Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT00864851
Registration number
NCT00864851
Ethics application status
Date submitted
18/03/2009
Date registered
19/03/2009
Date last updated
9/06/2021
Titles & IDs
Public title
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
Query!
Scientific title
A Multi-Center, Open-Label, Randomized Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in Adult Patients With Fabry Disease
Query!
Secondary ID [1]
0
0
2007-005543-22
Query!
Secondary ID [2]
0
0
TKT028
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Metabolic disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Other metabolic disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Other interventions - Replagal
Active Comparator: Replagal 0.2 mg/kg, IV, every other week - Patients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every other week for 52 weeks.
Active Comparator: Replagal 0.2 mg/kg, IV, weekly - Patients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every week for 52 weeks.
Active Comparator: Replagal 0.4 mg/kg, IV, weekly - Patients randomized to receive Replagal 0.4 mg/kg via intravenous infusion every week for 52 weeks.
Other interventions: Replagal
Intravenous (IV) infusion for 12 months
Query!
Intervention code [1]
0
0
Other interventions
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI)
Query!
Assessment method [1]
0
0
Left ventricular mass (LVM) was measured through echocardiography.
Query!
Timepoint [1]
0
0
Baseline, Month 12 (Week 53)
Query!
Secondary outcome [1]
0
0
Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise
Query!
Assessment method [1]
0
0
Exercise tolerance as measured by VO2 max at peak exercise using the standard exponential exercise protocol (STEEP).
Query!
Timepoint [1]
0
0
Baseline, Month 12 (Week 53)
Query!
Secondary outcome [2]
0
0
Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT)
Query!
Assessment method [2]
0
0
Exercise tolerance using the 6MWT was measured as the total distance walked in 6 minutes.
Query!
Timepoint [2]
0
0
Baseline, Month 12 (Week 53)
Query!
Secondary outcome [3]
0
0
Change From Baseline to Month 12 in the Minnesota Living With Heart Failure Questionnaire (MLHF-Q) Summary Score
Query!
Assessment method [3]
0
0
Quality of life (QoL) was evaluated using the MLHF-Q, version 2. The questionnaire is designed to assess the degree to which heart failure symptoms affect a patient's daily life. The summary score ranges from 0 to 105, with a score of 105 representing the highest adverse impact on a patient's QoL.
Query!
Timepoint [3]
0
0
Baseline, Month 12 (Week 53)
Query!
Secondary outcome [4]
0
0
Change From Baseline to Month 12 in New York Heart Association (NYHA) Functional Class
Query!
Assessment method [4]
0
0
The NYHA functional classification system relates symptoms to everyday activities and the patient's quality of life. NYHA Classification - The Stages of Heart Failure: Class I (Mild): No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, or dyspnea (shortness of breath). Class II (Mild): Slight limitation of physical activity. Comfortable at rest, but ordinary physical activity results in fatigue, palpitation, or dyspnea. Class III (Moderate): Marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV (Severe): Unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest. If any physical activity is undertaken, discomfort is increased.
Query!
Timepoint [4]
0
0
Baseline, Month 12 (Week 53)
Query!
Secondary outcome [5]
0
0
Change From Baseline to Month 12 in Plasma Globotriaosylceramide (GB3)
Query!
Assessment method [5]
0
0
Query!
Timepoint [5]
0
0
Baseline, Month 12 (Week 53)
Query!
Secondary outcome [6]
0
0
Change From Baseline to Month 12 in Estimated Glomerular Filtration Rate (eGFR)
Query!
Assessment method [6]
0
0
Renal function was assessed by an evaluation of change from baseline to Month 12 in eGFR as calculated using the Modification of Diet for Renal Disease (MDRD) equation.
Query!
Timepoint [6]
0
0
Baseline, Month 12 (Week 53)
Query!
Secondary outcome [7]
0
0
Change From Baseline to Month 12 in Urinary Albumin/Creatinine (A/Cr) Ratio
Query!
Assessment method [7]
0
0
Query!
Timepoint [7]
0
0
Baseline, Month 12 (Week 53)
Query!
Secondary outcome [8]
0
0
Safety Evaluation
Query!
Assessment method [8]
0
0
Adverse events were collected throughout the study, from the time of informed consent to approximately 30 days post-final infusion.
Query!
Timepoint [8]
0
0
56 Weeks
Query!
Eligibility
Key inclusion criteria
- >18 years-old;
- Male:Fabry disease confirmed by deficiency of alfa galactosidase A activity OR
Female:Fabry disease confirmed by a mutation of the alfa galactosidase A gene;
- ERT-naïve;
- LVM/h > 50g/m2.7 for males and >47 g/m2.7 for females;
- Negative pregnancy test at enrollment and contraception use required throughout study
for female patients;
- Signed informed consent;
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
- Class IV heart failure;
- Clinically significant hypertension;
- Hemodynamically significant valvular stenosis or regurgitation;
- Morbid obesity;
- Known autosomal dominant sarcoplasmic contractile protein gene mutation;
- Treatment with any investigational drug or device within the 30 days;
- Unable to comply with the protocol as determined by the Investigator;
- Positive for hepatitis B, hepatitis C or HIV
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
29/12/2008
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
5/07/2012
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
44
Query!
Recruitment in Australia
Recruitment state(s)
VIC
Query!
Recruitment hospital [1]
0
0
The Royal Melbourne Hospital - Parkville
Query!
Recruitment postcode(s) [1]
0
0
3052 - Parkville
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arizona
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Iowa
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
New York
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Virginia
Query!
Country [5]
0
0
Czechia
Query!
State/province [5]
0
0
Prague
Query!
Country [6]
0
0
Finland
Query!
State/province [6]
0
0
Turku
Query!
Country [7]
0
0
Paraguay
Query!
State/province [7]
0
0
Asuncion
Query!
Country [8]
0
0
Poland
Query!
State/province [8]
0
0
Krakow
Query!
Country [9]
0
0
Poland
Query!
State/province [9]
0
0
Warsaw
Query!
Country [10]
0
0
Slovenia
Query!
State/province [10]
0
0
Slovenj Gradec
Query!
Country [11]
0
0
United Kingdom
Query!
State/province [11]
0
0
Salford
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Shire
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The purpose of this study is to compare the safety and effectiveness of various doses of
Replagal in patients with cardiomyopathy due to Fabry disease.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT00864851
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Study Director
Query!
Address
0
0
Takeda
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT00864851
Download to PDF