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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: April 2, 2015
Last verified: March 2015
History of Changes
Results First Received: March 25, 2014  
Study Type: Interventional
Study Design: 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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment

Screening details:

Thirty-five of the 40 subjects who completed the parent study (Study TKT028 [NCT00864851]) were enrolled in this extension study.

Reporting Groups
  Description
Replagal® (0.2 mg/kg) Replagal 0.2 milligram per kilogram (mg/kg) intravenously (IV), every other week (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.
No text entered.

Reporting Groups
  Description
Replagal® (0.2 mg/kg) Replagal 0.2 mg/kg IV, EOW

Baseline Measures
   Replagal® (0.2 mg/kg) 
Overall Participants Analyzed 
[Units: Participants]
 		 35 
Age 
[Units: Years]
Mean (Standard Deviation) 		 52.3  (9.86) 
Age, Customized [1] 
[Units: Participants]
 		 
>=65 years   3 
Between 18 and 65 years   32 
[1] Age at time of consent
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) [1] 
[Units: Gram per meter to the power 2.7(g/m^2.7)]
Mean (Standard Deviation) 		 81.18  (32.13) 
[1] Number of participants analyzed for this baseline characteristic was 33
Maximal Oxygen Consumption (VO2max) [1] 
[Units: Milliliter/minute/kilogram (mL/min/kg)]
Mean (Standard Deviation) 		 20.2  (6.73) 
[1] Number of participants analyzed for this baseline characteristic was 32
Distance Walked in 6-Minute Walk Test (6MWT) [1] 
[Units: Meters]
Mean (Standard Deviation) 		 515.5  (144.13) 
[1] Number of participants analyzed for this baseline characteristic was 34
Minnesota Living with Heart Failure Questionnaire (MLHF-Q) Summary Score [1] 
[Units: Units on a scale]
Mean (Standard Deviation) 		 26.5  (26.95) 
[1] 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.
New York Heart Association (NYHA) Functional Class [1] 
[Units: Participants]
 		 
Class I (Very Mild)   21 
Class II (Mild)   13 
Class III (Moderate)   1 
Class IV (Severe)   0 
[1] 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
Plasma Gb3 (globotriaosylceramide) 
[Units: Nanomoles per milliliter (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 [1] 
[Units: Ratio]
Mean (Standard Deviation) 		 284.1  (712.90) 
[1] Number of participants analyzed for this baseline characteristic was 33


  Outcome Measures
  Show All Outcome Measures

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

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

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

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

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

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

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

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

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


  Serious Adverse Events
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  Other Adverse Events
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  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



Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01124643     History of Changes
Other Study ID Numbers: HGT-REP-060
2009-015985-75 ( EudraCT Number )
Study First Received: April 23, 2010
Results First Received: March 25, 2014
Last Updated: April 2, 2015