임상 시험 Nct 페이지

Summary
EudraCT Number:2004-000772-14
Sponsor's Protocol Code Number:TKT027
National Competent Authority:Czechia - SUKL
Clinical Trial Type:EEA CTA
Trial Status:Completed
Date on which this record was first entered in the EudraCT database:2004-06-28
Trial results Removed from public view
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 ConcernedCzechia - SUKL
A.2EudraCT number2004-000772-14
A.3Full title of the trial
A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027)
A.3.2Name or abbreviated title of the trial where available
TKT027
A.4.1Sponsor's protocol code numberTKT027
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 SponsorTKT Inc
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 Information not present in EudraCT
D.2.1.1.1Trade name Replagal
D.2.1.1.2Name of the Marketing Authorisation holderTKT Europe-5S AB
D.2.1.2Country which granted the Marketing AuthorisationCzech Republic
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/3/00/002
D.3 Description of the IMP
D.3.1Product nameReplagal
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 INNagalsidase alfa
D.3.10 Strength
D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
D.3.10.2Concentration typeequal
D.3.10.3Concentration number1
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 Information not present in EudraCT
D.3.11.8Extractive medicinal product Information not present in EudraCT
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 typeEnzyme replacement
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
Fabry Disease
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 6.1
E.1.2Level PT
E.1.2Classification code 10016016
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 assess the pharmacodynamic effects of alternative weekly and every 2 week dosing regimens (i.e. drug given every other week) of Replagal in comparison to the current standard Replagal treatment regimen of 0.2 mg/kg every 2 weeks.
E.2.2Secondary objectives of the trial
Secondary objectives are to evaluate the safety and pharmacokinetics at each of the dose levels and regimens.
E.2.3Trial contains a sub-study Information not present in EudraCT
E.3Principal inclusion criteria
1. Subject is a male hemizygote, age 18 years or older with confirmed diagnosis of Fabry Disease. Diagnosis of Fabry disease may be confirmed by proof of a mutation of the a-Galactosidase A gene compatible with Fabry Disease and/or a deficiency of a-Galactosidase A (< 4.0 nmol/mL/hour in plasma or serum or < 8% of average mean
normal in leukocytes).

2. Subject must have one or more clinical manifestations of Fabry disease including neuropathic pain, angiokeratoma, corneal verticillata, cardiomyopathy, hypo- or anhydrosis, abdominal pain and/or diarrhea, serum creatinine > 1.0 mg/dl or proteinuria > 300 mg/24 hours.

3. Subject must have voluntarily signed an Institutional Review Board (IRB) approved
informed consent form after all relevant aspects of the study have been explained and discussed with the subject.
E.4Principal exclusion criteria
1. Subject has been previously treated with Replagal or any other enzyme replacement therapy for Fabry Disease. If the patient has previously been treated with Replagal or another enzyme replacement therapy then they must have been off the therapy for at least 30 days and must have a base-line Day -14 antibody blood sample drawn and that test must be negative for antiagalsidase alfa IgG and IgE antibodies and not experienced a prior severe infusion reactions with prior enzyme replacement therapy.

2. Subject has been enrolled in another clinical investigative study in the past 30 days

3. Subject is unable to give informed consent or is deemed unable to comply with all aspects of the clinical trial.

4. Subject has plasma Gb3 drawn on Day –14 (base-line) less than 4.0 nmol/mL.

5. Subject is undergoing dialysis or who has received a renal transplant.

6. Subjects who cannot tolerate the study procedures or who are unable or unwilling to travel to the study center as required by this protocol.

7. Subjects with an inter-current medical condition that would render them unsuitable for the study (e.g. HIV, diabetes) by confounding an assessment of the effects of the experimental therapy and its adverse events.

8. Subjects who in the opinion of the investigator (for whatever reason) are thought to be unsuitable for the study.
E.5 End points
E.5.1Primary end point(s)
The pharmacodynamic parameter to be assessed is plasma globotriaosylceramide (Gb3).

Clinical parameters including sweating, heart rate variability, proteinuria, severity of neuropathic pain, pain and anti-diarrheal medication usage, frequency and severity of abdominal pain, and frequency of diarrhea also will be assessed.
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 No
E.6.6Pharmacokinetic Yes
E.6.7Pharmacodynamic Yes
E.6.8Bioequivalence No
E.6.9Dose response Yes
E.6.10Pharmacogenetic Information not present in EudraCT
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others No
E.7Trial type and phase
E.7.1Human pharmacology (Phase I) Information not present in EudraCT
E.7.1.1First administration to humans Information not present in EudraCT
E.7.1.2Bioequivalence study Information not present in EudraCT
E.7.1.3Other Information not present in EudraCT
E.7.1.3.1Other trial type description
E.7.2Therapeutic exploratory (Phase II) Yes
E.7.3Therapeutic confirmatory (Phase III) Information not present in EudraCT
E.7.4Therapeutic use (Phase IV) Information not present in EudraCT
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 Information not present in EudraCT
E.8.1.4Double blind Information not present in EudraCT
E.8.1.5Parallel group Information not present in EudraCT
E.8.1.6Cross over Information not present in EudraCT
E.8.1.7Other Information not present in EudraCT
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 Yes
E.8.5The trial involves multiple Member States Yes
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 Information not present in EudraCT
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
Last visit
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years
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 months6
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 No
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) No
F.1.1.6Adolescents (12-17 years) No
F.1.2Adults (18-64 years) Yes
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 Information not present in EudraCT
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
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 state10
F.4.2 For a multinational trial
F.4.2.2In the whole clinical trial 20
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)
12 months compassionate use with Replagal
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 Decision2004-07-01
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2004-07-23
P. End of Trial
P.End of Trial StatusCompleted

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