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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01124643
First received: April 23, 2010
Last updated: August 6, 2014
Last verified: July 2014

April 23, 2010
August 6, 2014
April 2010
July 2013   (final data collection date for primary outcome measure)
  • Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI) [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
  • Safety Evaluations [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: Yes ]
To evaluate the safety and effect of continued dosing with Replagal [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
To evaluate the long term safety and effect of continued dosing with Replagal (0.2 mg/kg administered intravenously [IV] every other week)following 53 weeks of treatment in Study TKT028 on the reduction from baseline in left ventricular mass (LVM) as measured by echocardiography
Complete list of historical versions of study NCT01124643 on ClinicalTrials.gov Archive Site
  • Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
  • Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT) [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
  • Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q) [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
    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.
  • Change From Baseline in New York Heart Association (NYHA) Functional Class [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
  • Change From Baseline in Plasma Gb3 [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
  • Change From Baseline in eGFR [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
  • Change From Baseline in Albumin/Creatinine (A/Cr) Ratio [ Time Frame: Baseline to 12 months ] [ Designated as safety issue: No ]
Effect on exercise tolerance as measured by maximal oxygen consumption and distance walked in meters [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Secondary Outcome Measures: Excercise tolerance, as measured by maximal oxygen consumption (VO2 max) using the standard exponential exercise protocol (STEEP) and by distance walked in meters (m) using the 6-Minute Walk Test (6MWT)
Not Provided
Not Provided
 
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease

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

HGT-REP-060 was designed to provide patients who participated in TKT028 an additional 52 weeks of treatment with Replagal at the standard dose of 0.2 mg/kg EOW to assess the effect of continued Replagal therapy on long-term safety and clinical outcomes for this patient population.

Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Fabry Disease
Biological: Replagal
0.2 mg/kg administered intravenously [IV] every other week
Other Name: Replagal, algalsidase alfa, alpha-Galactosidase
Experimental: Replagal 0.2 mg/kg EOW
Intravenous, 0.2mg/kg EOW
Intervention: Biological: Replagal
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
35
October 2013
July 2013   (final data collection date for primary outcome measure)

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 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 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
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Czech Republic,   Slovenia,   Poland,   United States,   United Kingdom,   Australia,   Finland
 
NCT01124643
HGT-REP-060, 2009-015985-75
No
Shire
Shire
Not Provided
Principal Investigator: Lubor Golan 1st School of Medicine, Charles University, Prague
Principal Investigator: Lidia Chojinowska Instytut Kardiologii
Principal Investigator: Kathleen Nicholls, MB.BS, M.D. Melbourne Health
Principal Investigator: Jacek Musial, MD, PhD Szpital Uniwersytecki w Krakowie
Principal Investigator: Ilkka Kantola, MD, PhD University of Turku
Principal Investigator: Reena Sharma, MD Salford Royal NHS Foundation Trust
Principal Investigator: Ozlem Goker-Alpan, MD O & O Alpan LLC
Principal Investigator: Bojan Vujkovac, MD General Hospital Slovenj Gradec
Principal Investigator: Myrl Holida, PA-C University of Iowa
Shire
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP