Growth Hormone DeficiencyPhase 3DETECTNCT04786873
A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is
A Phase 3 study testing Macimorelin, Arginine and Clonidine in people with Growth Hormone Deficiency, run by AEterna Zentaris at 45 sites in 11 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Growth Hormone Deficiency
- What is being tested?
- Macimorelin, Arginine and Clonidine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 101 participants
- Where
- 45 sitesin 11 countries
- Study dates
- 16 Nov 2021to 13 Jun 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
45 sites in 11 countries.
45 sites in 11 countries
United States12 sites
Emory Healthcare-Children's Center
Atlanta, Georgia, 30329
St. Luke's Children's Endocrinology
Boise, Idaho, 83712
UNC Hospitals
Chapel Hill, North Carolina, 27599
Pediatric Endocrine Associates, p.c.
Greenwood Village, Colorado, 80111
The Children's Mercy Hospital - Broadway
Kansas City, Missouri, 64108
University of Minnesota, Masonic Children's Hospital
Minneapolis, Minnesota, 55454
Icahn School of Medicine at Mount Sinai
New York, New York, 10129
Alchemi Research Center
Rosharon, Texas, 77583
John Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
Multicare Health System
Tacoma, Washington, 98405
Children's Hospital at Montefiore
The Bronx, New York, 10709
Angel Wing Clinic For Children With Diabetes
Tucson, Arizona, 85724
Romania8 sites
Cen Med de Diagn si Trat Amb NEOMED
Brasov, 500283
Sana Monitoring
Bucharest, 011025
Medicover Hospitals
Bucharest, 013982
Institutul de Endocrinologie "C.I. Parhon"
Bucharest, 10587
Spitalul Clinic Judetean de Urgenta "Sf. Apostol Andrei" Constanta
Constanța, 900591
Spitalul Clinic Judetean de Urgenta "Sf. Spiridon" Iasi
Iași, 700111
Spitalul Clinic Judetean Mures
Târgu Mureş, 540072
Spitalul Cl. de Urgenta pentru Copii Louis Turcanu Timisoara
Timișoara, 300011
Italy5 sites
Ospedale Pediatrico G. Salesi
Ancona, 60126
Azienda Ospedaliero-Universitaria Anna Meyer
Florence, 50139
Osp. dei Bambini V. Buzzi, ASST Fatebenefratelli Sacco
Milan, 20154
Azienda Ospedaliero-Universitaria di Parma Ospedale dei Bambini Pietro Barilla, Clinica Pediatrica
Parma, 43100
IRCCS Ospedale Pediatrico Bambino Gesù
Roma, 00165
Poland4 sites
MED-POLONIA Sp.z o.o.
Poznan, 60-693
Kliniczny Szpital Wojewodzki nr 2 im. Sw. Jadwigi Krolowej w Rzeszowie
Rzeszów, 35-301
SPSK Nr 1 im. prof. Tadeusza Sokolowskiego PUM
Szczecin, 71252
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
Wroclaw, 50368
Turkey (Türkiye)4 sites
Ankara University, Faculty of Medicine
Ankara, 6100
Antalya Training and Research Hospital
Antalya, 7050
Kocaeli University Faculty of Medicine
Kocaeli, 41380
Karadeniz Technical University
Ortahisar, 61080
Georgia3 sites
JSC Maritime Hospital
Batumi, 0179
National Institute of Endocrinology
Tbilisi, 0159
TSMU Givi Jvania Pediatric Academic Clinik
Tbilisi, 0159
Serbia3 sites
University children's clinic Belgrade - Department of Endocrinology
Belgrade, 11000
Clinical Center Nis - Clinic for Children's Internal Medicine
Niš, 18000
Institute for Child and Youth Health Care of Vojvodina - Endocrinology
Novi Sad, 21000
Slovakia3 sites
National Institute of Children's Diseases
Bratislava, 83340
Children's University Hospital Kosice
Košice, 4011
National Institute of Endocrinology and Diabetology
Ľubochňa, 03491
Armenia1 site
Yerevan State Medical University after Mkhitar Heraci
Yerevan, 0024
Slovenia1 site
Univerzitetni Klinicni Center Ljubljana - Pediatrics
Ljubljana, 1000
About this study
Official title: Multicenter, Open Label Trial to Investigate the Efficacy and Safety of a Single Oral Dose of 1.0 mg/kg Macimorelin Acetate as Growth Hormone Stimulation Test (GHST) in Pediatric Patients With Suspected Growth Hormone Deficiency (GHD)
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).
Read the detailed description
Each study participant (patient) will have 5 to 6 visits in total with the study doctor.
The study will last for about 1 to 4 months, dependent on how close the visits are done. At the visits 2, 3, 4 and 5, the patient will get a stimulation test done and blood samples will be taken. At those 4 visits, the patient will have either to drink a macimorelin drink, take some clonidine tablets or get an arginine infusion. In total, the patient will get 2 macimorelin, 1 clonidine and 1 arginine test done. The level of growth hormone (GH) will be measured 4 times during the clonidine and during the arginine test and 5 times during the macimorelin test. After the test, questions on the test tolerability will be captured from patients and parents. After the arginine test, a urine dipstick test is to be done by the patient at home.
- Conditions
- Growth Hormone Deficiency
- Being tested
- Macimorelin, Arginine, Clonidine
- Keywords
- childhood-onset growth hormone deficiency, diagnosis of growth hormone deficiency, diagnosis of childhood-onset growth hormone deficiency, diagnostic test for growth hormone deficiency
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MacimorelinDrug
Also called Macrilen, AEZS-130, Macimorelin GHST, Macimorelin test
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.
ArginineDiagnostic test
Also called R-Gene 10, arginine test, arginine GHST
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.
ClonidineDiagnostic test
Also called Catapressan 75, Clonidine test, Clonidine GHST
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.
| Study group | Role | What they receive |
|---|---|---|
| standard GHST order randomized: arginine - clonidine | Active comparator | At visit 2 (V2), all subjects will perform the macimorelin GHST and will be randomized 1:1 to the order of the clonidine and arginine GHSTs at visit 3 (V3) and visit 4 (V4).
In this arm, those subjects will be presented which will have been randomized to the arginine GHST at V3 and the clonidine GHST at V4.
At visit 5 (V5) all subjects will perform the macimorelin GHST. Receives: Drug: Macimorelin; Diagnostic Test: Arginine; Diagnostic Test: Clonidine |
| standard GHST order randomized: clonidine - arginine | Active comparator | At V2, all subjects will perform the macimorelin GHST and will be randomized 1:1 to the order of the clonidine and arginine GHSTs at V3 and V4.
In this arm, those subjects will be presented which will have been randomized to the clonidine GHST at V3 and to the arginine GHST at V4.
At V5 all subjects will perform the macimorelin GHST. Receives: Drug: Macimorelin; Diagnostic Test: Arginine; Diagnostic Test: Clonidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area under the Receiver Operator Characteristic curve (ROC AUC) based on GH concentration during GHST following macimorelin administration
Assuming the outcome of GHD status adjudication final clinical diagnosis as the "true" GHD status, the diagnostic efficacy (estimated sensitivity, specificity, misclassification) of the macimorelin GHST will be based on the area under the receiver operating characteristic curve (ROC AUC).
Measured: 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)).
Secondary outcomes (3)
Sensitivity for the macimorelin GHST
Sensitivity (confirmatory secondary endpoint) will be derived from the empirical ROC plot using this GH cut-off point.
Measured: 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)).
Specificity for the macimorelin GHST
Specificity (confirmatory secondary endpoint) will be derived from the empirical ROC plot using this GH cut-off point.
Measured: 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)).
Overall agreement between the outcome of the macimorelin GHST and the combined outcome from the 2 standard GHSTs
Agreement between the outcome of macimorelin and the combined outcome of the 2 standard GHSTs will be evaluated by 'percent overall agreement'.
Measured: Visit 4 (between day 11 and day 58)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2021
- Study start16 Nov 2021
- Primary completion13 Jun 2024
- Study completion13 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Diagnosis
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- This is an open label, single-dose trial to determine the diagnostic efficacy and safety of macimorelin in pediatric subjects aged 2 to less than 18 years of age in whom, based on auxological and clinical criteria, growth hormone stimulation test (GHST) is indicated due to suspected growth hormone deficiency. Four GHSTs will be conducted during the trial: the macimorelin GHST will be performed twice, and arginine and clonidine standard GHSTs will be performed once each. The macimorelin GHST will be administered at the beginning of the trial and after serial performance of the two different standard GHSTs. The macimorelin GHSTs will be performed twice to show repeatability of the macimorelin GHST. All subjects will be randomized 1:1 to the order of the arginine and clonidine GHSTs, and in a crossover manner all subjects will receive both these GHSTs.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 101 participants (actual)
Sponsor and investigators
- Lead sponsor
- AEterna Zentaris
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nicola K Ammer, MD, Study director, AEterna Zentaris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04786873
- Sponsor's protocol ID
- AEZS-130-P02
- Other IDs
- 2018-001989-42, U1111-1248-5075, 2018
- Acronym
- DETECT
- Status last verified
- Aug 2024
- First submitted
- 3 Mar 2021
- Last updated
- 12 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Open Label Trial to Investigate the Efficacy and Safety of a Single Oral Dose of 1.0 mg/kg Macimorelin Acetate as Growth Hormone Stimulation Test (GHST) in Pediatric Patients With Suspected Growth Hormone Deficiency (GHD)
- ClinicalTrials.gov ID
- NCT04786873
- Acronym / sponsor ID
- DETECT / AEZS-130-P02
- Conditions
- Growth Hormone Deficiency
- Keywords
- childhood-onset growth hormone deficiency, diagnosis of growth hormone deficiency, diagnosis of childhood-onset growth hormone deficiency, diagnostic test for growth hormone deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 101 participants (actual)
- Lead sponsor
- AEterna ZentarisIndustry (pharma, biotech, devices)
- Study start
- 16 Nov 2021 (actual)
- Primary completion
- 13 Jun 2024 (actual)
- Study completion
- 13 Jun 2024 (actual)
- First posted
- 8 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Each study participant (patient) will have 5 to 6 visits in total with the study doctor.
The study will last for about 1 to 4 months, dependent on how close the visits are done. At the visits 2, 3, 4 and 5, the patient will get a stimulation test done and blood samples will be taken. At those 4 visits, the patient will have either to drink a macimorelin drink, take some clonidine tablets or get an arginine infusion. In total, the patient will get 2 macimorelin, 1 clonidine and 1 arginine test done. The level of growth hormone (GH) will be measured 4 times during the clonidine and during the arginine test and 5 times during the macimorelin test. After the test, questions on the test tolerability will be captured from patients and parents. After the arginine test, a urine dipstick test is to be done by the patient at home.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- This is an open label, single-dose trial to determine the diagnostic efficacy and safety of macimorelin in pediatric subjects aged 2 to less than 18 years of age in whom, based on auxological and clinical criteria, growth hormone stimulation test (GHST) is indicated due to suspected growth hormone deficiency. Four GHSTs will be conducted during the trial: the macimorelin GHST will be performed twice, and arginine and clonidine standard GHSTs will be performed once each. The macimorelin GHST will be administered at the beginning of the trial and after serial performance of the two different standard GHSTs. The macimorelin GHSTs will be performed twice to show repeatability of the macimorelin GHST. All subjects will be randomized 1:1 to the order of the arginine and clonidine GHSTs, and in a crossover manner all subjects will receive both these GHSTs.
| Arm | Type | Receives |
|---|---|---|
| standard GHST order randomized: arginine - clonidine | Active comparator | Drug: Macimorelin, Diagnostic Test: Arginine, Diagnostic Test: ClonidineAt visit 2 (V2), all subjects will perform the macimorelin GHST and will be randomized 1:1 to the order of the clonidine and arginine GHSTs at visit 3 (V3) and visit 4 (V4). In this arm, those subjects will be presented which will have been randomized to the arginine GHST at V3 and the clonidine GHST at V4. At visit 5 (V5) all subjects will perform the macimorelin GHST. |
| standard GHST order randomized: clonidine - arginine | Active comparator | Drug: Macimorelin, Diagnostic Test: Arginine, Diagnostic Test: ClonidineAt V2, all subjects will perform the macimorelin GHST and will be randomized 1:1 to the order of the clonidine and arginine GHSTs at V3 and V4. In this arm, those subjects will be presented which will have been randomized to the clonidine GHST at V3 and to the arginine GHST at V4. At V5 all subjects will perform the macimorelin GHST. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MacimorelinAlso: Macrilen, AEZS-130, Macimorelin GHST, Macimorelin testDosage 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. | Drug | standard GHST order randomized: arginine - clonidine, standard GHST order randomized: clonidine - arginine |
| ArginineAlso: R-Gene 10, arginine test, arginine GHSTFor 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 | standard GHST order randomized: arginine - clonidine, standard GHST order randomized: clonidine - arginine |
| ClonidineAlso: Catapressan 75, Clonidine test, Clonidine GHSTFor 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. | Diagnostic test | standard GHST order randomized: arginine - clonidine, standard GHST order randomized: clonidine - arginine |
Primary outcome
Area under the Receiver Operator Characteristic curve (ROC AUC) based on GH concentration during GHST following macimorelin administration
Time frame: 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)).
Assuming the outcome of GHD status adjudication final clinical diagnosis as the "true" GHD status, the diagnostic efficacy (estimated sensitivity, specificity, misclassification) of the macimorelin GHST will be based on the area under the receiver operating characteristic curve (ROC AUC).
3 secondary outcomes
Sensitivity for the macimorelin GHST
Time frame: 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)).
Specificity for the macimorelin GHST
Time frame: 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)).
Overall agreement between the outcome of the macimorelin GHST and the combined outcome from the 2 standard GHSTs
Time frame: Visit 4 (between day 11 and day 58)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nicola K Ammer, MD study director
AEterna Zentaris
45 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Macimorelin, Arginine and Clonidine in people with Growth Hormone Deficiency, run by AEterna Zentaris at 45 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 45 sites in United States, Romania, Italy, Poland, Turkey (Türkiye) and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Area under the Receiver Operator Characteristic curve (ROC AUC) based on GH concentration during GHST following macimorelin administration (time frame: 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)).). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2024.