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   41211   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) .
     
    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:2011-004553-60
    Sponsor's Protocol Code Number:CP-4-004
    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:2012-02-09
    Trial 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 number2011-004553-60
    A.3Full title of the trial
    Safety and dose finding study of different MOD-4023 dose levels
    compared to daily r-hGH therapy in pre-pubertal growth hormone
    deficient children
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of MOD-4023 compared with daily GH therapy in children with lack of growth hormone in the body
    A.4.1Sponsor's protocol code numberCP-4-004
    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 SponsorOPKO Biologics Ltd.
    B.1.3.4CountryIsrael
    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 supportOPKO Biologics Ltd.
    B.4.2CountryIsrael
    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 pointClinical Trials Info
    B.5.3 Address:
    B.5.3.1Street Address50, Miskolci Street
    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-mailclinicaltrials@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 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 namen/a
    D.3.2Product code MOD-4023
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNno INN available yet
    D.3.9.2Current sponsor codeMOD-4023
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 Yes
    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 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 namen/a
    D.3.2Product code MOD-4023
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNno INN available yet
    D.3.9.2Current sponsor codeMOD-4023
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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 Yes
    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: 3
    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.2.1.1.1Trade name Genotropin
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameRecombinant human Growth Hormone
    D.3.4Pharmaceutical form Powder and solvent for suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNsomatropin
    D.3.9.1CAS number 12629-01-5
    D.3.9.3Other descriptive namerecombinant DNA-derived human growth hormone produced in E.coli
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5.3
    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 Yes
    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
    Pediatric Growth Hormone Deficiency
    E.1.1.1Medical condition in easily understood language
    Lack of Growth Hormone in Children
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10056438
    E.1.2Term Growth hormone deficiency
    E.1.2System Organ Class 10014698 - Endocrine disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare the safety, efficacy and tolerability of three MOD-4023
    doses to that of a commercially available standard daily recombinant human growth hormone (r-hGH) formulation, in pre-pubertal children with growth failure due to insufficient secretion of endogenous growth hormone.
    E.2.2Secondary objectives of the trial
    1.To evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) profiles of 3 different doses of MOD-4023 in pre-pubertal growth hormone deficient (GHD) children.
    2.To select the optimal dose of MOD-4023 for the subsequent phase III study on the basis of safety and efficacy.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Pre-pubertal child aged ≥ 3 yrs old and not above 10 years for girls or
    11 years for boys with either isolated GHD, or GH insufficiency as part of
    multiple pituitary hormone deficiency.
    2. Confirmed diagnosis of GHD by two different GH provocation tests
    defined as a peak plasma GH level of ≤10 ng/ml, determined by central
    laboratory using a validated assay. If the patient has already been tested
    locally and reserve samples that were taken at appropriate time-points
    are available, these will be reanalyzed by the central laboratory.
    Historical tests missing the -30 minutes time point will be accepted. If
    no reserve samples are kept (only for tests performed prior to site
    initiation), then the details of the locally performed tests will be
    reviewed by the Coordinating Investigator: if the results cannot be
    accepted, the patient will undergo both stimulation tests during the
    screening period and the samples will be analyzed by the central
    laboratory. At least one of the two stimulation tests (and preferably
    both) will be analyzed by the central laboratory. If the patient requires
    sex hormone priming (due to the age), and both stimulation tests must
    be performed during the Screening (no historical samples kept, or test
    was without priming), it is recommended to perform stimulation tests in
    consecutive setting in one day, or in two consecutive days, to avoid
    priming the patient twice. Local historical tests without sex-steroid
    priming will not be accepted for patients that require sex steroid priming
    according to the protocol.
    3.Bone age (BA) is not older than chronological age and should be no greater than 9 years for girls and 10 years for boys.
    4.Without prior exposure to any r-hGH therapy.
    5.Impaired height and height velocity defined as:
    a.Height (HT) of at least 2.0 standard deviations (SD) below the mean
    height for chronological age (CA) and gender according to the standards
    from Prader et. al, 1989 , (HT SDS ≤ -2.0).
    b.Annualized height velocity (HV) below the 25th percentile for CA (HV
    <-0.7 SDS) and gender according to the standards of Prader et al
    (1989). The interval between two height measurements should be at
    least 6 months, but should not exceed 18 months prior to inclusion.
    6.BMI must be within ±2 SD of mean BMI for the chronological age and
    sex according to the 2000 CDC standards.
    7.Baseline IGF-I level of at least 1 SD below the mean IGF-I level
    standardized for age and sex (IGF-I SDS ≤ -1.0) according to the central
    laboratory reference values;
    8.Children with normal fundoscopy (ophthalmoscopy) at screening
    (without signs/symptoms of intracranial hypertension as assessed by
    fundoscopy);
    9.Children with multiple hormonal deficiencies must be on stable
    replacement therapies for other hypothalamo-pituitary-organ axes for at
    least 3 months and 6 months for thyroid replacement therapy prior to
    the first study drug administration;
    10.Normal 46 XX karyotype for girls
    11.Written informed consent of the parent or legal guardian of the
    patient and assent of the patient (if the patient can read).
    E.4Principal exclusion criteria
    1. Children with past or present intracranial tumor growth as confirmed by an MRI scan (with contrast).
    2. History of radiation therapy or chemotherapy.
    3. Malnourished children defined as:
    a. Serum albumin below the lower limit of normal (LLN) according to the
    reference ranges of central laboratory; AND
    b. Serum iron below the lower limit of normal (LLN) according to the
    reference ranges of central laboratory; AND
    c. BMI < -2 SD for age and sex;
    4. Children with psychosocial dwarfism.
    5. Children born small for gestational age (SGA – birth weight and/or birth length < -2 SD for gestational age).
    6. Presence of anti-hGH antibodies at screening.
    7. Any clinically significant abnormality likely to affect growth or the ability to
    evaluate growth, such as, but not limited to, chronic diseases like renal
    insufficiency, spinal cord irradiation, etc.
    8. Patients with diabetes mellitus.
    9. Patients with impaired fasting sugar (based on WHO; fasting blood sugar >110 mg/dl or 6.1 mmol/l) after repeated blood analysis.
    10. Chromosomal abnormalities and medical “syndromes” (Turner’s syndrome,
    Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, Russell-Silver
    Syndrome, SHOX mutations/deletions and skeletal dysplasias), with the
    exception of septo-optic dysplasia.
    11. Closed epiphyses.
    12. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD), with the exception of hormone replacement therapies (thyroxin, hydrocortisone, desmopressin (DDAVP))
    13. Children requiring glucocorticoid therapy (e.g. asthma) that are taking a dose greater than 400 μg/d of inhaled budesonide or equivalents4 for longer than 1 month during a calendar year.
    14. Major medical conditions and/or presence of contraindication to r-hGH
    treatment.
    15. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
    16. Drug, substance, or alcohol abuse.
    17. Known hypersensitivity to the components of study medication.
    18. Other causes of short stature such as coeliac disease, hypothyroidism and rickets.
    19. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct.
    20. Participation in any other trial of an investigational agent within 30 days prior to Screening.
    E.5 End points
    E.5.1Primary end point(s)
    Annual Height Velocity in cm/year
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months after the Screening
    E.5.2Secondary end point(s)
    Auxology/Clinical:
    •Height velocity at 6 months
    •Delta height SDS at 6 and 12 months (compared to Screening value)
    Biochemical:
    •Absolute IGF-I levels on day 4 after MOD-4023 dosing
    •IGF-I SDS on day 4 after MOD-4023 dosing
    Other exploratory endpoints:
    •IGFBP-3 levels at on day 4 after MOD-4023 dosing
    •Bone maturation after 12 months of treatment
    •Predicted adult height change from start to 12 months
    PK/PD endpoints:
    PK/PD profile of MOD-4023 after 2nd dose administration at final allocated dose (AUC, MRT, Cmax, Ctrough, Tmax, T1/2)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Clinical:
    6 and 12 months after the Screening
    Biochemical:
    day 4 after MOD-4023 dosing
    Other:
    -day 4 after MOD-4023 dosing
    -after 12 months of treatment
    PK/PD endpoints:
    -after 2nd dose administration at final allocated dose
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    E.8.1.6Cross over No
    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 trial4
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    Belarus
    Bulgaria
    Czech Republic
    Georgia
    Greece
    Hungary
    Israel
    Poland
    Romania
    Russian Federation
    Slovakia
    Spain
    Ukraine
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    The end of the trial is defined as the last follow up visit of the last
    participant undergoing the trial.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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: 56
    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: 56
    F.1.1.6Adolescents (12-17 years) No
    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 No
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Children from 3 to 11 years
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 39
    F.4.2.2In the whole clinical trial 56
    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)
    none
    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 Decision2013-01-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-04-02
    P. End of Trial
    P.End of Trial StatusCompleted
    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