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

Primary Purpose

Fabry Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Replagal
Sponsored by
Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease focused on measuring Agalsidase alfa, Alpha-Galactosidase, Replagal, Enzyme Replacement Therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Iowa Hospitals and Clinics
  • O&O Alpan LLC
  • The Royal Melbourne Hospital
  • 1st School of Medicine Charles University
  • Turku University Central Hospital
  • Szpital Uniwersytecki w Krakowie
  • Instytut Kardiologii, I Klinika Choroby Wiencowej
  • General Hospital Slovenj Gradec
  • Salford Royal NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Replagal 0.2 mg/kg EOW

Arm Description

Intravenous, 0.2mg/kg EOW

Outcomes

Primary Outcome Measures

Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
Safety Evaluations

Secondary Outcome Measures

Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
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.
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.
Change From Baseline in Plasma Gb3
Change From Baseline in eGFR
Change From Baseline in Albumin/Creatinine (A/Cr) Ratio

Full Information

First Posted
April 23, 2010
Last Updated
May 19, 2021
Sponsor
Shire
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1. Study Identification

Unique Protocol Identification Number
NCT01124643
Brief Title
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
April 13, 2010 (Actual)
Primary Completion Date
July 8, 2013 (Actual)
Study Completion Date
July 8, 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire

4. Oversight

Data Monitoring Committee
No

5. Study Description

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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
Agalsidase alfa, Alpha-Galactosidase, Replagal, Enzyme Replacement Therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
35 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Replagal 0.2 mg/kg EOW
Arm Type
Experimental
Arm Description
Intravenous, 0.2mg/kg EOW
Intervention Type
Biological
Intervention Name(s)
Replagal
Other Intervention Name(s)
algalsidase alfa, alpha-Galactosidase, DRX005B
Intervention Description
0.2 mg/kg administered intravenously [IV] every other week
Primary Outcome Measure Information:
Title
Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
Time Frame
Baseline to 12 months
Title
Safety Evaluations
Time Frame
Baseline to 12 months
Secondary Outcome Measure Information:
Title
Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
Time Frame
Baseline to 12 months
Title
Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
Time Frame
Baseline to 12 months
Title
Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)
Description
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
Title
Change From Baseline in New York Heart Association (NYHA) Functional Class
Description
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
Title
Change From Baseline in Plasma Gb3
Time Frame
Baseline to 12 months
Title
Change From Baseline in eGFR
Time Frame
Baseline to 12 months
Title
Change From Baseline in Albumin/Creatinine (A/Cr) Ratio
Time Frame
Baseline to 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
O&O Alpan LLC
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
The Royal Melbourne Hospital
City
Parkville
ZIP/Postal Code
3050
Country
Australia
Facility Name
1st School of Medicine Charles University
City
Prague
Country
Czechia
Facility Name
Turku University Central Hospital
City
Turku
ZIP/Postal Code
FI-20520
Country
Finland
Facility Name
Szpital Uniwersytecki w Krakowie
City
Krakow
ZIP/Postal Code
31-066
Country
Poland
Facility Name
Instytut Kardiologii, I Klinika Choroby Wiencowej
City
Warsaw
Country
Poland
Facility Name
General Hospital Slovenj Gradec
City
Slovenj Gradec
ZIP/Postal Code
2380
Country
Slovenia
Facility Name
Salford Royal NHS Foundation Trust
City
Salford
State/Province
England
ZIP/Postal Code
M6 8HD
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
26185417
Citation
Golan L, Goker-Alpan O, Holida M, Kantola I, Klopotowski M, Kuusisto J, Linhart A, Musial J, Nicholls K, Gonzalez-Rodriguez D, Sharma R, Vujkovac B, Chang P, Wijatyk A. Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease. Drug Des Devel Ther. 2015 Jul 8;9:3435-44. doi: 10.2147/DDDT.S80928. eCollection 2015.
Results Reference
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Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

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