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

Fabry Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01124643
• Other study ID #: HGT-REP-060
• Secondary ID: 2009-015985-75
• Status: Completed
• Phase: Phase 3
• Start date: April 13, 2010
• Est. completion date: July 8, 2013

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 35
• Est. completion date: July 8, 2013
• Est. primary completion date: July 8, 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Biological:
• Replagal
0.2 mg/kg administered intravenously [IV] every other week

Locations

Country	Name	City	State
Australia	The Royal Melbourne Hospital	Parkville
Czechia	1st School of Medicine Charles University	Prague
Finland	Turku University Central Hospital	Turku
Poland	Szpital Uniwersytecki w Krakowie	Krakow
Poland	Instytut Kardiologii, I Klinika Choroby Wiencowej	Warsaw
Slovenia	General Hospital Slovenj Gradec	Slovenj Gradec
United Kingdom	Salford Royal NHS Foundation Trust	Salford	England
United States	O&O Alpan LLC	Fairfax	Virginia
United States	University of Iowa Hospitals and Clinics	Iowa City	Iowa

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

United States,  Australia,  Czechia,  Finland,  Poland,  Slovenia,  United Kingdom, 

References & Publications (1)

Golán 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 enzy — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)		Baseline to 12 months
Primary	Safety Evaluations		Baseline to 12 months
Secondary	Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise		Baseline to 12 months
Secondary	Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)		Baseline to 12 months
Secondary	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.	Baseline to 12 months
Secondary	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.	Baseline to 12 months
Secondary	Change From Baseline in Plasma Gb3		Baseline to 12 months
Secondary	Change From Baseline in eGFR		Baseline to 12 months
Secondary	Change From Baseline in Albumin/Creatinine (A/Cr) Ratio		Baseline to 12 months