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
Results First Received: March 25, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Fabry Disease
Intervention: Biological: Replagal

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Replagal® (0.2 mg/kg) Replagal 0.2 mg/kg IV, EOW

Participant Flow:   Overall Study
    Replagal® (0.2 mg/kg)  
STARTED     35  
COMPLETED     34  
NOT COMPLETED     1  
Death                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Replagal® (0.2 mg/kg) Replagal 0.2 mg/kg IV, EOW

Baseline Measures
    Replagal® (0.2 mg/kg)  
Number of Participants  
[units: participants]
  35  
Age  
[units: Years]
Mean ± Standard Deviation
  51.8  ± 9.88  
Age, Customized [1]
[units: Participants]
 
>=65 years     3  
Between 18 and 65 years     32  
Gender  
[units: Participants]
 
Female     16  
Male     19  
Region of Enrollment  
[units: Participants]
 
AUSTRALIA     2  
CZECH REPUBLIC     5  
FINLAND     5  
POLAND     13  
SLOVENIA     4  
UNITED KINGDOM     3  
UNITED STATES     3  
Left Ventricular Mass Indexed to Height (LVMI)  
[units: (g/m^2.7)]
Mean ± Standard Deviation
  81.18  ± 32.13  
Maximal Oxygen Consumption (VO2max)  
[units: (mL/min/kg)]
Mean ± Standard Deviation
  20.2  ± 6.73  
Distance Walked in 6-Minute Walk Test (6MWT)  
[units: meters]
Mean ± Standard Deviation
  515.5  ± 144.13  
Minnesota Living with Heart Failure Questionnaire (MLHF-Q) Summary Score [2]
[units: units on a scale]
Mean ± Standard Deviation
  26.5  ± 26.95  
New York Heart Association (NYHA) Functional Class [3]
[units: participants]
 
Class I (Very Mild)     21  
Class II (Mild)     13  
Class III (Moderate)     1  
Class IV (Severe)     0  
Plasma Gb3 (globotriaosylceramide)  
[units: (nmol/mL)]
Mean ± Standard Deviation
  4.35  ± 2.51  
Estimated Glomerular Filtration Rate (eGFR)  
[units: (mL/min/1.73m^2)]
Mean ± Standard Deviation
  77.60  ± 26.12  
Albumin-to-Creatinine (A/Cr) Ratio  
[units: Ratio]
Mean ± Standard Deviation
  284.1  ± 712.90  
[1] Age at time of consent
[2] 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.
[3] 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



  Outcome Measures
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1.  Primary:   Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)   [ Time Frame: Baseline to 12 months ]

2.  Primary:   Safety Evaluations   [ Time Frame: Baseline to 12 months ]

3.  Secondary:   Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise   [ Time Frame: Baseline to 12 months ]

4.  Secondary:   Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)   [ Time Frame: Baseline to 12 months ]

5.  Secondary:   Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)   [ Time Frame: Baseline to 12 months ]

6.  Secondary:   Change From Baseline in New York Heart Association (NYHA) Functional Class   [ Time Frame: Baseline to 12 months ]

7.  Secondary:   Change From Baseline in Plasma Gb3   [ Time Frame: Baseline to 12 months ]

8.  Secondary:   Change From Baseline in eGFR   [ Time Frame: Baseline to 12 months ]

9.  Secondary:   Change From Baseline in Albumin/Creatinine (A/Cr) Ratio   [ Time Frame: Baseline to 12 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Physician
Organization: Shire Development LLC
phone: +1 866 842 5335


No publications provided


Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01124643     History of Changes
Other Study ID Numbers: HGT-REP-060, 2009-015985-75
Study First Received: April 23, 2010
Results First Received: March 25, 2014
Last Updated: August 6, 2014
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Czech Republic: State Institute for Drug Control
Finland: Finnish Medicines Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Slovenia: Agency for Medicinal Products - Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration