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Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00864851
First received: March 18, 2009
Last updated: March 3, 2015
Last verified: May 2014
History of Changes
Results First Received: January 30, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel 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
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Reporting Groups
  Description
Replagal 0.2 mg/kg, IV, Every Other Week Patients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every other week for 52 weeks.
Replagal 0.2 mg/kg, IV, Weekly Patients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every week for 52 weeks.
Replagal 0.4 mg/kg, IV, Weekly Patients randomized to receive Replagal 0.4 mg/kg via intravenous infusion every week for 52 weeks.

Participant Flow:   Overall Study
    Replagal 0.2 mg/kg, IV, Every Other Week   Replagal 0.2 mg/kg, IV, Weekly   Replagal 0.4 mg/kg, IV, Weekly
STARTED   20   19   5 
COMPLETED   17   18   5 
NOT COMPLETED   3   1   0 
Withdrawal by Subject                0                1                0 
Physician Decision                1                0                0 
Death                1                0                0 
Patient uncooperative                1                0                0 



  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, IV, Every Other Week Patients who received Replagal 0.2 mg/kg via intravenous infusion every other week for 52 weeks.
Replagal 0.2 mg/kg, IV, Weekly Patients who received Replagal 0.2 mg/kg via intravenous infusion every week for 52 weeks.
Replagal 0.4 mg/kg, IV, Weekly Patients who received Replagal 0.4 mg/kg via intravenous infusion every week for 52 weeks.
Total Total of all reporting groups

Baseline Measures
   Replagal 0.2 mg/kg, IV, Every Other Week   Replagal 0.2 mg/kg, IV, Weekly   Replagal 0.4 mg/kg, IV, Weekly   Total 
Overall Participants Analyzed 
[Units: Participants]
 		 20   19   5   44 
Age 
[Units: Years]
Mean (Standard Deviation) 		 50.3  (7.23)   51.8  (11.42)   49.4  (9.75)   50.8  (9.34) 
Age, Customized 
[Units: Participants]
 		       
18 to <45 years   5   6   1   12 
>=45 years   15   13   4   32 
Gender 
[Units: Participants]
 		       
Female   6   9   3   18 
Male   14   10   2   26 
Ethnicity (NIH/OMB) 
[Units: Participants]
 		       
Hispanic or Latino   1   1   0   2 
Not Hispanic or Latino   19   18   5   42 
Unknown or Not Reported   0   0   0   0 
Race (NIH/OMB) 
[Units: Participants]
 		       
American Indian or Alaska Native   0   0   0   0 
Asian   0   0   0   0 
Native Hawaiian or Other Pacific Islander   0   0   0   0 
Black or African American   0   0   0   0 
White   20   19   5   44 
More than one race   0   0   0   0 
Unknown or Not Reported   0   0   0   0 
Baseline Left Ventricular Mass Indexed to Height (LVMI) 
[Units: G/m^2.7]
Mean (Standard Deviation) 		 76.1  (22.8)   82.6  (28.3)   99.3  (46.2)   81.5  (28.5) 
Baseline Maximal Oxygen Consumption (VO2 max) at Peak Exercise [1] 
[Units: mL/min/kg]
Mean (Standard Deviation) 		 20.2  (6.20)   22.6  (6.89)   24.0  (11.69)   21.6  (7.05) 
[1] 0.2 mg/kg Every Other Week: n=19 0.2 mg/kg Weekly: n=16 0.4 mg/kg Weekly: n=4 Total: n=39
Baseline Distance Walked in 6-Minute Walk Test (6MWT) [1] 
[Units: m]
Mean (Standard Deviation) 		 459.6  (103.0)   514.3  (87.3)   530.7  (90.2)   492.8  (97.0) 
[1] 0.2 mg/kg Every Other Week: n=18 0.2 mg/kg Weekly: n=19 0.4 mg/kg Weekly: n=5 Total: n=42
Baseline Minnesota Living with Heart Failure Questionnaire (MLHF-Q) Summary Score [1] 
[Units: Scores on a scale]
Mean (Standard Deviation) 		 37.0  (23.8)   21.1  (21.0)   19.6  (26.5)   28.1  (23.8) 
[1] Quality of life (QoL) was evaluated using the MLHF-Q, version 2. The questionnaire is designed to assess the degree to which heart failure symptoms affect a patient's daily life. The summary score ranges from 0 to 105, with a score of 105 representing the highest adverse impact on a patient's QoL.
Baseline New York Heart Association (NYHA) Functional Class [1] 
[Units: Participants]
 		       
Class I   6   10   2   18 
Class II   11   9   2   22 
Class III   3   0   1   4 
Class IV   0   0   0   0 
[1]

Class I: Cardiac disease without limitation of physical activity. Ordinary physical activity does not cause undue symptoms.

Class II: Cardiac disease with slight limitation of physical activity. Comfortable at rest, but ordinary physical activity causes symptoms.

Class III: Cardiac disease with marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes symptoms.

Class IV: Cardiac disease and unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest.

Note: Symptoms include fatigue, palpitation or dyspnea
Baseline Plasma Globotriaosylceramide (GB3) 
[Units: Nmol/ml]
Mean (Standard Deviation) 		 6.067  (3.388)   5.652  (5.212)   5.292  (1.865)   5.800  (4.104) 
Baseline Estimated Glomerular Filtration Rate (eGFR) 
[Units: mL/min/1.73 m^2]
Mean (Standard Deviation) 		 82.0  (34.1)   78.1  (33.8)   72.1  (31.2)   79.2  (33.1) 
Baseline Urinary Albumin/Creatinine (A/Cr) Ratio 
[Units: Mg/g]
Mean (Standard Deviation) 		 313.4  (614.07)   293.4  (521.23)   298.2  (175.85)   303.0  (532.27) 


  Outcome Measures
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1.  Primary:   Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI)   [ Time Frame: Baseline, Month 12 (Week 53) ]
  Show Outcome Measure 1

2.  Secondary:   Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise   [ Time Frame: Baseline, Month 12 (Week 53) ]
  Show Outcome Measure 2

3.  Secondary:   Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT)   [ Time Frame: Baseline, Month 12 (Week 53) ]
  Show Outcome Measure 3

4.  Secondary:   Change From Baseline to Month 12 in the Minnesota Living With Heart Failure Questionnaire (MLHF-Q) Summary Score   [ Time Frame: Baseline, Month 12 (Week 53) ]
  Show Outcome Measure 4

5.  Secondary:   Change From Baseline to Month 12 in New York Heart Association (NYHA) Functional Class   [ Time Frame: Baseline, Month 12 (Week 53) ]
  Show Outcome Measure 5

6.  Secondary:   Change From Baseline to Month 12 in Plasma Globotriaosylceramide (GB3)   [ Time Frame: Baseline, Month 12 (Week 53) ]
  Show Outcome Measure 6

7.  Secondary:   Change From Baseline to Month 12 in Estimated Glomerular Filtration Rate (eGFR)   [ Time Frame: Baseline, Month 12 (Week 53) ]
  Show Outcome Measure 7

8.  Secondary:   Change From Baseline to Month 12 in Urinary Albumin/Creatinine (A/Cr) Ratio   [ Time Frame: Baseline, Month 12 (Week 53) ]
  Show Outcome Measure 8

9.  Secondary:   Safety Evaluation   [ Time Frame: 56 Weeks ]
  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: Anna Wijatyk, MD, Medical Director
Organization: Shire Human Genetic Therapies, Inc. (Shire HGT)
phone: 781-482-9622
e-mail: awijatyk@shire.com



Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00864851     History of Changes
Other Study ID Numbers: TKT028
2007-005543-22 ( EudraCT Number )
Study First Received: March 18, 2009
Results First Received: January 30, 2014
Last Updated: March 3, 2015