Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
Secondary ID [1] 0 0
2007-005543-22
Secondary ID [2] 0 0
TKT028
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - 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.


Treatment: Other: Replagal
Intravenous (IV) infusion for 12 months
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI)
Timepoint [1] 0 0
Baseline, Month 12 (Week 53)
Secondary outcome [1] 0 0
Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise
Timepoint [1] 0 0
Baseline, Month 12 (Week 53)
Secondary outcome [2] 0 0
Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT)
Timepoint [2] 0 0
Baseline, Month 12 (Week 53)
Secondary outcome [3] 0 0
Change From Baseline to Month 12 in the Minnesota Living With Heart Failure Questionnaire (MLHF-Q) Summary Score
Timepoint [3] 0 0
Baseline, Month 12 (Week 53)
Secondary outcome [4] 0 0
Change From Baseline to Month 12 in New York Heart Association (NYHA) Functional Class
Timepoint [4] 0 0
Baseline, Month 12 (Week 53)
Secondary outcome [5] 0 0
Change From Baseline to Month 12 in Plasma Globotriaosylceramide (GB3)
Timepoint [5] 0 0
Baseline, Month 12 (Week 53)
Secondary outcome [6] 0 0
Change From Baseline to Month 12 in Estimated Glomerular Filtration Rate (eGFR)
Timepoint [6] 0 0
Baseline, Month 12 (Week 53)
Secondary outcome [7] 0 0
Change From Baseline to Month 12 in Urinary Albumin/Creatinine (A/Cr) Ratio
Timepoint [7] 0 0
Baseline, Month 12 (Week 53)
Secondary outcome [8] 0 0
Safety Evaluation
Timepoint [8] 0 0
56 Weeks

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;
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/12/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/07/2012
Sample size
Target
Accrual to date
Final
44
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Iowa
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Virginia
Country [5] 0 0
Czechia
State/province [5] 0 0
Prague
Country [6] 0 0
Finland
State/province [6] 0 0
Turku
Country [7] 0 0
Paraguay
State/province [7] 0 0
Asuncion
Country [8] 0 0
Poland
State/province [8] 0 0
Krakow
Country [9] 0 0
Poland
State/province [9] 0 0
Warsaw
Country [10] 0 0
Slovenia
State/province [10] 0 0
Slovenj Gradec
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Shire
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Takeda
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

TypeCitations or Other Details
Journal Malek LA, Chojnowska L, Spiewak M, Klopotowski M, ... [More Details]

Results are available at https://clinicaltrials.gov/study/NCT00864851