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    Summary
    EudraCT Number:2010-019980-13
    Sponsor's Protocol Code Number:A-95-58035-017
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2011-07-11
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2010-019980-13
    A.3Full title of the trial
    Predictive value of baseline and stimulated serum IGF-I and IGFBP-3 during a dose-escalation IGF-I generation test with NutropinAq for the 1 year growth response to growth hormone (GH) therapy in short children with low IGF-I and a normal GH peak in a provocation test
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Predictive value of the IGF-I generation test to predict the results of growth hormone treatment in short children.
    A.3.2Name or abbreviated title of the trial where available
    IGF-I generation test
    A.4.1Sponsor's protocol code numberA-95-58035-017
    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 SponsorIpsen Farmaceutica b.v.
    B.1.3.4CountryNetherlands
    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 supportIpsen Farmaceutica b.v.
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIpsen farmaceutica b.v.
    B.5.2Functional name of contact pointAdrie Stoffels
    B.5.3 Address:
    B.5.3.1Street AddressTaurusavenue 33B
    B.5.3.2Town/ cityHoofddorp
    B.5.3.3Post code2132 LS
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+3123554 1600
    B.5.5Fax number+3123554 1609
    B.5.6E-mailadrie.stoffels@ipsen.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 NutropinAq
    D.2.1.1.2Name of the Marketing Authorisation holderIpsen
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameNutropin Aq
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPSubcutaneous use
    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
    Severe idiopathic short stature
    E.1.1.1Medical condition in easily understood language
    short stature for no clear reason
    E.1.1.2Therapeutic area Body processes [G] - Bones and nerves physological processes [G11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level LLT
    E.1.2Classification code 10066333
    E.1.2Term Idiopathic short stature
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assessment of the value of the short-term (2 weeks) response of IGF-I (peak IGF-I SDS) to GH in a dosage of 1.4 mg/m2/day (as part of a dose-escalation IGF-I generation test) in comparison to baseline IGF-I to predict the 1 year growth response (expressed as studentized residual for children with ISS) to GH in a dose of 1.4 mg/m2/day.
    E.2.2Secondary objectives of the trial
    Assessment of the value of other parameters of the dose-escalation IGF-I generation test (peak values and changes versus baseline of IGF-I, IGFBP-3, IGF-I/IGFBP-3 ratio, and IGF-I/IGFBP-2 ratio) in comparison to baseline IGF-I to predict the 1 year growth response (expressed as studentized residual for children with ISS) to GH in a dose of 1.4 mg/m2/day.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Prepubertal children aged 2.0-9.0 (females) or 2.0-10.0 (males) years, bone age <9 (females) or <10 (males) years, height SDS<-2.5 (for ethnically adequate references), peak GH after provocation >30 mU/L, IGF-I SDS < -2 (at least twice, one of which determined in UMCU), body mass index (BMI) SDS > -2
    E.4Principal exclusion criteria
    • Has a history of hypersensitivity to growth hormone or phenol (conservative added to GH in NutropinAq), or drugs with a similar chemical structure.
    • Was treated with any other Investigational Medicinal Product (IMP) within the last 30 days before study entry.
    • Has any mental condition rendering the subject unable to understand the nature, scope and possible consequences of the study, and/or evidence of an uncooperative attitude.
    • Has abnormal baseline findings, any other medical condition(s) or laboratory findings that, in the opinion of the Investigator, might jeopardise the subject’s safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
    • Has a birth weight and/or length below -2 SDS for Swedish reference charts (2). Patients will not be excluded due to an unknown birth weight or length.
    • Has a known cause of short stature, or any significant concomitant disease that is likely to interfere with growth or with the study schedule/objectives, or is a known contraindication to GH treatment such as: chromosomal abnormalities (known syndromes associated with short stature, and skeletal dysplasias); growth failure due to high doses of glucocorticosteroids, hypothyroidism, chronic illnesses; malignancy, intra-cranial tumor; chronic disease such as insulin-dependent diabetes mellitus; chronic infectious disease; chronic renal insufficiency; chronic heart failure; chronic hepatic disease; celiac disease; chronic pulmonary disease; active rheumatic disease; psychosis; neurofibromatosis; McCune Albright syndrome; dysmorphic syndromes such as Russell-Silver syndrome, Leri-Weill syndrome, achondroplasia, etc; and emotional deprivation. Hypothyroidism adequately substituted with thyroid hormone replacement therapy is not an exclusion criterion. For girls, the karyotype, to eliminate a Turner syndrome, is mandatory. In case of a positive Rappold score (> 4) (3), a SHOX defect has to be excluded.
    • Has dysmorphic features suspect for chromosomal breakage syndromes.
    • Has known causes of decreased IGF-I (e.g. undernutrition).
    • Has an abnormal sitting height:height ratio SDS (<-2 or >+2), corrected for bone age, according to Dutch references (4).
    • Has any abnormality at laboratory screening according to the Dutch consensus protocol (see appendix 3).
    • Is likely to require treatment during the study with drugs that are not permitted by the study protocol (see appendix 4).
    • Has active neoplasia or suspected neoplasia.
    • The parents are investigator site personnel directly affiliated with the study, or are the immediate family of investigator site personnel directly affiliated with the study. Immediate family is defined as spouse, parent, child, or sibling, whether biological or legally adopted.
    • Is unable or unwilling to comply with the study visits or the test schedule required by the protocol.
    • Patients not affiliated to the health insurance system will not be allowed to participate in this trial.
    E.5 End points
    E.5.1Primary end point(s)
    Assessment of the value of the short-term (2 weeks) response of IGF-I (peak IGF-I SDS) to GH in a dosage of 1.4 mg/m2/day (as part of a dose-escalation IGF-I generation test) in comparison to baseline IGF-I to predict the 1 year growth response (expressed as studentized residual for children with ISS) to GH in a dose of 1.4 mg/m2/day
    E.5.1.1Timepoint(s) of evaluation of this end point
    At 19.5 months (after screening and treatment of the patients).
    E.5.2Secondary end point(s)
    - peak IGF-I after 2 weeks on GH 1.4 mg/m2/day
    -Delta IGF-I SDS on 0.7 mg GH/m2/day for 2 weeks
    -Delta IGF-I SDS on 1.4 mg GH/m2/day for 2 weeks
    -Delta IGFBP-3 SDS on 0.7 mg GH/m2/day for 2 weeks
    -Delta IGFBP-3 SDS on 1.4 mg GH/m2/day for 2 weeks
    -Delta of the ratio IGF-I:IGFBP-3 SDS on 0.7 mg GH/m2/day for 2 weeks
    -Delta of the ratio IGF-I:IGFBP-3 SDS on 1.4 mg GH/m2/day for 2 weeks
    -Delta of the ratio IGF-I:IGFBP-2 SDS on 0.7 mg GH/m2/day for 2 weeks
    -Delta of the ratio IGF-I:IGFBP-2 SDS on 1.4 mg GH/m2/day for 2 weeks
    -Peak IGF-I SDS on 0.7 mg GH/m2/day for 2 weeks
    -Peak IGFBP-3 SDS on 0.7 mg GH/m2/day for 2 weeks
    -Peak IGFBP-3 SDS on 1.4 mg GH/m2/day for 2 weeks
    -Peak of the ratio IGF-I:IGFBP-3 SDS on 0.7 mg GH/m2/day for 2 weeks
    -Peak of the ratio IGF-I:IGFBP-3 SDS on 1.4 mg GH/m2/day for 2 weeks
    -Peak of the ratio IGF-I:IGFBP-2 SDS on 0.7 mg GH/m2/day for 2 weeks
    -Peak of the ratio IGF-I:IGFBP-2 SDS on 1.4 mg GH/m2/day for 2 weeks
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 2 weeks after start treatment of the patients
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 Information not present in EudraCT
    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 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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    The Sponsor may terminate this study at any time. Reasons for termination may include, but are not limited to, the following:
    • The incidence or severity of adverse events (AE) in this or other studies point to a potential health hazard for trial subjects.
    • Insufficient subject enrolment.
    • Any information becoming available during the study that substantially changes the expected benefit risk profile of the study treatments.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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: 20
    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: 20
    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 Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Small children
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    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)
    If patietns respond during the trial, but do not fullfill the Dutch criteria for treatment, Ipsen will provide the medication free of charge.
    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 Decision2011-07-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-05-14
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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