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

PHASE3CompletedINTERVENTIONAL
Enrollment

101

Participants

Timeline

Start Date

November 16, 2021

Primary Completion Date

June 13, 2024

Study Completion Date

June 13, 2024

Conditions
Growth Hormone Deficiency
Interventions
DRUG

Macimorelin

Dosage form: granules for oral solution, Dosage: 1.0 mg/kg body weight, Frequency and duration: single oral dose administration. Macimorelin will be supplied in single-use aluminum pouches (synonymous: sachets) each containing 63.6 mg macimorelin as acetate, which provide 0.5 mg/mL of macimorelin when dissolved in 120 mL of water. The excess amount of 3.6 mg represents an overfill, which is needed to obtain the target concentration.

DIAGNOSTIC_TEST

Arginine

For the arginine GHST, R-Gene® 10 from Pfizer will be provided as labelled investigational medicinal product (IMP). After an overnight fast, soluble arginine hydrochloride (0.5 g/kg) will be given i.v. as an infusion with an infusion duration of 30 min.

DIAGNOSTIC_TEST

Clonidine

"For the clonidine GHST, CATAPRESAN® 75 tablets (Boehringer Ingelheim) will be provided as labelled IMP. Each tablet contains 75 ug clonidine hydrochloride. The tablets will be provided in boxes containing 10 tablets. The target dose is 0.15 mg/m2 body surface with a dose range of 0.08 - 0.15 mg/m2. Maximum dose will be 0.25 mg.~After an overnight fast, clonidine (0.15 mg/m2 body surface) will be given orally."

Trial Locations (45)

1000

Univerzitetni Klinicni Center Ljubljana - Pediatrics, Ljubljana

4011

Children's University Hospital Kosice, Košice

6100

Ankara University, Faculty of Medicine, Ankara

7050

Antalya Training and Research Hospital, Antalya

10129

Icahn School of Medicine at Mount Sinai, New York

10587

"Institutul de Endocrinologie C.I. Parhon", Bucharest

10709

Children's Hospital at Montefiore, The Bronx

11000

University children's clinic Belgrade - Department of Endocrinology, Belgrade

18000

Clinical Center Nis - Clinic for Children's Internal Medicine, Niš

20154

Osp. dei Bambini V. Buzzi, ASST Fatebenefratelli Sacco, Milan

21000

Institute for Child and Youth Health Care of Vojvodina - Endocrinology, Novi Sad

27599

UNC Hospitals, Chapel Hill

30329

Emory Healthcare-Children's Center, Atlanta

33617

Evangelisches Klinikum Bethel, Bielefeld

33701

John Hopkins All Children's Hospital, St. Petersburg

41380

Kocaeli University Faculty of Medicine, Kocaeli

43100

Azienda Ospedaliero-Universitaria di Parma Ospedale dei Bambini Pietro Barilla, Clinica Pediatrica, Parma

50139

Azienda Ospedaliero-Universitaria Anna Meyer, Florence

50368

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu, Wroclaw

55454

University of Minnesota, Masonic Children's Hospital, Minneapolis

60126

Ospedale Pediatrico G. Salesi, Ancona

61080

Karadeniz Technical University, Ortahisar

64108

The Children's Mercy Hospital - Broadway, Kansas City

71252

SPSK Nr 1 im. prof. Tadeusza Sokolowskiego PUM, Szczecin

77583

Alchemi Research Center, Rosharon

80111

Pediatric Endocrine Associates, p.c., Greenwood Village

83340

National Institute of Children's Diseases, Bratislava

83712

St. Luke's Children's Endocrinology, Boise

85724

Angel Wing Clinic For Children With Diabetes, Tucson

98405

Multicare Health System, Tacoma

300011

Spitalul Cl. de Urgenta pentru Copii Louis Turcanu Timisoara, Timișoara

500283

Cen Med de Diagn si Trat Amb NEOMED, Brasov

540072

Spitalul Clinic Judetean Mures, Târgu Mureş

700111

"Spitalul Clinic Judetean de Urgenta Sf. Spiridon Iasi", Iași

900591

"Spitalul Clinic Judetean de Urgenta Sf. Apostol Andrei Constanta", Constanța

0024

Yerevan State Medical University after Mkhitar Heraci, Yerevan

0179

JSC Maritime Hospital, Batumi

0159

National Institute of Endocrinology, Tbilisi

TSMU Givi Jvania Pediatric Academic Clinik, Tbilisi

00165

IRCCS Ospedale Pediatrico Bambino Gesù, Roma

60-693

MED-POLONIA Sp.z o.o., Poznan

35-301

Kliniczny Szpital Wojewodzki nr 2 im. Sw. Jadwigi Krolowej w Rzeszowie, Rzeszów

011025

Sana Monitoring, Bucharest

013982

Medicover Hospitals, Bucharest

03491

National Institute of Endocrinology and Diabetology, Ľubochňa

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