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Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

This study has been completed.
Information provided by (Responsible Party):
Shire Identifier:
First received: April 23, 2010
Last updated: April 2, 2015
Last verified: March 2015
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).

Condition Intervention Phase
Fabry Disease
Biological: Replagal
Phase 3

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

Further study details as provided by Shire:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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) [ Time Frame: Baseline to 12 months ]
    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 ]

    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 ]
  • 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 ]

Enrollment: 35
Study Start Date: April 2010
Study Completion Date: July 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Replagal 0.2 mg/kg EOW
Intravenous, 0.2mg/kg EOW
Biological: Replagal
0.2 mg/kg administered intravenously [IV] every other week
Other Name: Replagal, algalsidase alfa, alpha-Galactosidase, DRX005B

Detailed Description:
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.

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its 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
Czech Republic
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
Warsaw, Poland
General Hospital Slovenj Gradec
Slovenj Gradec, Slovenia, 2380
United Kingdom
Salford Royal NHS Foundation Trust
Salford, England, United Kingdom, M6 8HD
Sponsors and Collaborators
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
  More Information

Responsible Party: Shire Identifier: NCT01124643     History of Changes
Other Study ID Numbers: HGT-REP-060
2009-015985-75 ( EudraCT Number )
Study First Received: April 23, 2010
Results First Received: March 25, 2014
Last Updated: April 2, 2015

Keywords provided by Shire:
Agalsidase alfa
Enzyme Replacement Therapy

Additional relevant MeSH terms:
Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders processed this record on April 28, 2017