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    Summary
    EudraCT Number:2004-000356-17
    Sponsor's Protocol Code Number:2-79-58035-700
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2025-07-16
    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 number2004-000356-17
    A.3Full title of the trial
    PHASE IIIB, INTERNATIONAL, SINGLE GROUP, OPEN STUDY TO DEFINE AN OPTIMAL MONITORING OF IGF-I IN CHILDREN TREATED WITH NUTROPINAQ USING A NOVEL CAPILLARY BLOOD COLLECTION METHOD (OPTIMA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Define the Optimal Insulin-like Growth Factor 1 (IGF-1) Monitoring Regimen in Children Treated with NutropinAq
    A.3.2Name or abbreviated title of the trial where available
    OPTIMA
    A.4.1Sponsor's protocol code number2-79-58035-700
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00234533
    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 SponsorBeaufour Ipsen Pharma
    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 supportBeaufour Ipsen Pharma
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBeaufour Ipsen Pharma
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street Address24, rue Erlanger
    B.5.3.2Town/ cityParis Cedex 16
    B.5.3.3Post code75781
    B.5.3.4CountryFrance
    B.5.6E-mailclinical.trials@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 holderBeaufour Ipsen Pharma
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameNutropinAq
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.11 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 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
    Growth Hormone Deficiency
    E.1.1.1Medical condition in easily understood language
    Children presenting growth failure associated with Growth Hormone Deficiency (GHD), Tuner Syndrome (TS) or Chronic Renal Insufficiency (CRI).
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To establish an optimal monitoring regimen for IGF-I in NutropinAq treated children, using a newly developed capillary blood spot IGF-I measurements technology.
    E.2.2Secondary objectives of the trial
    (1) To assess under clinical practice conditions, the biological variability of IGF-I (if any) in children treated with NutropinAq (physiological intra-individual variations, timing, dose of NutropinAq) and in different diseases conditions.
    (2) Assuming a significant biological variability was observed, to identify parameters contributing to or related to this variability, e.g., patient demographic characteristics, disease conditions, timing of last GH injection.
    (3) To further validate the filter paper versus serum method for IGF-I assay in the study setting.
    (4) To assess auxological parameters during treatment
    (5) To assess the acceptability of the NutropinAq Pen
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Children under 18 with growth failure associated with inadequate growth hormone secretion, or Turner syndrome or chronic renal insufficiency.
    E.4Principal exclusion criteria
    Children with closed epiphyses
    Children with active neoplasm
    Children with acute critical illness
    E.5 End points
    E.5.1Primary end point(s)
    (1) Timed Capillary Blood Spot IGF-I Measurements
    E.5.1.1Timepoint(s) of evaluation of this end point
    (1) At week 21, 22 and 23
    E.5.2Secondary end point(s)
    2. Factors Affecting the Variability of Timed Capillary Blood Spots: Influence of week and daily timing (evening and morning)
    3. Factors Affecting the Variability of Timed Capillary Blood Spots: Influence of sex, age and prepubertal status
    4. Factors Affecting the Variability of Timed Capillary Blood Spots: Influence of individual factors/Multivariate linear regression analysis.
    5. Factors Affecting the Variability of Timed Capillary Blood Spots: Influence of time of year and calculated age at enrolment and Disease condition.
    6. Precision Profile of Capillary Blood Random Spot versus Serum IGF-I
    7. IGFBP3 Measurements
    8. Auxological Parameter - Height
    9. Auxological Parameter - Weight
    10. Auxological Parameter - Annualised GV
    11. Acceptability and Tolerance of NutropinAq and its Administration Device: NutropinAq Pen
    12. Safety Evaluation - Posology
    13. Safety Evaluation - Extent of Exposure
    14. Auxological Parameter - Calculated Height SDS
    15. Auxological Parameter - Calculated Weight SDS
    16. Auxological Parameter - Calculated annualised GV SDS
    E.5.2.1Timepoint(s) of evaluation of this end point
    2. At week 21, 22 and 23
    3. At week 21, 22, and 23
    4. Up to week 24
    5. Up to week 24
    6. Up to week 24
    7. At Visit 1 (Day 0), Visit 2 (Month 3) and Visit 3 (Month 6)
    8. At visit 3 (month 6)
    9. At visit 3 (month 6)
    10. At visit 3 (month 6)
    11. Up to week 22
    12. Up to week 24
    13. Up to week 24
    14. At visit 3 (month 6)
    15. At visit 3 (month 6)
    16. At visit 3 (month 6)
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Acceptability of delivery device
    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.8.2.4Number of treatment arms in the trial1
    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
    Ukraine
    Russian Federation
    United Kingdom
    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 years3
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days21
    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: 251
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 3
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 145
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 103
    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 No
    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 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 251
    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)
    NA
    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: United Kingdom
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