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    Summary
    EudraCT Number:2007-001746-40
    Sponsor's Protocol Code Number:PRO-ARX201-701
    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:2007-04-02
    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 number2007-001746-40
    A.3Full title of the trial
    A Phase IIb study to Evaluate the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Profile of ARX201 Following Repeated Dosing to Young Adult Patients with Childhood Onset Growth Hormone Deficiency (GHD)
    A.4.1Sponsor's protocol code numberPRO-ARX201-701
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmbrx, Inc., USA
    B.1.3.4CountryUnited States
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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.3.2Product code ARX201
    D.3.4Pharmaceutical form Powder for injection*
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codePEG-ahGH
    D.3.9.3Other descriptive namePegylated recombinant human growth hormone
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number13.2
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 Yes
    D.3.11.13.1Other medicinal product typemodified human recombinant growth hormone conjugated to a 30,000 Dalton linear poly(ethylene) glycol.
    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
    Growth Hormone Deficiency
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10056438
    E.1.2Term Growth hormone deficiency
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the safety, tolerability, and pharmacodynamic response of three different ARX201 doses when administered repeatedly to young adult patients with childhood onset GHD
    E.2.2Secondary objectives of the trial
    - To establish the single and multidose pharmacokinetic and pharmacodynamic profiles of ARX201 administered to young adult patients with childhood onset GHD.
    - To select, based on the safety, pharmacokinetic, and pharmacodynamic findings, the dosage regimens of ARX201 that will be evaluated in subsequent multiple dose studies in patients with GHD.
    - To evaluate the efficacy of ARX201 following six months of treatment in young patients with GHD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Young adults, male or female, age 18 to 30 years, with growth
    hormone deficiency of childhood onset whom have completed growth
    (and in the investigators opinion have achieved their final height);
    2. hGH level below the predetermined cut-off value in one of the dynamic
    endocrine testing (If there are more than two tests performed all should
    have peak hGH levels below predetermined cut-off values):
    a. If Insulin Tolerance Test, the peak hGH value must be below 3
    ng/ml;
    b. If Arginine-GHRH Test, the peak hGH value must be below 5
    ng/ml. This test is however not acceptable in patients with
    GHD of hypothalamic origin (e.g. patients having received
    irradiation of the hypothalamic-pituitary region);
    c. If Arginine Test alone, the GH peak must be below 1.4 ng/ml.
    This test is acceptable only in patients in which Insulin
    Tolerance Test is contraindicated and are suffering from GHD
    of hypothalamic origin;
    OR
    d. Patients without results of any dynamic endocrine testing
    having at least 3 other pituitary hormone deficiencies;
    3. Screening IGF-I level of ≤ - 2 SDS standardised for age and sex
    according to the central laboratory reference values;
    4. rhGH treatment naïve or stopped this treatment for at least 6 months
    prior to study entry ;
    5. Receiving replacement therapies for any other hypothalamic-pituitary
    axes deficiencies for at least 3 months prior to study entry; (For
    patients receiving estrogen, ONLY transdermal application is
    acceptable. Temporary adjustment of glucocorticoid replacement
    therapy, as appropriate, is acceptable)
    6. Confirmed to be negative for anti-hGH antibodies;
    7. Females must not intend to conceive during or shortly after the study.
    They must be either post-menopausal, surgically incapable of bearing
    children, or practicing an acceptable method of birth control (e.g.,
    intrauterine device or spermicide and barrier but NOT hormonal
    contraceptives) and be willing to continue the same method of birth
    control during and for 30 days after the last dose of study medication.
    Oral contraceptives must be discontinued at least 14 days prior to
    receipt of the first dose of study drug and are not permitted during the
    study. Females of child-bearing potential must have a negative serum
    pregnancy test at screening and a negative urine pregnancy test before
    the first dose of study drug;
    8. Have negative drugs of abuse screen at Screening and on Day -1 of
    check in ;
    9. Willing and able to give informed consent; and
    10. Willing and able to undergo procedures required by this protocol.
    E.4Principal exclusion criteria
    1. Evidence of growth of pituitary adenoma or other intracranial tumor
    within the last 12 months which has to be confirmed by computer
    tomography (CT) or magnetic resonance imaging (MRI) scan (with
    contrast) within 3 months prior to study entry. Patients with primary
    growth hormone deficiency are exempt from this requirement
    2. History of malignancy other than I) cranial tumor or leukemia causing
    GHD or II) fully treated basal cell carcinoma;
    3. Subjects presenting with any clinically significant ECG abnormality,
    including a corrected QT interval (QTc) > 450 msec for males or a
    corrected QT interval (QTc) > 470 msec for females using Bazett’s
    formula ;
    4. Evidence of active malignancy or concurrent anti-tumor therapy;
    5. Evidence of intracranial hypertension;
    6. Significant hepatic dysfunction (persistent elevation of alanine
    transaminase [ALT] or aspartate transaminase [AST] >1.5 x upper limit
    of normal);
    7. Significant renal impairment as indicated by serum creatinine levels
    above the normalized range for age;
    8. Any other major medical conditions, including e.g., clinically manifest
    diabetes mellitus, hypertension, tuberculosis, major surgery within the
    last three months, or significantly abnormal laboratory tests (e.g.,
    disturbed calcium homeostasis); or any other conditions (e.g., acute
    infections) that may influence drug absorption, metabolism or excretion
    or that may interfere with any study variables in the judgment of the
    investigator;
    9. Inadequate T4 or adrenocorticoid replacement;
    10. Positive results from serology examination for HBV, HCV or HIV;
    11. History of alcohol or drug abuse as specified by the Diagnostic and
    Statistical Manual of Mental Disorders, 4th edition (DSM-IV) in the year
    before screening;
    12. Hypersensitivity to the study treatment;
    13. Systemic corticosteroids other than in replacement doses within the 3
    months before study entry. (Temporary adjustment of glucocorticoids,
    as appropriate, is acceptable);
    14. Anabolic steroids other than gonadal steroid replacement therapy
    within 2 months before study entry;
    15. History of non-compliance with medications, un-cooperativeness or
    drug abuse;
    16. Blood donation or any major blood loss >500 mL within the past 90
    days prior to study entry;
    17. History of any medical or psychiatric condition that in the opinion of the
    investigator would pose a risk for participation in this study or interfere
    with the compliance needed for this study;
    18. Females who are pregnant or breast-feeding;
    19. History of poor compliance with other chronic therapy; and
    20. Participation in any other trial of an investigational agent within 90 days
    prior to screening.
    E.5 End points
    E.5.1Primary end point(s)
    • Primary efficacy endpoint
    - Temporal profiling of circulating IGF-I levels
    • Secondary efficacy endpoints
    - Biochemical endpoints (IGF-1, and IGFBP-3 will also serve as pharmacodynamic endpoints):
    a) IGF-1 SDS; changes to baseline in IGF-I level, IGF-I SDS;
    b) IGFBP-3, IGFBP-3 SDS (actual values and changes to baseline)
    c) Lipid parameters (Cholesterol, LDL, HDL, triglycerides; actual values and changes to baseline)
    • Clinical endpoints:
    a) Change in body fat mass (FM) expressed in kg-s from the baseline to the end of study as measured with DXA
    b) Relative change in body fat,
    c) Change in trunk fat (kg-s)
    d) Relative change in trunk fat
    e) Change in lean body mass (LBM) expressed in kg-s; from the baseline to the end of study (as measured with DXA)
    f) Change in waist circumference
    g) Change in hip circumference
    h) Change in waist-to-hip ratio
    i) Change in sum of skinfolds thickness
    j) Change in BMI
    k) Change in QoL scores
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    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 No
    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 No
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 26
    F.4.2.2In the whole clinical trial 45
    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 Decision2008-08-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-08-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2010-03-30
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