Clinical trial NCT01124643: Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Fabry Disease
Keywords: Agalsidase alfa
Alpha-Galactosidase
Replagal
Enzyme Replacement Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2010-05-17
Enrollment: 35
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
timeFrame: Baseline to 12 months
measure: Safety Evaluations
timeFrame: Baseline to 12 months
Secondary outcomes:
measure: Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
timeFrame: Baseline to 12 months
measure: Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
timeFrame: Baseline to 12 months
measure: Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)
timeFrame: Baseline to 12 months
measure: Change From Baseline in New York Heart Association (NYHA) Functional Class
timeFrame: Baseline to 12 months
measure: Change From Baseline in Plasma Gb3
timeFrame: Baseline to 12 months
measure: Change From Baseline in eGFR
timeFrame: Baseline to 12 months
measure: Change From Baseline in Albumin/Creatinine (A/Cr) Ratio
timeFrame: Baseline to 12 months
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Fairfax
country: United States
facility: O&O Alpan LLC
state: Virginia
city: Parkville
country: Australia
facility: The Royal Melbourne Hospital
city: Prague
country: Czechia
facility: 1st School of Medicine Charles University
city: Turku
country: Finland
facility: Turku University Central Hospital
city: Krakow
country: Poland
facility: Szpital Uniwersytecki w Krakowie
city: Warsaw
country: Poland
facility: Instytut Kardiologii, I Klinika Choroby Wiencowej
city: Slovenj Gradec
country: Slovenia
facility: General Hospital Slovenj Gradec
city: Salford
country: United Kingdom
facility: Salford Royal NHS Foundation Trust
state: England
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Golan 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 enzyme replacement therapy in adults with Fabry disease. Drug Des Devel Ther. 2015 Jul 8;9:3435-44. doi: 10.2147/DDDT.S80928. eCollection 2015.