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A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00140621
Recruitment Status : Completed
First Posted : September 1, 2005
Results First Posted : April 15, 2015
Last Update Posted : May 12, 2015
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fabry Disease
Intervention Drug: Agalsidase beta
Enrollment 6
Recruitment Details The study was conducted at 7 centers between July 06, 2005 and August 6, 2012. One participant was treated at 2 study sites because the participant had to be transferred to another site during the study.
Pre-assignment Details  
Arm/Group Title Agalsidase Beta (Fabrazyme [Recombinant Form])
Hide Arm/Group Description Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.
Period Title: Overall Study
Started 6
Completed 6
Not Completed 0
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description Agalsidase beta 1 mg/kg intravenously once every 2 weeks up to 156 weeks.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
Efficacy evaluable population (EEP) included all registered participants except those who had serious deviations from protocol compliance requirements, did not receive study drug, had no data available at baseline or after study drug administration, and who were found to have no condition in cardiac Fabry disease.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants
53.8  (5.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
5
  83.3%
Male
1
  16.7%
Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms (kg)
Number Analyzed 6 participants
58.50  (9.24)
Height  
Mean (Standard Deviation)
Unit of measure:  Centimeters (cm)
Number Analyzed 6 participants
156.63  (7.20)
Left Ventricular Hypertrophy  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants
No 0
Yes 6
Globotriaosylceramide (GL-3) Accumulation in the Myocardium  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants
No 0
Yes 1
Not Measured 5
1.Primary Outcome
Title Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Hide Description Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Time Frame Baseline to Week 156
Hide Outcome Measure Data
Hide Analysis Population Description
EEP.
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description:
Agalsidase beta 1 mg/kg once every 2 weeks as an intravenous infusion up to 156 weeks.
Overall Number of Participants Analyzed 6
Least Squares Mean (95% Confidence Interval)
Unit of Measure: percent change
Interventricular septum wall thickness
-3.31
(-19.67 to 16.39)
Left ventricular posterior wall thickness
-6.34
(-21.47 to 11.71)
2.Primary Outcome
Title Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Hide Description Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Time Frame Baseline to Week 156
Hide Outcome Measure Data
Hide Analysis Population Description
EEP.
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description:
Agalsidase beta 1 mg/kg once every 2 weeks as an intravenous infusion up to 156 weeks.
Overall Number of Participants Analyzed 6
Least Squares Mean (95% Confidence Interval)
Unit of Measure: mm
Interventricular septum wall thickness
0.37
(-3.88 to 4.61)
Left ventricular posterior wall thickness
-0.33
(-3.31 to 2.64)
3.Primary Outcome
Title Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156
Hide Description Left ventricular mass was assessed by echocardiogram.
Time Frame Baseline to Week 156
Hide Outcome Measure Data
Hide Analysis Population Description
EEP.
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description:
Agalsidase beta 1 mg/kg once every 2 weeks as an intravenous infusion up to 156 weeks.
Overall Number of Participants Analyzed 6
Least Squares Mean (95% Confidence Interval)
Unit of Measure: percent change
-4.14
(-24.35 to 21.47)
4.Primary Outcome
Title Change From Baseline in LVM at Week 156
Hide Description Left ventricular mass was assessed by echocardiogram.
Time Frame Baseline to Week 156
Hide Outcome Measure Data
Hide Analysis Population Description
EEP.
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description:
Agalsidase beta 1 mg/kg once every 2 weeks as an intravenous infusion up to 156 weeks.
Overall Number of Participants Analyzed 6
Least Squares Mean (95% Confidence Interval)
Unit of Measure: gm
22.70
(-106.57 to 151.97)
5.Secondary Outcome
Title Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test
Hide Description Overall cardiac function assessment was assessed by tests (echocardiogram,cardiac catheterization (optional),electrocardiogram,B-type natriuretic peptide [BNP]), clinical symptoms (subjective symptoms) and the New York Heart Association (NYHA) cardiac functional classification.Overall assessment of cardiac function was assessed based on the evaluation items including interventricular septum thickness, left ventricular posterior wall thickness, left ventricular mass, clinical function tests and clinical symptoms. A subject was considered to be Improved: if Improved in 2 items or more, Unchanged: Improved in one item and unchanged in 2 items or unchanged in all 3 items, Aggravated: Aggravated in one item or more.
Time Frame Baseline to Week 156
Hide Outcome Measure Data
Hide Analysis Population Description
EEP.
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description:
Agalsidase beta 1 mg/kg once every 2 weeks as an intravenous infusion up to 156 weeks.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
Improved 5
Unchanged 0
Aggravated 1
6.Secondary Outcome
Title Percent Change From Baseline in GL-3 Plasma Levels at Week 156
Hide Description [Not Specified]
Time Frame Baseline to Week 156
Hide Outcome Measure Data
Hide Analysis Population Description
EEP.
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description:
Agalsidase beta 1 mg/kg once every 2 weeks as an intravenous infusion up to 156 weeks.
Overall Number of Participants Analyzed 6
Least Squares Mean (95% Confidence Interval)
Unit of Measure: percent change
-18.91
(-42.35 to 14.07)
7.Secondary Outcome
Title Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156
Hide Description The 36-Item Short-Form Health Survey (SF-36) is a standardized survey evaluating 8 aspects of functional health and well-being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. These 8 aspects can also be summarized as physical component score (PCS) and mental component score (MCS). The score for a section is an average of the individual question scores, which are scaled 0-100 (100=highest level of functioning).
Time Frame Baseline to Week 156
Hide Outcome Measure Data
Hide Analysis Population Description
EEP.
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description:
Agalsidase beta 1 mg/kg once every 2 weeks as an intravenous infusion up to 156 weeks.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
Physical Health: PCS 6.868  (15.238)
Mental Health: MCS 4.380  (7.425)
Time Frame All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (Week 156) regardless of seriousness or relationship to investigational product.
Adverse Event Reporting Description Reported adverse events are treatment-emergent adverse events that is AEs that developed/worsened during the ‘on treatment period’ (from the first study infusion up to Week 156).
 
Arm/Group Title Agalsidase Beta
Hide Arm/Group Description Agalsidase beta 1 mg/kg once every 2 weeks as an intravenous infusion up to 156 weeks.
All-Cause Mortality
Agalsidase Beta
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Agalsidase Beta
Affected / at Risk (%)
Total   1/6 (16.67%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Large intestine carcinoma  1  1/6 (16.67%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDra 15.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Agalsidase Beta
Affected / at Risk (%)
Total   6/6 (100.00%) 
Cardiac disorders   
Palpitations  1  4/6 (66.67%) 
Atrial fibrillation  1  1/6 (16.67%) 
Atrioventricular block  1  1/6 (16.67%) 
Bradycardia  1  1/6 (16.67%) 
Bundle branch block right  1  1/6 (16.67%) 
Left ventricular hypertrophy  1  1/6 (16.67%) 
Supraventricular extrasystoles  1  1/6 (16.67%) 
Tachycardia  1  1/6 (16.67%) 
Ventricular hypertrophy  1  1/6 (16.67%) 
Gastrointestinal disorders   
Abdominal pain upper  1  1/6 (16.67%) 
Colonic polyp  1  1/6 (16.67%) 
Dental caries  1  1/6 (16.67%) 
Diarrhoea  1  1/6 (16.67%) 
Gastritis erosive  1  1/6 (16.67%) 
Nausea  1  1/6 (16.67%) 
Oral pain  1  1/6 (16.67%) 
Stomatitis  1  1/6 (16.67%) 
Vomiting  1  1/6 (16.67%) 
General disorders   
Oedema  1  2/6 (33.33%) 
Chest discomfort  1  1/6 (16.67%) 
Chest pain  1  1/6 (16.67%) 
Chills  1  1/6 (16.67%) 
Feeling hot  1  1/6 (16.67%) 
Injection site inflammation  1  1/6 (16.67%) 
Puncture site haemorrhage  1  1/6 (16.67%) 
Tenderness  1  1/6 (16.67%) 
Infections and infestations   
Nasopharyngitis  1  5/6 (83.33%) 
Chronic sinusitis  1  1/6 (16.67%) 
Cystitis  1  1/6 (16.67%) 
Pharyngitis  1  1/6 (16.67%) 
Rhinitis  1  1/6 (16.67%) 
Investigations   
Aspartate aminotransferase increased  1  2/6 (33.33%) 
Alanine aminotransferase increased  1  1/6 (16.67%) 
Blood pressure decreased  1  1/6 (16.67%) 
Blood urea increased  1  1/6 (16.67%) 
Body temperature decreased  1  1/6 (16.67%) 
Heart rate increased  1  1/6 (16.67%) 
Occult blood  1  1/6 (16.67%) 
Protein urine present  1  1/6 (16.67%) 
White blood cell count decreased  1  1/6 (16.67%) 
Metabolism and nutrition disorders   
Dehydration  1  1/6 (16.67%) 
Hyperlipidaemia  1  1/6 (16.67%) 
Musculoskeletal and connective tissue disorders   
Back pain  1  3/6 (50.00%) 
Pain in extremity  1  2/6 (33.33%) 
Arthralgia  1  1/6 (16.67%) 
Joint swelling  1  1/6 (16.67%) 
Musculoskeletal stiffness  1  1/6 (16.67%) 
Neck pain  1  1/6 (16.67%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Large intestine carcinoma  1  1/6 (16.67%) 
Nervous system disorders   
Headache  1  2/6 (33.33%) 
Dizziness  1  1/6 (16.67%) 
Intercostal neuralgia  1  1/6 (16.67%) 
Paraesthesia  1  1/6 (16.67%) 
Syncope  1  1/6 (16.67%) 
Psychiatric disorders   
Anxiety disorder  1  2/6 (33.33%) 
Insomnia  1  1/6 (16.67%) 
Middle insomnia  1  1/6 (16.67%) 
Renal and urinary disorders   
Dysuria  1  1/6 (16.67%) 
Respiratory, thoracic and mediastinal disorders   
Rhinitis allergic  1  1/6 (16.67%) 
Throat tightness  1  1/6 (16.67%) 
Skin and subcutaneous tissue disorders   
Pruritus  1  2/6 (33.33%) 
Dermatitis contact  1  1/6 (16.67%) 
Eczema  1  1/6 (16.67%) 
Rash  1  1/6 (16.67%) 
Seborrhoeic dermatitis  1  1/6 (16.67%) 
Urticaria  1  1/6 (16.67%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDra 15.0
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.
Prior to publishing results from this study in academic meetings, medical journals, etc., the investigator and subinvestigator of the post-manufacturing/marketing clinical study must obtain the approval from the sponsor of the post-manufacturing/marketing clinical study in writing.
Results Point of Contact
Name/Title: Trial Transparency Team
Organization: Sanofi
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00140621     History of Changes
Other Study ID Numbers: AGAL03204
First Submitted: August 30, 2005
First Posted: September 1, 2005
Results First Submitted: April 1, 2015
Results First Posted: April 15, 2015
Last Update Posted: May 12, 2015