAPR Applied Pharma Research s.a.

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-11-27

Results submitted2025-12-30

View on ClinicalTrials.gov More Phenylketonurias trials