Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official 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|
- Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI) [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
- Safety Evaluations [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: Yes ]
- Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
- Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT) [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
- Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q) [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]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.
- Change From Baseline in New York Heart Association (NYHA) Functional Class [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
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.
- Change From Baseline in Plasma Gb3 [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
- Change From Baseline in eGFR [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
- Change From Baseline in Albumin/Creatinine (A/Cr) Ratio [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
|Study Start Date:||April 2010|
|Study Completion Date:||July 2013|
|Primary Completion Date:||July 2013 (Final data collection date for primary outcome measure)|
Experimental: Replagal 0.2 mg/kg EOW
Intravenous, 0.2mg/kg EOW
0.2 mg/kg administered intravenously [IV] every other week
Other Name: Replagal, algalsidase alfa, alpha-Galactosidase, DRX005B
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01124643
|United States, Iowa|
|University of Iowa Hospitals and Clinics|
|Iowa City, Iowa, United States, 52242|
|United States, Virginia|
|O&O Alpan LLC|
|Fairfax, Virginia, United States, 22030|
|The Royal Melbourne Hospital|
|Parkville, Victoria, Australia, 3050|
|1st School of Medicine Charles University|
|Prague, Czech Republic|
|Turku University Central Hospital|
|Turku, Finland, FI-20520|
|Szpital Uniwersytecki w Krakowie|
|Krakow, Poland, 31-066|
|Instytut Kardiologii, I Klinika Choroby Wiencowej|
|General Hospital Slovenj Gradec|
|Slovenj Gradec, Slovenia, 2380|
|Salford Royal NHS Foundation Trust|
|Salford, England, United Kingdom, M6 8HD|
|Principal Investigator:||Lubor Golan||1st School of Medicine, Charles University, Prague|
|Principal Investigator:||Lidia Chojinowska||Instytut Kardiologii|
|Principal Investigator:||Kathleen Nicholls, MB.BS, M.D.||Melbourne Health|
|Principal Investigator:||Jacek Musial, MD, PhD||Szpital Uniwersytecki w Krakowie|
|Principal Investigator:||Ilkka Kantola, MD, PhD||University of Turku|
|Principal Investigator:||Reena Sharma, MD||Salford Royal NHS Foundation Trust|
|Principal Investigator:||Ozlem Goker-Alpan, MD||O & O Alpan LLC|
|Principal Investigator:||Bojan Vujkovac, MD||General Hospital Slovenj Gradec|
|Principal Investigator:||Myrl Holida, PA-C||University of Iowa|