A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Horm… (NCT04786873) | Clinical Trial Compass
CompletedPhase 3
A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is
United States101 participantsStarted 2021-11-16
Plain-language summary
This research study will find out if a new growth hormone stimulation test is safe and works as well as other tests to diagnose growth hormone deficiency (GHD) in children. The stimulation test will use a new growth hormone stimulating substance called macimorelin. By now, only adults in the USA can get this new stimulation test. The results of this study are expected to help children and teenagers with suspected GHD to get the macimorelin stimulation test.
The macimorelin test will be compared to a clonidine and an arginine test. Both are known standard stimulation tests. Altogether two macimorelin tests are planned to be performed in the study, to show how repeatable macimorelin tests results are (under a set of similar conditions).
Who can participate
Age range2 Years – 17 Years
SexALL
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Inclusion criteria
✓. Informed consent of subject, parent(s) or legally acceptable representative (LAR) of subject and child assent, if appropriate, must be obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
✓. Male and female pediatric subjects from 2 to less than 18 years of age at the time of signing informed consent.
✓. Indication for the performance of growth hormone stimulation test.
✓. Presence of a height measurement minimum 6 and maximum 18 months prior to screening.
Exclusion criteria
✕. Established diagnosis of a disease that is sufficient to explain growth deficiency or metabolic disorders that are also associated with short stature (e.g., Turner syndrome, skeletal dysplasia's, celiac disease, etc.).
✕. Ongoing growth hormone therapy.
✕. Presence of hypothyroidism and/or adrenal insufficiency without adequate and stable replacement therapy treatment for at least 30 days prior to first GHST.
What they're measuring
1
Area under the Receiver Operator Characteristic curve (ROC AUC) based on GH concentration during GHST following macimorelin administration
Timeframe: Derived from Cmax GH measurements collected in the time frame from 0 to 90 minutes after initial macimorelin GHST (visit 2 (day 0)) and GH adjudication status performed by the adjudication committee after visit 4 (between day 11 and day 58)).
. Treatment with drugs directly affecting the pituitary secretion of somatotropin (e.g., somatostatin analogues, clonidine, levodopa and dopamine agonists) or provoking the release of somatostatin (antimuscarinic agents e.g., atropine).
✕. Medical history of ongoing clinically symptomatic psychiatric disorders.
✕. 2nd or 3rd degree atrioventricular-block, prolongation of the QRS complex over 120 milliseconds, prolongation of the QTc interval over 450 milliseconds, or any other clinically significant abnormal electrocardiogram results at the V2 pre-dose electrocardiogram (ECG) as judged by the investigator.
✕. Previous participation in this trial. Participation is defined as signed informed consent.
✕. Participation in any clinical trial of an approved or non-approved investigational medicinal product within 30 days before screening.