Follow on study in adault Fabry Disease patients

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2009-015985-75-PL

Enrollment

Registered

2009-11-30

Start date

2010-02-03

Completion date

Unknown

Last updated

2013-08-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fabry disease MedDRA version: 14.0 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Replagal Product Name: Replagal Pharmaceutical Form: Solution for infusion INN or Proposed INN: AGALSIDASE ALFA CAS Number: 104138-64-9 Concentration unit: mg/ml milligram(s)/millilitre Co

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Complete all study requirements and assessments for Study TKT028 less than 30 days (± 7 days) prior to the first dose in this extension protocol. 2. 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. 3. Has received and tolerated at least 80% of the total planned Replagal infusions in Study TKT028. 4. 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 their participation in the study. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 39 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4

Exclusion criteria

Exclusion criteria: 1. Has received treatment with any investigational drug (other than Replagal) or device within 30 days prior to study entry. 2. 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. 3. Has a positive test for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibody, or human immunodeficiency virus (HIV) antibody. 4. Is pregnant or lactating. 5. Is morbidly obese, defined as body mass index (BMI) >39 kg/m2. 6. 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.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objectives of this study are: • To evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered intravenously [IV] every other week) following 53 weeks of treatment in Study TKT028 (at 0.2 mg/kg IV every other week or 0.2 mg/kg or 0.4 mg/kg IV every week) on the reduction from baseline in left ventricular mass (LVM) as measured by echocardiography • To collect long-term safety and clinical outcome data in adult patients with Fabry disease who are receiving enzyme replacement therapy (ERT) with Replagal ;Secondary Objective: To evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered IV every other week) following 53 weeks of treatment in Study TKT028 on: • Exercise tolerance, as measured by maximal oxygen consumption (Vo2max) using the standard exponential exercise protocol (STEEP) and by distance walked in meters (m) using the 6-Minute Walk Test (6MWT) • The improvement from baseline in disease-specific quality of life (QoL) using the summary score of the Minnesota Living with Heart Failure questionnaire (MLHF-Q) • Improvement from baseline in New York Heart Association (NYHA) functional class for heart failure symptoms ;Primary end point(s): Change from baseline to Week 53 in this study in LVM normalized to height (LVM/h) as measured by echocardiography.;Timepoint(s) of evaluation of this end point: At 53 weeks	—

Measure

Time frame

Main Objective: The primary objectives of this study are: • To evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered intravenously [IV] every other week) following 53 weeks of treatment in Study TKT028 (at 0.2 mg/kg IV every other week or 0.2 mg/kg or 0.4 mg/kg IV every week) on the reduction from baseline in left ventricular mass (LVM) as measured by echocardiography • To collect long-term safety and clinical outcome data in adult patients with Fabry disease who are receiving enzyme replacement therapy (ERT) with Replagal ;Secondary Objective: To evaluate the effect of continued dosing with Replagal (0.2 mg/kg administered IV every other week) following 53 weeks of treatment in Study TKT028 on: • Exercise tolerance, as measured by maximal oxygen consumption (Vo2max) using the standard exponential exercise protocol (STEEP) and by distance walked in meters (m) using the 6-Minute Walk Test (6MWT) • The improvement from baseline in disease-specific quality of life (QoL) using the summary score of the Minnesota Living with Heart Failure questionnaire (MLHF-Q) • Improvement from baseline in New York Heart Association (NYHA) functional class for heart failure symptoms ;Primary end point(s): Change from baseline to Week 53 in this study in LVM normalized to height (LVM/h) as measured by echocardiography.;Timepoint(s) of evaluation of this end point: At 53 weeks

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Secondary

Measure	Time frame
Secondary end point(s): The secondary endpoints of this study are: • Change from baseline to Week 53 in this study in exercise tolerance, as measured by Vo2max at peak exercise using the STEEP exercise protocol and by distance walked in meters (m) using the 6MWT • Change from baseline to Week 53 in this study in NYHA Class • Change from baseline to Week 53 in this study in QoL using the MLHF-Q summary score;Timepoint(s) of evaluation of this end point: At 53 weeks	—

Measure

Time frame

Secondary end point(s): The secondary endpoints of this study are: • Change from baseline to Week 53 in this study in exercise tolerance, as measured by Vo2max at peak exercise using the STEEP exercise protocol and by distance walked in meters (m) using the 6MWT • Change from baseline to Week 53 in this study in NYHA Class • Change from baseline to Week 53 in this study in QoL using the MLHF-Q summary score;Timepoint(s) of evaluation of this end point: At 53 weeks

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Countries

Australia, Czech Republic, Finland, Paraguay, Poland, Slovenia, United Kingdom, United States

Contacts

Public ContactMedical Monitor

Shire Human Genetic Therapies

ecrombez@shire.com+1781482-9287

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026