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Summary
EudraCT Number:2007-007872-40
Sponsor's Protocol Code Number:ROPP-2008-01
National Competent Authority:Sweden - MPA
Clinical Trial Type:EEA CTA
Trial Status:Temporarily Halted
Date on which this record was first entered in the EudraCT database:2008-09-22
Trial 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
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A. Protocol Information
A.1Member State ConcernedSweden - MPA
A.2EudraCT number2007-007872-40
A.3Full title of the trial
Determination of the rhIGF-I/rhIGFBP-3 Dose, Administered as a Continuous Infusion, Required to Establish and Maintain Longitudinal Serum IGF-I Levels within Physiological Levels in Premature Infants, to prevent Retinopathy of Prematurity

A Phase II, Randomized Controlled, Assessor-blind, Dose-Confirming, pharmacokinetic, Safety and Efficacy, Multicenter Study.

C O N F I D E N T I A L
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
Phase II multicenter trial regarding pharmacokinetics, safety and efficacy of continuous infusion of IGF-I to premature infants to prevent the onset of retinopathy of prematurity
A.3.2Name or abbreviated title of the trial where available
ROP Phase-II - C O N F I D E N T I A L
A.4.1Sponsor's protocol code numberROPP-2008-01
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 Human Genetic Therapies Inc.
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 number+17814829513
B.5.6E-mailejochim@shire.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 No
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/3/06/399
D.3 Description of the IMP
D.3.1Product namerhIGF-I/rhIGFBP-3
D.3.4Pharmaceutical form Solution for injection/infusion
D.3.4.1Specific paediatric formulation Yes
D.3.7Routes of administration for this IMPIntravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8INN - Proposed INNNot known
D.3.9.2Current sponsor codeSHP-607
D.3.9.3Other descriptive namerhIGF-I/rhIGFBP-3
D.3.10 Strength
D.3.10.1Concentration unit µg/ml microgram(s)/millilitre
D.3.10.2Concentration typeequal
D.3.10.3Concentration number50
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
Damage to the retina of the eye in preterm infants
E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 16.1
E.1.2Level SOC
E.1.2Classification code 10015919
E.1.2Term Eye disorders
E.1.2System Organ Class 10015919 - Eye disorders
E.1.3Condition being studied is a rare disease Yes
E.2 Objective of the trial
E.2.1Main objective of the trial
Section A:To establish the dose and sampling schedule to be used in Study Sections B and C
Sections B and C: To determine the dose of IMP, administered by continuous IV infusion, required to reach and maintain a physiological range of serum IGF-1 of
20-60 μg/L, defined as the in utero levels of IGF-1 for corresponding GA
in a normal population. To determine serum concentrations of IGF-1 and associated pharmacokinetic parameters after continuous IV infusion of IMP
Section D: To determine the effect of IMP on the severity of ROP as compared to the severity of ROP in an untreated control population. To evaluate the dose of IMP administered by continuous IV infusion, required to reach and maintain a physiological range of serum IGF-1 of 28-109 μg/L To determine serum concentrations of IGF-1 and associated pharmacokinetic parameters after continuous IV infusion of IMP. To determine serum concentration of IGFBP-3 and acid labile subunit (ALS) after continuous IV infusion of IMP
E.2.2Secondary objectives of the trial
To determine the effect of rhIGF-1/rhIGFBP-3 on other efficacy
parameters and determine the safety profile of rhIGF-1/rhIGFBP-3 when
compared with standard neonatal care in preterm infants
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- A signed written informed consent from the subject's parents/guardians prior to any study-related procedures that has been
approved by the Institutional Review Board (IRB)/Independent Ethics
Committee (IEC)
2- Subject must be between GA of 26 weeks + 0 days and 27 weeks +6
days (Study Section A) or between GA of 23 weeks + 0 days and 27
weeks + 6 days (Study Sections B, C, and D), inclusive
E.4Principal exclusion criteria
Subjects who meet any of the following criteria will be excluded from the
study:
1- Subjects born small for gestational age (SGA), ie, weight at birth <-2
SDS (Study Section A only)
2- Detectable gross malformation
3- Known or suspected chromosomal abnormality, genetic disorder, or
syndrome, according to the investigator's opinion
4- Persistent plasma glucose level <2.5 mmol/L or >10 mmol/L at Study
Day 0 (day of birth) to exclude severe congenital abnormalities of
glucose metabolism
5- Anticipated need of administration of erythropoietin (rhEPO) during
treatment with study drug
6- Maternal history of gestational diabetes or any diabetes requiring
insulin while pregnant
7- Clinically significant neurological disease according to the
investigator's opinion (Stage 1 IVH allowed)
8- 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 interpretation of results
9- Monozygotic twins
10- Subject participating or plans to participate in a clinical study of
another investigational study drug
E.5 End points
E.5.1Primary end point(s)
Sections A, B, C: Primary endpoint: Severity of ROP
Sections D: Primary endpoint: Maximum severity of ROP stage across all
retinal examinations
E.5.1.1Timepoint(s) of evaluation of this end point
First ROP examination shall occur at 5 to 6 weeks or PMA 31 weeks.
Follow-up examination should occur at least 1 to 2 weeks depending
upon the clinical evaluation of the paediatric ophtalmologist.
Final ROP assessment will occur at 40 weeks +/- 4 days.
E.5.2Secondary end point(s)
The key secondary endpoint of this study is in Section D: time to discharge from the neonatal intensive care (TDNIC).
E.5.2.1Timepoint(s) of evaluation of this end point
TDNIC will be calculated from the day of birth to the day of the discharge
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 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) Yes
E.7.3Therapeutic confirmatory (Phase III) No
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 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
Assessor blind and comparison with untreated control group.
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.8.2.3.1Comparator description
Serum concentration of IGF-1 in untreated controls
E.8.2.4Number of treatment arms in the trial1
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 Yes
E.8.5.1Number of sites anticipated in the EEA20
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 Yes
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.8.9.1In the Member State concerned years4
E.8.9.1In the Member State concerned months
E.8.9.1In the Member State concerned days
E.8.9.2In all countries concerned by the trial years4
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) Yes
F.1.1.2.1Number of subjects for this age range: 120
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) Yes
F.1.1.4.1Number of subjects for this age range: 120
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 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
Premature infants - informed consent will be obtained by parents
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state30
F.4.2 For a multinational trial
F.4.2.1In the EEA 120
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)
None
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 Decision2009-07-29
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2009-05-18
P. End of Trial
P.End of Trial StatusTemporarily Halted
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