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    Summary
    EudraCT Number:2014-003556-31
    Sponsor's Protocol Code Number:SHP-607-201
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-01-21
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2014-003556-31
    A.3Full title of the trial
    Long-term Outcome of Children Enrolled in Study ROPP-2008-01 Previously Treated with rhIGF-1/rhIGFBP-3 for the Prevention of Retinopathy of Prematurity (ROP) or Who Received Standard Neonatal Care
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-term Outcome of Children Enrolled in Study ROPP-2008-01 Previously Treated with rhIGF-1/rhIGFBP-3 for the Prevention of Retinopathy of Prematurity (ROP) or Who Received Standard Neonatal Care
    A.4.1Sponsor's protocol code numberSHP-607-201
    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 SponsorPremacure AB, A Member of the Shire Group of Companies
    B.1.3.4CountrySweden
    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 supportPremacure AB, A Member of the Shire Group of Companies
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire
    B.5.2Functional name of contact pointEmily Jochim
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number1781482-9513
    B.5.6E-mailejochim@shire.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP Role
    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.3.1Product namemecasermin rinfabate
    D.3.2Product code rhIGF-1/rhIGFBP-3
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntravenous 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 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
    Retinopathy of Prematurity (ROP)
    E.1.1.1Medical condition in easily understood language
    ROP is a rare disorder of the developing retinal blood vessels and retinal neurons of the preterm infant and is one of the leading causes of preventable blindness in children
    E.1.1.2Therapeutic area Body processes [G] - Ocular Physiological Phenomena [G14]
    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 objectives of this study are:
    - To evaluate the long-term efficacy outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D) as assessed by ROP-associated visual outcomes
    - To evaluate the long-term safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D)
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate the effect following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D) on: Growth parameters; Cognitive development; Physical development; Child behavior; Pulmonary morbidity; Survival; Health-related quality of life (HRQoL); Health utility; Health care resource use (HCRU)

    The exploratory objectives of this study are to evaluate the effect following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D):
    • Brain volume and neuroanatomic abnormalities as assessed by
    magnetic resonance imaging (MRI) at PMA 40 weeks (optional)
    • Pulmonary function (optional)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Each subject must meet the following criteria to be enrolled in this study.
    1. Subject was randomized in Study ROPP-2008-01, Section D
    2. Subject’s parent or legally authorized representative(s) must provide written informed consent prior to performing any study-related activities. Study-related activities are any procedures that would not have been performed during normal management of the subject.
    E.4Principal exclusion criteria
    Subjects who meet any of the following criteria will be excluded from the study.
    1. Any other condition or therapy that, in the Investigator’s opinion, may pose a risk to the
    subject or interfere with the subject’s ability to be compliant with this protocol or interfere
    with the interpretation of results
    2. The subject or subject’s parent or legally authorized representative(s) is unable to comply
    with the protocol as determined by the Investigator
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoints of this study are:
    - Visual acuity as assessed by an age appropriate method
    - Ocular alignment and ocular motor examination in primary gaze and in as many of 9 positions of gaze as possible as assessed by corneal light reflex and by the cover test
    - Assessment of nystagmus by observation
    - Refraction as assessed by retinoscopy with cycloplegia
    - Stereoacuity as assessed with the Lang Stereotest
    E.5.1.1Timepoint(s) of evaluation of this end point
    will vary depending upon age at enrollment, but subjects will not be followed beyond age 5.5 years corrected age (CA)
    E.5.2Secondary end point(s)
    will vary depending upon age at enrollment, but subjects will not be followed beyond age 5.5 years corrected age (CA)
    The secondary efficacy endpoints of this study are:
    - Growth parameters including body weight, body length (or height), and head circumference
    - Cognitive development as assessed by the following standardized, age-appropriate tools:
    o Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)
    o Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition (WPPSI-IV)
    - Physical development as assessed by standardized, age appropriate tools including physical exam, neurological examination for assessment of cerebral palsy, and hearing assessment
    - Child behavior as assessed by the following:
    o Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
    o Child Behavior Checklist (CBCL; 1 ½ to 5)
    o Attention Deficit/Hyperactivity Disorder Rating Scale-fourth edition (ADHD RS-IV) for the assessment of symptoms of attention deficit/hyperactivity disorder (ADHD)
    o Social Communication Questionnaire (SCQ) for screening of Autism Spectrum Disorder (ASD)
    - Pulmonary morbidity data (eg, hospitalizations, emergency room [ER] visits, pulmonary medications)
    - Cerebral magnetic resonance imaging (MRI): Participation in the MRI assessment is optional and has no impact on participating in main study (volumetric analyses of all cortical gray matter, cortical white matter, corpus collosum, frontal lobes, cerebellum and total volume will be analyzed for the purposes of the study)
    - Survival as assessed by death during the study due to any cause
    - Blood pressure, heart rate and respiratory rate will be measured at the 5 year CA visit.

    Exploratory Efficacy Endpoints
    • Brain volume and neuroanatomic abnormalities as assessed with MRI
    (optional)
    • Pulmonary function, as assessed by spirometry (optional)
    The health economic outcome research endpoints of this study are:
    - Health-related quality of life (HRQoL) will be assessed by the Pediatric Quality of Life Inventory (PedsQL™) Scales appropriate for the child's age of development with the Total Scale Score and 5 domains within Physical Health (Physical Functioning and Physical Symptoms) and Psychosocial Health Scores (Emotional, Social, and Cognitive Functioning, respectively)
    - Health status (eg, health utility) will be measured by the Health Status Classification System-Preschool (HSCS-PS) and the Health Utilities Index Mark 2 (HUI2) and Mark 3 (HUI3)
    - Resource use associated with inpatient visits, outpatient visits, medical utilization and
    pharmacy utilization will be assessed

    The safety endpoints of this study are:
    - Physical examination including tonsil examination
    - Adverse events (AEs), as follows:
    o those considered related to rhIGF-1/rhIGFBP-3 (as administered in Study ROPP-2008-01, Section D)
    o those considered related to procedures performed in this study (Study SHP-607-201)
    o specified targeted medical events regardless of causality
    o fatal SAEs regardless of causality
    - Cardiac size as assessed by echocardiogram
    - Kidney and spleen size and any other gross abnormalities as assessed by abdominal ultrasound

    Exploratory Endpoints:
    The exploratory endpoints of this study are related to the topography of the retinal layers and optic nerve as assessed by optical coherence tomography (OCT).
    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 No
    E.6.5Efficacy No
    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
    long-term developmental outcome study
    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 Yes
    E.8.1.7.1Other trial design description
    long-term developmental outcome study
    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 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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Italy
    Netherlands
    Poland
    Sweden
    United Kingdom
    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
    Duration for an individual subject’s participation in the study will vary depending upon age at enrollment, but subjects will not be followed beyond age 5.5 years corrected age (CA)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months6
    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: 120
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 120
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    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 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.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 120
    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)
    Not applicable
    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 Decision2016-03-09
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-09-28
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