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

Shire

Status and phase: Completed; Phase 3

Conditions: Fabry Disease

Treatments: Biological: Replagal

Study type: Interventional

Funder types: Industry

Identifiers

NCT01124643

HGT-REP-060

2009-015985-75 (EudraCT Number)

Details and patient eligibility

About

This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).

Full 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.

Enrollment: 35 patients

Sex: All

Ages: 18+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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35 participants in 1 patient group

Replagal 0.2 mg/kg EOW

Experimental group

Description:

Intravenous, 0.2mg/kg EOW

Treatment:

Biological: Replagal