Clinical Trial Details
— Status: Terminated
Administrative data
NCT number |
NCT05229549 |
Other study ID # |
GLK-2021 |
Secondary ID |
|
Status |
Terminated |
Phase |
N/A
|
First received |
|
Last updated |
|
Start date |
May 6, 2022 |
Est. completion date |
February 7, 2023 |
Study information
Verified date |
January 2022 |
Source |
APR Applied Pharma Research s.a. |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial 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 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.
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, in this protocol referred to as GOLIKE PLUS
3-16) 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). A telephone call will be made to the
patients during each washout period. Test days will occur preferably on four consecutive
weeks with a 1-week washout period between each test day. Washout can be extended up to 2
weeks in case a dose delay (i.e. delay of the test day) is deemed necessary.
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 following 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) No change in the randomization sequence will be allowed.
Diet will be standardized among different patients in terms of calories and nutrients ranges
on each test day, according to the age and body weight of the patients. Moreover, the type of
food ingested in 24 hours will be the same on each test day in the same patient.
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. Timing of
self-administrations, meals and blood spots will be defined for each test day. On the test
days, no food will be allowed outside of the defined time windows. The first
self-administration of each test day will be performed after overnight fasting (10 -12 h) and
before any food intake takes place. Urine should be voided before the first
self-administration and not collected. 24-hour urine collection begins thereafter.
The day after each test day a designated courier will collect urine samples and blood spots
at the patient's home and will bring them to the center.
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.
Adverse events will be continuously monitored during the study, starting from informed
consent. Adverse events will be collected by the patients (or by a parent/guardian) in the
diary and during the telephone calls made by the Investigator to the patients. Moreover,
patients (or parents/guardians) will be instructed to promptly report adverse events
occurring during the study to the Investigator.
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. Patients prematurely
discontinued from the study will be asked to attend a discontinuation visit possibly taking
place within 2 weeks from the last product administration. At the final/discontinuation
visit, patients will be asked to return their diaries and the Investigator will verify all
information reported in the patient's diary and record it in the electronic Case Report Form
(eCRF).