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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT01124643
First received: April 23, 2010
Last updated: January 14, 2014
Last verified: January 2014
  Purpose

This study will evaluate safety and clinical outcomes of treatment with Replagal in adult patients with Fabry disease who have completed Study TKT028.


Condition Intervention Phase
Fabry Disease
Biological: Replagal
Phase 3

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Shire Human Genetic Therapies, Inc.:

Primary Outcome Measures:
  • Evaluate the effect of continued dosing with Replagal following 53 weeks of treatment in TKT028 (NCT00864851) on the change in Left Ventricular Mass (LVM) normalized to height (LVM/h) [ Time Frame: Baseline to week 53 ] [ Designated as safety issue: No ]
  • Safety evaluation measured by change from baseline in vital signs, physical examination, 12-lead electrocardiograms (ECGs), standard clinical laboratory test results, presence of antibodies, concomitant medications, and adverse events. [ Time Frame: Baseline to week 53 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered IV EOW) following 53 weeks of treatment in Study TKT028 (NCT00864851) on exercise tolerance. [ Time Frame: Baseline to week 53 ] [ Designated as safety issue: No ]
    Measured by Vo2max using the STEEP exercise protocol and by distance walked in meters (m)

  • Evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered IV EOW) following 53 weeks of treatment in Study TKT028 (NCT00864851) on the improvement from baseline in disease-specific quality of life (QoL) [ Time Frame: Baseline to week 53 ] [ Designated as safety issue: No ]
  • Evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered IV EOW) following 53 weeks of treatment in Study TKT028 (NCT00864851) based on New York Heart Association (NYHA) classification [ Time Frame: Baseline to week 53 ] [ Designated as safety issue: No ]
  • Evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered IV EOW) following 53 weeks of treatment in Study TKT028 (NCT00864851) on magnitude or reduction from baseline in plasma Gb3. [ Time Frame: Baseline to week 53 ] [ Designated as safety issue: No ]
  • Evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered IV EOW) following 53 weeks of treatment in Study TKT028 (NCT00864851) on the rate of decline in renal function by assessment of GFR (Glomerular Filtration Rate). [ Time Frame: Baseline to week 53 ] [ Designated as safety issue: No ]
  • Evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered IV EOW) following 53 weeks of treatment in Study TKT028 (NCT00864851) on the improvement in severity of proteinuria/albuminuria. [ Time Frame: Baseline to week 53 ] [ Designated as safety issue: No ]

Enrollment: 35
Study Start Date: April 2010
Study Completion Date: October 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

Detailed Description:

The objective of the study is to evaluate the long term safety and effect of continued dosing with Replagal (0.2 mg/kg administered intravenously [IV] every other week[EOW]) following 53 weeks of treatment in Study TKT028 on the reduction from baseline in left ventricular mass (LVM)

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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 patients 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 ClinicalTrials.gov identifier: NCT01124643

Locations
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, Victoria, Australia, 3050
Czech Republic
1st School of Medicine Charles University
Prague, Czech Republic
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 Human Genetic Therapies, Inc.
Investigators
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

No publications provided

Responsible Party: Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier: NCT01124643     History of Changes
Other Study ID Numbers: HGT-REP-060
Study First Received: April 23, 2010
Last Updated: January 14, 2014
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Czech Republic: State Institute for Drug Control
Finland: Finnish Medicines Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Slovenia: Agency for Medicinal Products - Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration

Keywords provided by Shire Human Genetic Therapies, Inc.:
Replagal
Algalsidase alfa
Alpha-Galactosidase

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on July 23, 2014