A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is

Completed · Phase 3

Conditions studied: Growth Hormone Deficiency

In brief

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).

Key facts

Study ID

NCT04786873

Run by

AEterna Zentaris

People needed

101

Starts

2021-11-16

Expected to finish

2024-06-13

Last updated by the study team

2024-08-12

Who can join

Age: 2 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.
• 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.
• Known or suspected hypersensitivity to trial product(s) or related products;
• Any disorder, which in the investigator's opinion might jeopardize subject's safety or compliance with the protocol.
• Concomitant treatment with any drugs that might prolong QT/QTc Note: A subject who receives such treatment will not be a candidate for this study, if his/her condition does not allow for a treatment-free period of at least 5 elimination half-lives of the drug that might prolong QT/QTc before the GHST;
• Elevation of laboratory parameters indicating hepatic or renal dysfunction or damage (aspartate amino transferase (AST), alkaline phosphatase (ALT), gamma-glutamyl transferase (GGT) > 2.5 x upper limit of normal (ULN); creatinine or bilirubin > 1.5x ULN);
• Current active malignancy other than non-melanoma skin cancer;
• Female of child-bearing potential and not using an adequate contraceptive method (adequate contraceptive measures as required by local regulation or practice).
• Male of reproductive age who or whose partner(s) is not using an adequate contraceptive method (adequate contraceptive measures as required by local regulation or practice).
• Lack of ability or willingness to give informed consent by the subject and/or his/her legal representative;
• Anticipated non-availability for trial visits/procedures.

Where it is running

• Angel Wing Clinic For Children With Diabetes — Tucson, Arizona, United States
• Pediatric Endocrine Associates, p.c. — Greenwood Village, Colorado, United States
• John Hopkins All Children's Hospital — St. Petersburg, Florida, United States
• Emory Healthcare-Children's Center — Atlanta, Georgia, United States
• St. Luke's Children's Endocrinology — Boise, Idaho, United States
• University of Minnesota, Masonic Children's Hospital — Minneapolis, Minnesota, United States
• The Children's Mercy Hospital - Broadway — Kansas City, Missouri, United States
• Icahn School of Medicine at Mount Sinai — New York, New York, United States
• Children's Hospital at Montefiore — The Bronx, New York, United States
• UNC Hospitals — Chapel Hill, North Carolina, United States
• Alchemi Research Center — Rosharon, Texas, United States
• Multicare Health System — Tacoma, Washington, United States
• Yerevan State Medical University after Mkhitar Heraci — Yerevan, Armenia
• JSC Maritime Hospital — Batumi, Georgia
• National Institute of Endocrinology — Tbilisi, Georgia
• TSMU Givi Jvania Pediatric Academic Clinik — Tbilisi, Georgia
• Evangelisches Klinikum Bethel — Bielefeld, Germany
• Ospedale Pediatrico G. Salesi — Ancona, Italy
• Azienda Ospedaliero-Universitaria Anna Meyer — Florence, Italy
• Osp. dei Bambini V. Buzzi, ASST Fatebenefratelli Sacco — Milan, Italy
• Azienda Ospedaliero-Universitaria di Parma Ospedale dei Bambini Pietro Barilla, Clinica Pediatrica — Parma, Italy
• IRCCS Ospedale Pediatrico Bambino Gesù — Roma, Italy
• MED-POLONIA Sp.z o.o. — Poznan, Poland
• Kliniczny Szpital Wojewodzki nr 2 im. Sw. Jadwigi Krolowej w Rzeszowie — Rzeszów, Poland
• SPSK Nr 1 im. prof. Tadeusza Sokolowskiego PUM — Szczecin, Poland

Full record on ClinicalTrials.gov

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