Follow on study in adault Fabry Disease patients

• Register: EUCTR
• Last refreshed on: 19 August 2013
• Main ID: EUCTR2009-015985-75-PL
• Date of registration: 30/11/2009
• Prospective Registration: Yes
• Primary sponsor: Shire Human Genetic Therapies (HGT), Inc.
• Public title: Follow on study in adault Fabry Disease patients
• Scientific 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
• Date of first enrolment: 03/02/2010
• Target sample size: 43
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-015985-75
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

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

Contacts

• Name: Medical Monitor
• Affiliation: Shire Human Genetic Therapies

• Name: Medical Monitor
• Affiliation: Shire Human Genetic Therapies

Key inclusion & exclusion 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:
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.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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

Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Intervention(s)

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
Concentration type: equal
Concentration number: 1-

Primary Outcome(s)

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

Timepoint(s) of evaluation of this end point: At 53 weeks

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

Primary end point(s): Change from baseline to Week 53 in this study in LVM normalized to height (LVM/h) as measured by echocardiography.

Secondary Outcome(s)

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

Secondary ID(s)

HGT-REP-060

Source(s) of Monetary Support

Shire Human Genetic Therapies