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Summary
EudraCT Number:2007-006044-22
Sponsor's Protocol Code Number:HGT-ELA-038
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:2008-04-21
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
A. Protocol Information
A.1Member State ConcernedPoland - Office for Medicinal Products
A.2EudraCT number2007-006044-22
A.3Full title of the trial
A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy
A.3.2Name or abbreviated title of the trial where available
not available
A.4.1Sponsor's protocol code numberHGT-ELA-038
A.5.1ISRCTN (International Standard Randomised Controlled Trial) Numbernot available
A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
B.Sponsor: 1
B.1.1Name of SponsorShire Human Genetic Therapies, Inc. (Shire HGT)
B.1.3.4CountryUnited States
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 support
B.4.2Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisation
B.5.2Functional name of contact point
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 Elaprase 2mg/ml
D.2.1.1.2Name of the Marketing Authorisation holderShire Human Genetic Therapies AB
D.2.1.2Country which granted the Marketing AuthorisationSweden
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/3/01/078
D.3 Description of the IMP
D.3.1Product nameIdursulfase (I2S)
D.3.2Product code I2S
D.3.4Pharmaceutical form Concentrate for solution for infusion
D.3.4.1Specific paediatric formulation Information not present in EudraCT
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 INNIdursulfase
D.3.9.1CAS number 50936-59-9
D.3.9.3Other descriptive nameIdursulfase
D.3.10 Strength
D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
D.3.10.2Concentration typeequal
D.3.10.3Concentration number2
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) Information not present in EudraCT
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 Information not present in EudraCT
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
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 Information not present in EudraCT
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 Yes
D.3.11.13.1Other medicinal product typestandard recombinant product
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
Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 10.1
E.1.2Level PT
E.1.2Classification code 10056889
E.1.2Term <Manually entered code. Term in E.1.1>
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 this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years-old.
E.2.2Secondary objectives of the trial
The secondary objectives are:
• To evaluate the pharmacodynamic effects of idursulfase in these patients as measured by urinary GAG clearance
• To evaluate idursulfase single- and repeat-dose pharmacokinetic parameters in these patients at Week 1 and Week 27, respectively

Exploratory objectives are:
• To evaluate liver and spleen volumes by abdominal ultrasound
• To evaluate routine development milestones using the DDST II
• To evaluate growth velocity through height and weight measurements
• To evaluate significant clinical events that reflect disease progression in Hunter syndrome patients, e.g., the occurrence of respiratory-related events, hearing-related events, and surgical procedures
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
1. The patient has a diagnosis of Hunter syndrome based upon biochemical criteria either documented in their medical history or established at Screening:
1a.A deficiency in I2S enzyme activity of ≤ 10 % of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory)
AND
1b. A normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory
2. The patient is ≤ 5 years of age.
3. The patient is male.
4. The patient’s parent(s), or patient’s legal guardian must have voluntarily signed an Institutional Review Board approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient’s parent(s), or a the patient’s legal guardian.
E.4Principal exclusion criteria
1. The patient has received treatment with another investigational therapy within 30 days prior to enrollment.
2. The patient has clinically relevant medical condition(s) making implementation of the protocol difficult.
3. The patient has previously received idursulfase.
4. The patient has known hypersensitivity to any of the components of idursulfase.
5. The patient has had a tracheostomy.
E.5 End points
E.5.1Primary end point(s)
The primary endpoint of this study is safety as measured by incidence of adverse events (including infusion-related adverse events), changes in 12-lead electrocardiograph (ECG), vital signs, standard laboratory parameters (hematology, serum chemistry, and urinalysis), and anti-idursulfase antibody status.
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 Yes
E.6.7Pharmacodynamic Yes
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.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 Yes
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.8.3 The trial involves single site in the Member State concerned No
E.8.4 The trial involves multiple sites in the Member State concerned No
E.8.4.1Number of sites anticipated in Member State concerned1
E.8.5The trial involves multiple Member States No
E.8.5.1Number of sites anticipated in the EEA1
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 Information not present in EudraCT
E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
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.8.9.1In the Member State concerned years2
E.8.9.1In the Member State concerned months4
E.8.9.1In the Member State concerned days
E.8.9.2In all countries concerned by the trial years2
E.8.9.2In all countries concerned by the trial months4
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 Yes
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.1.1.4Infants and toddlers (28 days-23 months) Yes
F.1.1.5Children (2-11years) Yes
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 No
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
Children ≤5 years old
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state6
F.4.2 For a multinational trial
F.4.2.1In the EEA 6
F.4.2.2In the whole clinical trial 30
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)
After completion of this study, patients in countries/regions where idursulfase is commercially available will go on to receive commercial drug. Patients in countries/regions where idursulfase is not commercially available at the end of this study will be allowed to continue receiving idursulfase therapy under an expanded access program.
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 Decision2008-06-03
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 trial2011-07-08

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