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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
Secondary ID [1] 0 0
2009-015985-75
Secondary ID [2] 0 0
HGT-REP-060
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

Experimental: Replagal 0.2 mg/kg EOW - Intravenous, 0.2mg/kg EOW


Treatment: Other: Replagal
0.2 mg/kg administered intravenously \[IV\] every other week
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
Timepoint [1] 0 0
Baseline to 12 months
Primary outcome [2] 0 0
Safety Evaluations
Timepoint [2] 0 0
Baseline to 12 months
Secondary outcome [1] 0 0
Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
Timepoint [1] 0 0
Baseline to 12 months
Secondary outcome [2] 0 0
Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
Timepoint [2] 0 0
Baseline to 12 months
Secondary outcome [3] 0 0
Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)
Timepoint [3] 0 0
Baseline to 12 months
Secondary outcome [4] 0 0
Change From Baseline in New York Heart Association (NYHA) Functional Class
Timepoint [4] 0 0
Baseline to 12 months
Secondary outcome [5] 0 0
Change From Baseline in Plasma Gb3
Timepoint [5] 0 0
Baseline to 12 months
Secondary outcome [6] 0 0
Change From Baseline in eGFR
Timepoint [6] 0 0
Baseline to 12 months
Secondary outcome [7] 0 0
Change From Baseline in Albumin/Creatinine (A/Cr) Ratio
Timepoint [7] 0 0
Baseline to 12 months

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Single group
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
13/04/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
8/07/2013
Sample size
Target
Accrual to date
Final
35
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Iowa
Country [2] 0 0
United States of America
State/province [2] 0 0
Virginia
Country [3] 0 0
Czechia
State/province [3] 0 0
Prague
Country [4] 0 0
Finland
State/province [4] 0 0
Turku
Country [5] 0 0
Poland
State/province [5] 0 0
Krakow
Country [6] 0 0
Poland
State/province [6] 0 0
Warsaw
Country [7] 0 0
Slovenia
State/province [7] 0 0
Slovenj Gradec
Country [8] 0 0
United Kingdom
State/province [8] 0 0
England

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

Ethics approval
Ethics application status

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).
Trial website
https://clinicaltrials.gov/study/NCT01124643
Trial related presentations / publications
Golan L, Goker-Alpan O, Holida M, Kantola I, Klopotowski M, Kuusisto J, Linhart A, Musial J, Nicholls K, Gonzalez-Rodriguez D, Sharma R, Vujkovac B, Chang P, Wijatyk A. Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease. Drug Des Devel Ther. 2015 Jul 8;9:3435-44. doi: 10.2147/DDDT.S80928. eCollection 2015.
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01124643