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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01124643
Recruitment Status : Completed
First Posted : May 17, 2010
Results First Posted : August 22, 2014
Last Update Posted : June 8, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( Shire )

Study Description
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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).

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: Replagal Phase 3

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 35 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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
Actual Study Start Date : April 13, 2010
Actual Primary Completion Date : July 8, 2013
Actual Study Completion Date : July 8, 2013

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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 Names:
  • algalsidase alfa
  • alpha-Galactosidase
  • DRX005B


Outcome Measures
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Primary Outcome Measures :
  1. Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI) [ Time Frame: Baseline to 12 months ]
  2. Safety Evaluations [ Time Frame: Baseline to 12 months ]

Secondary Outcome Measures :
  1. Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise [ Time Frame: Baseline to 12 months ]
  2. Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT) [ Time Frame: Baseline to 12 months ]
  3. 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.

  4. 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.

  5. Change From Baseline in Plasma Gb3 [ Time Frame: Baseline to 12 months ]
  6. Change From Baseline in eGFR [ Time Frame: Baseline to 12 months ]
  7. Change From Baseline in Albumin/Creatinine (A/Cr) Ratio [ Time Frame: Baseline to 12 months ]

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

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01124643


Locations
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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
Australia
The Royal Melbourne Hospital
Parkville, Australia, 3050
Czechia
1st School of Medicine Charles University
Prague, Czechia
Finland
Turku University Central Hospital
Turku, Finland, FI-20520
Poland
Szpital Uniwersytecki w Krakowie
Krakow, Poland, 31-066
Instytut Kardiologii, I Klinika Choroby Wiencowej
Warsaw, Poland
Slovenia
General Hospital Slovenj Gradec
Slovenj Gradec, Slovenia, 2380
United Kingdom
Salford Royal NHS Foundation Trust
Salford, England, United Kingdom, M6 8HD
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Takeda
More Information
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Publications of Results:

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01124643    
Other Study ID Numbers: HGT-REP-060
2009-015985-75 ( EudraCT Number )
First Posted: May 17, 2010    Key Record Dates
Results First Posted: August 22, 2014
Last Update Posted: June 8, 2021
Last Verified: May 2021
Keywords provided by Takeda ( Shire ):
Agalsidase alfa
Alpha-Galactosidase
Replagal
Enzyme Replacement Therapy
Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
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
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders