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    Summary
    EudraCT Number:2004-002992-17
    Sponsor's Protocol Code Number:A6281271
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2015-05-14
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2004-002992-17
    A.3Full title of the trial
    EVOLUTION OF GROWTH RATE IN CHILDREN WITH GROWTH RETARDATION RELATED TO
    LONG-TERM GLUCOCORTICOSTEROID THERAPY AND TREATED BY GENOTONORM®
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    EVOLUTION OF GROWTH RATE IN CHILDRE N WITH GROWTH RETARDATION RELATED TO
    LONG-TERM GLUCOCORTICOSTEROID THERAPY AND TREATED BY GENOTONORM®
    A.4.1Sponsor's protocol code numberA6281271
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00163189
    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 SponsorPfizer, Inc.
    B.1.3.4CountryFrance
    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 supportPfizer
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer, Inc.
    B.5.2Functional name of contact pointClinicalTrials.gov Call Center
    B.5.3 Address:
    B.5.3.1Street Address235 E. 42nd Street
    B.5.3.2Town/ cityNew York, NY
    B.5.3.3Post code10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number18667181021
    B.5.5Fax number13037391119
    B.5.6E-mailClinicalTrials.gov.inquiries@pfizer.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 GENOTONORM
    D.2.1.1.2Name of the Marketing Authorisation holderPFIZER HOLDING FRANCE
    D.2.1.2Country which granted the Marketing AuthorisationDenmark
    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 nameGENOTONORM
    D.3.2Product code somotropin
    D.3.4Pharmaceutical form Powder for solution 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 INNTBD
    D.3.9.1CAS number TBD
    D.3.9.2Current sponsor codeA628
    D.3.9.3Other descriptive nameSOMATROPIN FOR INJECTION
    D.3.9.4EV Substance CodeSUB12322MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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
    Growth retardation related to long-term glucocorticoid therapy
    E.1.1.1Medical condition in easily understood language
    Evolution of growth rate in children with growth retardation related to long-term glucocorticosteroid therapy and treated by genotonorm®
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10053759
    E.1.2Term Growth retardation
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To show an increase in height after 3 years of GH treatment. However subjects will be followed for up to 5 years of treatment. Height in SD for chronological age (CA) after 3 years will be compared to Height in SD for CA before inclusion in the trial.
    E.2.2Secondary objectives of the trial
    • To estimate the evolution of height and growth rate over 1,2 and 3 years of GH treatment.
    • To estimate the prognostic factors of total height increase (in SD for CA) and growth rate (SD for CA) after 3 years of GH treatment.
    • To confirm the good clinical and biological safety of GH treatment in such children.
    • To confirm the efficacy on height after 5 years of GH treatment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Evidence of a personally signed and dated informed consent document indicating that
    the subject (or a legal representative) has been informed of all pertinent aspects of the study.
    • Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
    • Male and female subjects of childbearing potential must agree to use a highly effective method of contraception throughout the study and for 28 days after the last dose of assigned treatment. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active.
    • Female subjects who are not of childbearing potential (ie, meet at least one of the following criteria):
    • Have undergone hysterectomy or bilateral oophorectomy;
    • Have medically confirmed ovarian failure or
    • Are medically confirmed to be post menopausal (cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; laboratory confirmation LH and FSH may be indicated if menopause is in doubt.
    • Subjects who are clinically pre pubertal

    • A request for GH treatment may be issued if all the following criteria are fulfilled
    1. Bone age < 15 years for a boy and <13 years for a girl.
    2. Naïve child: Measured Height < -2 SD.
    3. Child currently treated by GH: Annual Growth Rate ≥ 0 SD for CA.
    4. Glucocorticosteroid treatment over the last 12 months at least.
    5. Glucocorticosteroid treatment is anticipated to be sustained for 1 more year at least.
    6. GH treatment request by a physician who can initiate Genotonorm® treatment according to the French SCP. That is to say a physician working in a hospital pediatric department or in a hospital endocrinology and metabolic disease department and, who is a pediatrician and/or a pediatric endocrinologist.
    7. The child benefits of the French social security cover.
    8. Evidence of a personally signed and dated informed consent document indicating that the patient's parents/guardians and from the patient himself/herself if he/she is able to receive and understand the information have been informed of all pertinent aspects of the trial.
    • GH treatment approved by the Treatment monitoring committee
    E.4Principal exclusion criteria
    1. Glucose intolerance on an Oral Glucose Tolerance Test dated less than 3 months or
    diabetes mellitus.
    2. Syndrome known to be associated with an increased risk of cancer eg, family history
    of adenomatous polyposis.
    3. Pathological condition or disease for which GH treatment is already approved in France.
    4. Participation in any other studies involving investigational or marketed products, concomitantly or within 30 days prior to entry in the study.
    5. Unable and/or unlikely to comprehend and/or follow GH treatment and/or the protocol.
    6. A previous history of intolerance or hypersensitivity to the study drug, or to drugs with
    similar chemical structures.
    7. Patients who are known or are suspected allergic to the preservative metacresol
    8. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and in the judgment of the investigator, would make the subject inappropriate for entry into this trial. Somatropin must not be used when there is any evidence of activity of a tumour. Intracranial tumours must be inactive and antitumour therapy must be completed prior to starting growth hormone therapy. Treatment should be discontinued if there is evidence of tumour growth.
    9. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the subject inappropriate for entry into this trial.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy: Difference between Height in SD for chronological age (CA) after 3 years and Height in SD for CA at Visit 2.
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 years after the visit 2.
    E.5.2Secondary end point(s)
    1. Height in cm: The height measurements are consistently performed at the same time of
    the day by using a wall mounted device (eg, Harpenden Stadiometer). The standing height of the patients is measured at each visit. Each child will be measured two times and the mean of these measurements recorded in the Case Report Form as the present height.
    2. Height in SD: Sempe reference, for bone age (BA) at each Visit.
    3. Growth rate (cm/year): If the interval between 2 available heights is less or more than
    one year, then growth rate will be supposed constant over the interval and calculated on the corresponding interval and then corrected in order to be transformed in cm/year.
    4. Growth rate SD for BA: 1st, 2nd and 3rd year.
    5. Body mass index (BMI) [weight/height² (kg/m²)] at each Visit.
    6. ∆Height in cm: Yearly measurement (cm) – baseline (cm).
    7. ∆Height in SD for CA: Yearly measurement (SD) – baseline (SD).
    8. ∆Height in SD for BA: Yearly measurement (SD) – baseline (SD).
    9. ∆BA in year: Yearly measurement (SD – baseline (SD).
    10. BA/CA: Yearly evaluation.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1, 2 and 3 years after the visit 2.
    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 No
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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 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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    France
    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.8.9.2In all countries concerned by the trial years6
    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: 97
    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: 37
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 60
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 1
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric patients; parents signed consent form when applicable.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 98
    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)
    5 visits including 3 for follow-up
    1 Subcutaneous injection daily
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: France
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