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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01124643
Registration number
NCT01124643
Ethics application status
Date submitted
23/04/2010
Date registered
17/05/2010
Date last updated
8/06/2021
Titles & IDs
Public title
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
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Scientific title
An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease
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Secondary ID [1]
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2009-015985-75
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Secondary ID [2]
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HGT-REP-060
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - Replagal
Experimental: Replagal 0.2 mg/kg EOW - Intravenous, 0.2mg/kg EOW
Other interventions: Replagal
0.2 mg/kg administered intravenously [IV] every other week
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
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Assessment method [1]
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Timepoint [1]
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Baseline to 12 months
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Primary outcome [2]
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Safety Evaluations
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Assessment method [2]
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Timepoint [2]
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Baseline to 12 months
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Secondary outcome [1]
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Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
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Assessment method [1]
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Timepoint [1]
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Baseline to 12 months
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Secondary outcome [2]
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Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
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Assessment method [2]
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Timepoint [2]
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Baseline to 12 months
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Secondary outcome [3]
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Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)
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Assessment method [3]
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The MLHF-Q contains 21 questions with answers ranging from 0 (no) to 5 (very much). The final score ( 0 to 105) is the sum of the points for the 21 questions. A higher score indicates a worse quality of life.
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Timepoint [3]
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Baseline to 12 months
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Secondary outcome [4]
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Change From Baseline in New York Heart Association (NYHA) Functional Class
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Assessment method [4]
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Class I: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea. Class II: Slight limitation of physical activity. Comfortable at rest, but ordinary physical activity results in fatigue, palpitation, or dyspnea. Class III: Marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV: Unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest. If any physical activity is undertaken, discomfort is increased.
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Timepoint [4]
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Baseline to 12 months
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Secondary outcome [5]
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Change From Baseline in Plasma Gb3
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Assessment method [5]
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Timepoint [5]
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Baseline to 12 months
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Secondary outcome [6]
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Change From Baseline in eGFR
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Assessment method [6]
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Timepoint [6]
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Baseline to 12 months
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Secondary outcome [7]
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Change From Baseline in Albumin/Creatinine (A/Cr) Ratio
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Assessment method [7]
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Timepoint [7]
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Baseline to 12 months
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Eligibility
Key inclusion criteria
- Complete all study requirements and assessments for Study TKT028 less than 30 days
(+/- 7 days) prior to the first dose in this extension protocol
- Voluntarily signed an Institutional Review Board/Independent Ethics Committee-
approved informed consent form after all relevant aspects of the study have been
explained and discussed.
- Has received and tolerated at least 80% of the total planned Replagal infusions in
Study TKT028
- Female participants of child-bearing potential must agree to use a medically
acceptable method of contraception at all times during the study and must have a
negative pregnancy test at the time of study entry and as required throughout
participation in study
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Has received treatment with any investigational drug (other than Replagal) or device
within 30 days prior to study entry.
- Is unable to comply with the protocol, (eg, is unable to return for safety
evaluations, or is otherwise unlikely to complete the study), as determined by the
Investigator
- Has a positive test for hepatitis B surface antigen (HBsAg), hepatitis C(HCV)
antibody, or human immunodeficiency virus (HIV) antibody
- Is pregnant or lactating
- Is morbidly obese, defined as body mass index (BMI) >39 kg/m2
- Has any safety or medical issues, as assessed by the Investigator, that contraindicate
participation in the study (eg, has experienced an adverse reaction to treatment with
Replagal or has a known hypersensitivity to any of the components of Replagal
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
13/04/2010
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
8/07/2013
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Sample size
Target
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Accrual to date
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Final
35
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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The Royal Melbourne Hospital - Parkville
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Recruitment postcode(s) [1]
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3050 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Iowa
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Country [2]
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United States of America
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State/province [2]
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Virginia
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Country [3]
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Czechia
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State/province [3]
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Prague
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Country [4]
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Finland
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State/province [4]
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Turku
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Country [5]
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Poland
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State/province [5]
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Krakow
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Country [6]
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Poland
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State/province [6]
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Warsaw
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Country [7]
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Slovenia
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State/province [7]
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Slovenj Gradec
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Country [8]
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United Kingdom
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State/province [8]
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England
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Shire
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult
participants with Fabry disease who have completed Study TKT028 (NCT00864851).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01124643
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Study Director
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Address
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Takeda
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01124643
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