NCT05229549

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

Terminated NA Last updated 7 July 2023

What this trial tests

NA trial testing GOLIKE PLUS 3-16 in Phenylketonurias in 5 participants. Terminated before completion.

Timeline

6 May 2022

Primary endpoint
29 November 2022

7 February 2023

Quick facts

Lead sponsor	APR Applied Pharma Research s.a.
Phase	NA
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	none
Primary purpose	treatment
Enrollment	5
Start date	6 May 2022
Primary completion	29 November 2022
Estimated completion	7 February 2023
Sites	1 location across Italy

Drugs / interventions tested

• GOLIKE PLUS 3-16
• Free AAs

Conditions studied

• Phenylketonurias — all drugs for Phenylketonurias →

Sponsor

APR Applied Pharma Research s.a. — full company profile →

Who can join

Adults 3 to 20, any sex, with Phenylketonurias. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own 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 product (PKU GOLIKE PLUS 3-16, a prolonged-release amino-acids (AAs) mixture) and the reference product (an immediate-release free AAs mixture with the same qualitative and quantitative composition of PKU GOLIKE PLUS 3-16). PKU GOLIKE PLUS 3-16 is a food for special medicinal purposes (FSMP) for the dietary management of PKU.

The study will consist of a screening visit (T0), four test days taking place on Sunday (T1-T4), each separated by a washout period (a period of time when a participant is taken off a treatment in order to eliminate its effects) and with the study products being self-administered at home, and a final visit (T5).

Following informed consent and verification of eligibility criteria, 24 patients with PKU will be randomized in a 1:1:1:1 ratio to one of the four randomization sequences ABCD, BDAC, DCBA or CADB.

A, B, C and D will be:

A. One day treatment with 3 self-administrations of GOLIKE PLUS 3-16 (1.2 g/kg total daily dose of AA) B. One day treatment with 2 self-administrations of GOLIKE PLUS 3-16 (1.2 g/kg total daily dose of AA) C. One day treatment with 3 self-administrations of free AAs (1.2 g/kg total daily dose of AA) D. One day treatment with 2 self-administrations of free AAs (1.2 g/kg total daily dose of AA)

GOLIKE PLUS 3-16 and the free AAs mixture will be the only protein substitute allowed on each test day, and no sports activities will be allowed on the test days.

24-hour urines and five blood spots will be collected on each test day. A patient's diary will be used to collect information on patient compliance, 24-hour urines and blood spot collections, diet, daily activities, adverse events and concomitant medications.

Patients completing all four test days will be asked to go to the center for a final visit (T5) taking place within 2 weeks from the last product administration.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.