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    EudraCT Number:2019-001020-36
    Sponsor's Protocol Code Number:MS301
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-05-31
    Trial results View results
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    A. Protocol Information
    A.2EudraCT number2019-001020-36
    A.3Full title of the trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of Children With Growth Failure Associated With Primary IGF-1 Deficiency
    A.4.1Sponsor's protocol code numberMS301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00125164
    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 Pharma
    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 Pharma
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIpsen Pharma
    B.5.2Functional name of contact pointGlobal Medical Affairs
    B.5.3 Address:
    B.5.3.1Street Address65 quai Georges Gorse
    B.5.3.2Town/ cityBoulogne Billancourt Cedex
    B.5.3.3Post code92650
    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. name Increlex
    D. of the Marketing Authorisation holderIpsen Pharma
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/05/307
    D.3 Description of the IMP
    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 INNMECASERMIN
    D.3.9.1CAS number 68562-41-4
    D.3.9.3Other descriptive namerhIGF-1
    D.3.9.4EV Substance CodeSUB08672MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Children with growth failure associated with primary insulin-like growth factor deficiency (IGFD).
    E.1.1.1Medical condition in easily understood language
    Growth Disorder
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to demonstrate the safety and efficacy of rhIGF-1 in promoting the growth of children with growth failure associated with Primary IGFD.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are to:
    1. Demonstrate the dose-response relationship between rhIGF-1 and linear growth in prepubertal children
    2. Identify a dose range with an acceptable risk-to-benefit profile
    3. Assess the treatment-outcome predictive powers of an IGF generation test
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - IGF-1 deficiency as evidenced by an IGF-1 SD score of < –2.0 for age and gender at Visit 1, using provided normal data; or as the average of the IGF-1 SD score at Visit 1 and the most recent prior IGF-1 SD score from an approved IGF-1 assay.
    - Short stature as defined by a height SD score of < –2.0 for age and gender using provided normal data
    - Chronological age equals or more than 3 years and chronological or bone age equals or less than 11 in girls and equals or less than 12 in boys (if bone age alone was used, the bone age must have been taken within 6 months of screening)
    - GH sufficiency, defined at Visit 2 as a maximal arginine-clonidine stimulated GH response of equials or more than 7 ng/mL (or with other stimulants approved by the Sponsor).
    - Prepubertal Status: Girls were at less than Tanner stage 2 breast development and boys had testicular volume equials or more than 3 mL, as measured by the standardized orchidometer (Prader type). The onset of pubic hair development, up to and including Tanner stage 2, was allowed at study entry.
    - Adequate nutrition as evidenced by a body mass index greater than or equal to the 5th percentile for age and gender using provided normal data
    E.4Principal exclusion criteria
    - Documented mutation of the GH receptor (Laron syndrome) or documented deletion of the GH gene
    - Any previous therapeutic use of IGF-1, GH, Lupron®, or sex steroids. A short-term (i.e., 7 days or fewer) exposure to GH for a diagnostic test was permitted.
    - Known or suspected allergy to trial product or related products
    - Chronic illness, including diabetes mellitus, inborn errors of metabolism, osteo- or chondrodystrophies, disease of the genitourinary, cardiopulmonary,
    gastrointestinal, or central nervous system.
    - Chromosomal aneuploidy (eg, Down syndrome, trisomies, Turner syndrome, etc.)
    - Syndromes with known predisposition to develop malignant tumors (eg, Bloom syndrome, Fanconi syndrome)
    - Prior bone marrow transplantation
    - Any named syndrome known associated with short stature, including but not
    limited to Prader-Willi syndrome, Russell-Silver syndrome, Turner syndrome, and Noonan syndrome
    - Active seizure disorders, defined as one or more seizures per month, irrespective of anticonvulsant therapy
    - Administration of any growth-altering medication within 3 months of Visit 1
    - Significant abnormality in clinical screening laboratories, as determined by the investigator
    - Any current or previous exposure to therapeutic spinal irradiation
    - Evidence of clinical malnutrition or growth deficit attributable to emotional deprivation
    - Evidence of any active malignancy or intracranial tumors
    - Use of investigational medication(s) within the last month
    - Any other social or medical condition that, in the opinion of the investigator,
    would be detrimental to either the subject or the study.
    E.5 End points
    E.5.1Primary end point(s)
    First-year height velocity
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline (Visit 4) to 12 months (Visit 9)
    E.5.2Secondary end point(s)
    Change in height SD score, bone age, changes in serum concentrations of IGF-1, IGF-2, insulin-like growth factor binding protein-2 (IGFBP-2), and insulin-like growth factor binding protein-3 (IGFBP-3)
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline (Visit 4) to 12 months (Visit 9)
    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 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. 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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E. description
    Untreated control group
    E.8.2.4Number of treatment arms in the trial3
    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 Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    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.8.9.2In all countries concerned by the trial years1
    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: 137
    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. of subjects for this age range: 70
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 67
    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 137
    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)
    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 States
    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
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