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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);
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    The EU Clinical Trials Register currently displays   41210   clinical trials with a EudraCT protocol, of which   6750   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) .
     
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    Summary
    EudraCT Number:2018-001988-23
    Sponsor's Protocol Code Number:AEZS-130-P01
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-09-05
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2018-001988-23
    A.3Full title of the trial
    Open label, group comparison, dose escalation trial to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of macimorelin acetate after single oral dosing of 0.25 mg/kg, 0.5 mg/kg, and 1 mg/kg in pediatric patients with suspected growth hormone deficiency (GHD)
    Nyílt elrendezésű, összehasonlító csoportos, dózisemeléses vizsgálat a macimorelin-acetát biztonságosságának, tolerálhatóságának, farmakokinetikájának és farmakodinámiájának vizsgálatára 0,25 mg/kg-os, 0,5 mg/kg-os, és 1 mg/kg-os egyszeri orális adagolást követően a feltételezett növekedési hormon hiányban szenvedő gyermekek esetén (GHD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study with macimorelin acetate comparing three doses in their safety and tolerability in children with suspected growth hormone deficiency (GHD)
    A.4.1Sponsor's protocol code numberAEZS-130-P01
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/105/2017
    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 organisationAccelsiors CRO and Consultancy Services Ltd
    B.5.2Functional name of contact pointRegulatory Unit
    B.5.3 Address:
    B.5.3.1Street Address50 Miskolci Str
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1147
    B.5.3.4CountryHungary
    B.5.4Telephone number+3612990091
    B.5.5Fax number+3612990096
    B.5.6E-mailregulatory@accelsiors.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 Yes
    D.2.1.1.1Trade name Macrilen
    D.2.1.1.2Name of the Marketing Authorisation holderStongbridge Ireland Ltd.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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.3.4Pharmaceutical form Granules for oral suspension
    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
    D.3.9.1CAS number 381231-18-1
    D.3.9.2Current sponsor codeAEZS-130
    D.3.9.3Other descriptive nameMACIMORELIN ACETATE
    D.3.9.4EV Substance CodeSUB190334
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number63.6
    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.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 Growth Hormone Deficiency
    E.1.1.1Medical condition in easily understood language
    Diagnosis of lack of growth hormone in the body
    E.1.1.2Therapeutic area Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10073227
    E.1.2Term Growth hormone stimulation test
    E.1.2System Organ Class 100000004848
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate the safety and tolerability of macimorelin acetate after ascending single oral doses of macimorelin in pediatric patients with suspected GHD.
    E.2.2Secondary objectives of the trial
    - To investigate the pharmacokinetics (PK) of macimorelin acetate in pediatric subjects with suspected GHD;
    -To investigate the pharmacodynamics (PD) of macimorelin acetate as measured by growth hormone (GH) release in pediatric subjects with suspected GHD;
    - To explore the PK/PD relationship following single oral dose administration of macimorelin acetate in pediatric subjects with suspected GHD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female pediatric patients from 2 to less than 18 years of age;
    2. Suspected GHD based on auxological and clinical criteria;
    3. Indication for the performance of provocative GHST
    4. A subject with sex steroid priming prior to GHSTs that are part of the standard diagnostic procedures must also have sex steroid priming for the macimorelin GHST.
    E.4Principal exclusion criteria
    Lack of suitability for the trial:
    1. Established diagnosis of a disease that is sufficient to explain growth deficiency or metabolic disorders that are also associated with GHD (e.g. Turner syndrome, skeletal dysplasias, malnutrition, etc.);
    2. Ongoing GH therapy;
    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 macimorelin test day;
    4. Untreated hypogonadism or not on a stable substitution treatment within 30 days prior to the anticipated macimorelin test day;
    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. Concomitant use of a CYP3A4 inhibitor;
    8. Medical history of ongoing clinically symptomatic psychiatric disorders;
    9. Cushing disease or subjects on supraphysiologic glucocorticoid therapy within 30 days prior to the anticipated macimorelin test day;
    10. Participation in a trial with any investigational drug within 30 days prior to trial entry;
    11. Vigorous physical exercise within 24 hours prior to the macimorelin test dose.
    Safety concerns:
    12. Known hypersensitivity to any of the constituents of the macimorelin preparation;
    13. Prolonged ECG QT interval, defined as QTc > 500 msec;
    14. 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
    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;
    15. Elevation of laboratory parameters indicating hepatic or renal dysfunction or damage (AST, ALT, GGT > 2.5 x ULN; creatinine or bilirubin > 1.5x ULN);
    16. Current active malignancy other than non-melanoma skin cancer;
    17. Girls of childbearing age without effective contraception, defined as hormonal contraception or use of condom (male or female) and spermicides or use of diaphragm and spermicides or Intra Uterine Device (IUD).
    Administrative reasons:
    18. Lack of ability or willingness to give informed consent by the subject and/or his/her legal representative;
    19. Anticipated non-availability for trial visits/procedures.
    E.5 End points
    E.5.1Primary end point(s)
    SAFETY AND TOLERABILITY
    - Subjective tolerability (including acceptability of taste and impact on sleep, appetite, and gastrointestinal symptoms), adverse events (AEs);
    - Determination of changes in laboratory parameters which are relevant to safety;
    - Influence on vital parameters (pulse rate, blood pressure, electrocardiogram [ECG]).
    PHARMACOKINETICS
    - Concentration-time profiles of macimorelin;
    - Target parameters: AUC, Cmax, Tmax, T1/2.
    PHARMACODYNAMICS
    - Concentration-time profiles of GH;
    - Target parameters: Cmax, Tmax;
    - Preliminary PK/PD: Tmax for macimorelin vs. Tmax for GH; Cmax for macimorelin vs. Cmax for GH
    OTHER
    - Establishment of a recommended dose for diagnostic purposes in pediatric subjects with suspected GHD;
    - Exploration of a suitable GH cut-off point for a subsequent testing to establish the diagnosis of GHD in pediatric subjects.
    E.5.1.1Timepoint(s) of evaluation of this end point
    SAFETY AND TOLERABILITY - D1, D7+, D14+, D21+
    PHARMACOKINETICS - Blood sampling for macimorelin plasma concentrations: pre-dose (sampling time window: +/- 15 minutes, then 15, 30, 45, 60, 90, 120 (sampling time window: +/- 5 minutes) and 360 minutes (sampling time window: +/- 10 minutes) after the oral administration of the macimorelin dose.
    PHARMACODYNAMICS: - Blood sampling for GH serum concentrations: pre-dose (sampling time window: +/- 15 minutes, then 15, 30, 45, 60, 90, 120 (sampling time window: +/- 5 minutes) and 360 minutes (sampling time window: +/- 10 minutes) after the oral administration of the macimorelin dose;
    E.5.2Secondary end point(s)
    Not applicable.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable.
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    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 No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    open label, group-comparison, single dose, dose-escalation trial
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial3
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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
    Hungary
    Poland
    Russian Federation
    Serbia
    Ukraine
    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 years0
    E.8.9.1In the Member State concerned months12
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months12
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 24
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) No
    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 No
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 14
    F.4.2.2In the whole clinical trial 24
    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)
    All subjects with confirmed GHD will receive appropriate medical care per Investigators' discretion and local practice.
    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 Decision2018-11-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-01-24
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