This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
HGT-REP-060 was designed to provide participants who participated in TKT028 an additional 52 weeks of treatment with Replagal at the standard dose of 0.2 milligram per kilogram (mg/kg) every other week (EOW) to assess the effect of continued Replagal therapy on long-term safety and clinical outcomes for this participant population.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
35
0.2 mg/kg administered intravenously \[IV\] every other week
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
O&O Alpan LLC
Fairfax, Virginia, United States
The Royal Melbourne Hospital
Parkville, Australia
1st School of Medicine Charles University
Prague, Czechia
Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
Time frame: Baseline to 12 months
Safety Evaluations
Time frame: Baseline to 12 months
Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
Time frame: Baseline to 12 months
Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
Time frame: Baseline to 12 months
Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)
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.
Time frame: Baseline to 12 months
Change From Baseline in New York Heart Association (NYHA) Functional Class
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.
Time frame: Baseline to 12 months
Change From Baseline in Plasma Gb3
Time frame: Baseline to 12 months
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Turku University Central Hospital
Turku, Finland
Szpital Uniwersytecki w Krakowie
Krakow, Poland
Instytut Kardiologii, I Klinika Choroby Wiencowej
Warsaw, Poland
General Hospital Slovenj Gradec
Slovenj Gradec, Slovenia
Salford Royal NHS Foundation Trust
Salford, England, United Kingdom
Change From Baseline in eGFR
Time frame: Baseline to 12 months
Change From Baseline in Albumin/Creatinine (A/Cr) Ratio
Time frame: Baseline to 12 months