Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
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
2 other identifiers
interventional
44
8 countries
12
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Dec 2008
Typical duration for phase_3
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 29, 2008
CompletedFirst Submitted
Initial submission to the registry
March 18, 2009
CompletedFirst Posted
Study publicly available on registry
March 19, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
July 5, 2012
CompletedResults Posted
Study results publicly available
June 6, 2014
CompletedJune 9, 2021
May 1, 2021
3.4 years
March 18, 2009
January 30, 2014
May 14, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI)
Left ventricular mass (LVM) was measured through echocardiography.
Baseline, Month 12 (Week 53)
Secondary Outcomes (8)
Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise
Baseline, Month 12 (Week 53)
Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT)
Baseline, Month 12 (Week 53)
Change From Baseline to Month 12 in the Minnesota Living With Heart Failure Questionnaire (MLHF-Q) Summary Score
Baseline, Month 12 (Week 53)
Change From Baseline to Month 12 in New York Heart Association (NYHA) Functional Class
Baseline, Month 12 (Week 53)
Change From Baseline to Month 12 in Plasma Globotriaosylceramide (GB3)
Baseline, Month 12 (Week 53)
- +3 more secondary outcomes
Study Arms (3)
Replagal 0.2 mg/kg, IV, every other week
ACTIVE COMPARATORPatients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every other week for 52 weeks.
Replagal 0.2 mg/kg, IV, weekly
ACTIVE COMPARATORPatients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every week for 52 weeks.
Replagal 0.4 mg/kg, IV, weekly
ACTIVE COMPARATORPatients randomized to receive Replagal 0.4 mg/kg via intravenous infusion every week for 52 weeks.
Interventions
Intravenous (IV) infusion for 12 months
Eligibility Criteria
You may qualify if:
- \>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;
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
Study Sites (12)
AKDHC Tucson Access Center
Tucson, Arizona, 85719, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
New York Unversity School of Medicine
New York, New York, 10016, United States
O & O Alpan, LLC
Springfield, Virginia, 22152, United States
The Royal Melbourne Hospital
Parkville, Victoria, 3052, Australia
The Charles University Hospital
Prague, 128 OO, Czechia
Turku University Central Hospital
Turku, FI-20520, Finland
Gobemador Irala y Coronel Lopez - Barrio Sojania
Asunción, Paraguay
Szpital Uniwersytecki w Krakowie
Krakow, 31-066, Poland
Instytut Kardiologii
Warsaw, 04-628, Poland
General Hospital Slovenj Gradec
Slovenj Gradec, SI 2380, Slovenia
Salford Royal NHS Foundation Trust
Salford, M6 8HD, United Kingdom
Related Publications (1)
Malek LA, Chojnowska L, Spiewak M, Klopotowski M, Misko J, Petryka J, Milosz B, Ruzyllo W. Cardiac magnetic resonance imaging in patients with Fabry's disease. Kardiol Pol. 2010 Aug;68(8):929-34.
PMID: 20730727RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Shire
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 18, 2009
First Posted
March 19, 2009
Study Start
December 29, 2008
Primary Completion
June 1, 2012
Study Completion
July 5, 2012
Last Updated
June 9, 2021
Results First Posted
June 6, 2014
Record last verified: 2021-05