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

NCT04786873 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 101

Last updated 2024-08-12

No results posted yet for this study

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).

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.

Sponsors & Collaborators

  • AEterna Zentaris

    lead INDUSTRY

Principal Investigators

  • Nicola K Ammer, MD · AEterna Zentaris

Study Design

Allocation
RANDOMIZED
Purpose
DIAGNOSTIC
Masking
SINGLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-16
Primary Completion
2024-06-13
Completion
2024-06-13
FDA Drug
Yes

Countries

  • United States
  • Armenia
  • Georgia
  • Germany
  • Italy
  • Poland
  • Romania
  • Serbia
  • Slovakia
  • Slovenia
  • Turkey (Türkiye)

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04786873 on ClinicalTrials.gov