Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   41225   clinical trials with a EudraCT protocol, of which   6755   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2015-002337-22
    Sponsor's Protocol Code Number:AEZS-130-052
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-02-01
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2015-002337-22
    A.3Full title of the trial
    Confirmatory validation of oral macimorelin as a Growth Hormone (GH) Stimulation Test (ST) for the diagnosis of Adult Growth Hormone Deficiency (AGHD) in comparison with the Insulin Tolerance Test (ITT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Comparison of the substance macimorelin (drinking solution) with insulin (injection) to confirm that it works as diagnostic test for lacking growth hormone in adults
    A.3.2Name or abbreviated title of the trial where available
    Validation of macimorelin as a test for Adult Growth Hormone Deficiency
    A.4.1Sponsor's protocol code numberAEZS-130-052
    A.5.4Other Identifiers
    Name:IND numberNumber:073196
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAeterna Zentaris GmbH
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAeterna Zentaris GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAeterna Zentaris GmbH
    B.5.2Functional name of contact pointClinical trial information desk
    B.5.3 Address:
    B.5.3.1Street AddressWeismuellerstraße 50
    B.5.3.2Town/ cityFrankfurt am Main
    B.5.3.3Post code60314
    B.5.3.4CountryGermany
    B.5.4Telephone number+4969426023472
    B.5.5Fax number+4969426023404
    B.5.6E-mailclinical.trials@aezsinc.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMacimorelin
    D.3.2Product code AEZS-130
    D.3.4Pharmaceutical form Granules for oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMacimorelin acetate
    D.3.9.1CAS number 945212-59-9
    D.3.9.2Current sponsor codeAEZS-130
    D.3.9.3Other descriptive nameMACIMORELIN ACETATE
    D.3.9.4EV Substance CodeSUB177751
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number69.9 to 72.3
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameInsulin
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNInsulin
    D.3.9.1CAS number 9004-10-8
    D.3.9.2Current sponsor codeInsulin
    D.3.9.3Other descriptive nameINSULIN
    D.3.9.4EV Substance CodeSUB02689MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number0.10 to 0.20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Diagnosis of Adult Growth Hormone Deficiency (AGHD)
    E.1.1.1Medical condition in easily understood language
    Diagnosis of lacking growth hormone in adults
    E.1.1.2Therapeutic area Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To validate the use of single dose oral macimorelin for the dagnosis of AGHD ('macimorelin GHST'), using the Insulin Tolerance Test (ITT) as comparator (non-reference standard) GHST.
    E.2.2Secondary objectives of the trial
    To characterize the safety profile of single dose oral macimorelin in suspected AGHD subjects
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female, aged between 18 and 65 years.
    2. Suspected growth hormone deficiency (GHD), based on either of the following:
    - structural hypothalamic or pituitary disease, or
    - surgery or irradiation in these areas, or
    - head trauma as an adult, or
    - evidence of other pituitary hormone deficiencies, or
    - idiopathic childhood onset GHD (without known hypothalamic or pituitary lesion or injury).
    OR (recruitment at dedicated trial sites only)
    3. Group D: Healthy* control
    Subject matching a Group A subject by
    - sex, age (+/- 5 years), BMI (+/- 2 kg/m2), and estrogen status** (women only);
    * ‘healthy’ comprising:
    - history of normal growth and development,
    - serum prolactin concentration within normal range limits,
    - history of regular, age-appropriate menses in females
    - serum testosterone concentration within normal range limits for males.
    ** matching for ‘estrogen status’:
    - Group A subjects below 50 years and on oral estrogen therapy will be matched to control subjects who are also taking estrogen (as an oral contraceptive or for replacement); the route of estrogen administration (e.g. oral vs transdermal) must also be matched.
    - Group A subjects 50 years or older and with untreated estrogen deficiency will be matched to female control subjects who are not receiving estrogen.
    E.4Principal exclusion criteria
    Lack of suitability for the trial:
    1. GH therapy within 1 month prior to anticipated first GHST within this trial (within 3 months in case of long-acting GH formulation).
    Note: A patient with ongoing GH therapy will not be a candidate for this trial, if a corresponding treatment-free interval is not medically justifiable.
    2. GHST within 7 days prior to the anticipated first test day within the trial.
    3. Subjects with a medical history and clinical signs of a not adequately treated thyroid dysfunction, or subjects who had a change in thyroid therapy within 30 days prior to anticipated first test day within the trial.
    4. Untreated hypogonadism or not on a stable substitution treatment within 30 days prior to anticipated first test day within the trial.
    Note: Postmenopausal status will not be considered as an exclusion.
    5. 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 (atropine).
    6. Concomitant use of a CYP3A4 inducer (e.g., carbamazepine, phenobarbital, phenytoin, pioglitazone, rifabutin, rifampin, St. John's Wort).
    7. Medical history of ongoing clinically symptomatic severe psychiatric disorders.
    8. Parkinson’s disease.
    9. Cushing disease or patients on supraphysiologic glucocorticoid therapy within 30 days prior to the anticipated first test day within the trial.
    10. Type 1 diabetes or untreated or poorly controlled Type 2 diabetes, as defined by HbA1c > 8%.
    11. Body mass index (BMI) ≥ 40.0 kg/m2.
    12. Participation in a trial with any investigational drug within 30 days prior to trial entry.
    13. Vigorous physical exercise within 24 hours prior to each GHST within this trial.

    Safety concerns:
    14. Known hypersensitivity to macimorelin or insulin, or any of the constituents of either preparation.
    15. Clinically significant cardiovascular or cerebrovascular disease.
    16. Prolonged ECG QT interval, defined as QTc > 500 msec.
    17. Concomitant treatment with any drugs that might prolong QT/QTc (see list of drugs at http://www.torsades.org/medical-pros/drug-lists/printable-drug-list.cfm (registration required), a copy of which is included in the Investigator’s File).
    Note: A patient who receives such treatment will not be a candidate for this trial, 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.
    18. Elevation of laboratory parameters indicating hepatic or renal dysfunction or damage (ASAT, ALAT, GGT > 2.5 x ULN; creatinine, or bilirubin > 1.5x ULN).
    19. Medical history of seizure disorders.
    20. Known immunosuppression.
    21. Current active malignancy other than non-melanoma skin cancer.
    22. Breastfeeding or positive urine pregnancy test (for women of childbearing potential only).
    23. Women of childbearing age without contraception, such as hormonal contraception or use of condom and spermicides or use of diaphragm and spermicides or Intra Uterine Device (IUD).

    Administrative reasons:
    24. Lack of ability or willingness to give informed consent.
    25. Anticipated non-availability for trial visits/procedures.
    E.5 End points
    E.5.1Primary end point(s)
    Co-Primary efficacy variables will be 'Percent Negative Agreement' and 'Percent Positive Agreement' when using predefined cut-points of both GHSTs.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Evaluation of the Primary endpoints will be performed at two timepoints (i.e. during two separate visits per Growth Hormone Stimulation Test): For Test 1 on Day 1, for Test 2 on D8-28 (i.e. on one day within a time period from Day8 to Day28)
    E.5.2Secondary end point(s)
    Secondary efficacy criteria will be 'Percent Overall Agreement' and estimated sensitivity and specificity of both GHSTs when using predefined cut-points of both GHSTs.

    Safety:
    Adverse events, clinical laboratory and 12-lead ECG.

    Other:
    Test acceptance/Preference by Trial subjects and investigators.
    Preliminary PK: Tmax and Cmax of macimorelin Plasma concentrations in the sampling period.
    Preliminary PK/PD: Tmax for macimorelin vs Tmax for GH; Cmax for macimorelin vs Cmax for GH.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Evaluation of the secondary endpoints will be:
    - efficacy related at two timepoints (i.e. during two separate visits per Growth Hormone Stimulation Test), as mentioned in Section E.5.1.1: For Test 1 on Day 1, for Test 2 on D8-28 (i.e. on one day within a time period from Day8 to Day28)
    - safety related at four timepoints, i.e. at all four visits of this Trial (Screening visit, Test 1, Test 2, End-of-Trial visit)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA24
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Austria
    France
    Germany
    Italy
    Poland
    Serbia
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 110
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers Yes
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 110
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    The trial is being performed within the diagnostic work-up of subjects suspected to have AGHD, by applying the experimental assay in addition to the diagnostic standard. The acutal conclusion on the AGHD diagnosis and the implementation of treatment, if needed, is not subject of this trial.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-04-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-02-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-11-29
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA