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

May 19, 2021 updated by: Shire

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to 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 Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

35

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Parkville, Australia, 3050
        • The Royal Melbourne Hospital
  • Czechia
      • Prague, Czechia
        • 1st School of Medicine Charles University
  • Finland
      • Turku, Finland, FI-20520
        • Turku University Central Hospital
  • Poland
      • Krakow, Poland, 31-066
        • Szpital Uniwersytecki w Krakowie
      • Warsaw, Poland
        • Instytut Kardiologii, I Klinika Choroby Wiencowej
  • Slovenia
      • Slovenj Gradec, Slovenia, 2380
        • General Hospital Slovenj Gradec
  • United Kingdom
    • England
      • Salford, England, United Kingdom, M6 8HD
        • Salford Royal NHS Foundation Trust
  • United States
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • O&O Alpan LLC

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
Time Frame: Baseline to 12 months
Baseline to 12 months
Safety Evaluations
Time Frame: Baseline to 12 months
Baseline to 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
Time Frame: Baseline to 12 months
Baseline to 12 months
Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
Time Frame: Baseline to 12 months
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.
Baseline to 12 months
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.
Baseline to 12 months
Change From Baseline in Plasma Gb3
Time Frame: Baseline to 12 months
Baseline to 12 months
Change From Baseline in eGFR
Time Frame: Baseline to 12 months
Baseline to 12 months
Change From Baseline in Albumin/Creatinine (A/Cr) Ratio
Time Frame: Baseline to 12 months
Baseline to 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

April 13, 2010

Primary Completion (ACTUAL)

July 8, 2013

Study Completion (ACTUAL)

July 8, 2013

Study Registration Dates

First Submitted

April 23, 2010

First Submitted That Met QC Criteria

May 13, 2010

First Posted (ESTIMATE)

May 17, 2010

Study Record Updates

Last Update Posted (ACTUAL)

June 8, 2021

Last Update Submitted That Met QC Criteria

May 19, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HGT-REP-060
  • 2009-015985-75 (EUDRACT_NUMBER)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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