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Trial record 1 of 1 for:    NCT00837824
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Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

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ClinicalTrials.gov Identifier: NCT00837824
Recruitment Status : Terminated (The trial was terminated early due to inadequate study design.)
First Posted : February 5, 2009
Results First Posted : February 5, 2009
Last Update Posted : April 7, 2015
Sponsor:
Collaborator:
CRL/Medinet
Information provided by:
Sanofi

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Fabry Disease
Chronic Kidney Disease, Stage IV (Severe)
Intervention Biological: Fabrazyme (agalsidase beta)
Enrollment 20
Recruitment Details  
Pre-assignment Details Because the study was ended early only 20 patients from 9 sites were randomized into this study using a 1:1 randomization scheme to receive biweekly infusions of either 1 mg/kg (11 patients) or 3 mg/kg (9 patients) of Fabrazyme.
Arm/Group Title Fabrazyme 1mg/kg Every 2 Weeks Fabrazyme 3mg/kg Every 2 Weeks
Hide Arm/Group Description Fabrazyme 1.0 mg/kg every 2 weeks Fabrazyme 3.0 mg/kg every 2 weeks
Period Title: Overall Study
Started 11 9
Completed 11 9
Not Completed 0 0
Arm/Group Title Fabrazyme 1mg/kg Every 2 Weeks Fabrazyme 3mg/kg Every 2 Weeks Total
Hide Arm/Group Description Fabrazyme 1.0 mg/kg every 2 weeks Fabrazyme 3.0 mg/kg every 2 weeks Total of all reporting groups
Overall Number of Baseline Participants 11 9 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Number
Unit of measure:  Years
Number Analyzed 11 participants 9 participants 20 participants
< 40 Years 1 1 2
≥ 40 Years 10 8 18
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants 9 participants 20 participants
49.4  (6.96) 48.9  (7.55) 49.2  (7.04)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 9 participants 20 participants
Female
1
   9.1%
2
  22.2%
3
  15.0%
Male
10
  90.9%
7
  77.8%
17
  85.0%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Number of Participants
Number Analyzed 11 participants 9 participants 20 participants
Caucasian 7 7 14
Black 3 2 5
Hispanic 1 0 1
1.Primary Outcome
Title Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease
Hide Description The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.
Time Frame 7 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population-consisted of all 20 patients enrolled in the trial. The original sample size was 120 patients. Due to early termination, only 20 patients were enrolled in this trial. No imputation of data was performed. During the 7 months study period, only 1 patient had a clinical event in the Fabrazyme 1 mg/kg treatment arm.
Arm/Group Title Fabrazyme 1mg/kg Every 2 Weeks Fabrazyme 3mg/kg Every 2 Weeks
Hide Arm/Group Description:
Fabrazyme 1.0 mg/kg every 2 weeks
Fabrazyme 3.0 mg/kg every 2 weeks
Overall Number of Participants Analyzed 1 0
Measure Type: Number
Unit of Measure: Days
35
2.Secondary Outcome
Title Plasma Globotriaosylceramide (GL-3)
Hide Description This outcome measure evaluated the mean plasma GL-3 values for all patients to see if it decreased while on Fabrazyme. Normal plasma GL-3 level is defined as ≤ 7.03 µg/mL.
Time Frame Evaluated at Baseline, Month 3, and Final Visit
Hide Outcome Measure Data
Hide Analysis Population Description

Fabrazyme 1mg/kg every 2 weeks: ITT population – 11 patients at baseline, 7 patients at Month 3, and 9 patients at Final Visit had Plasma GL-3 values.

Fabrazyme 3mg/kg every 2 weeks: ITT population - 9 patients at baseline, 2 patients at Month 3, and 7 patients at Final Visit had Plasma GL-3 values

Arm/Group Title Fabrazyme 1mg/kg Every 2 Weeks Fabrazyme 3mg/kg Every 2 Weeks
Hide Arm/Group Description:
Fabrazyme 1.0 mg/kg every 2 weeks
Fabrazyme 3.0 mg/kg every 2 weeks
Overall Number of Participants Analyzed 11 9
Mean (Standard Deviation)
Unit of Measure: µg/mL
Plasma GL-3 at Baseline 8.7  (3.33) 8.3  (3.92)
Plasma GL-3 at Month 3 5.1  (2.05) 5.6  (0.28)
Plasma GL-3 at Final Visit 5.1  (1.53) 4.9  (1.74)
Time Frame [Not Specified]
Adverse Event Reporting Description In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
 
Arm/Group Title Fabrazyme 1mg/kg Every 2 Weeks Fabrazyme 3mg/kg Every 2 Weeks Total
Hide Arm/Group Description Fabrazyme 1.0 mg/kg every 2 weeks Fabrazyme 3.0 mg/kg every 2 weeks Total of all reporting groups
All-Cause Mortality
Fabrazyme 1mg/kg Every 2 Weeks Fabrazyme 3mg/kg Every 2 Weeks Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Fabrazyme 1mg/kg Every 2 Weeks Fabrazyme 3mg/kg Every 2 Weeks Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   4/11 (36.36%)   1/9 (11.11%)   5/20 (25.00%) 
Cardiac disorders       
Atrial fibrillation  1  2/11 (18.18%)  0/9 (0.00%)  2/20 (10.00%) 
Atrial flutter  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Coronary artery occlusion  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
General disorders       
Pyrexia  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Infections and infestations       
Sepsis  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Urinary tract infection  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Injury, poisoning and procedural complications       
Arteriovenous fistula thrombosis  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Nervous system disorders       
Cerebrovascular accident  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 8.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Fabrazyme 1mg/kg Every 2 Weeks Fabrazyme 3mg/kg Every 2 Weeks Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   10/11 (90.91%)   9/9 (100.00%)   19/20 (95.00%) 
Blood and lymphatic system disorders       
Lymphadenopathy  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Ear and labyrinth disorders       
Ear congestion  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Ear discomfort  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Gastrointestinal disorders       
Abdominal distension  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Abdominal pain  1  2/11 (18.18%)  0/9 (0.00%)  2/20 (10.00%) 
Constipation  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Diarrhoea  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Food poisoning  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Gastrointestinal hypermotility  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Vomiting  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
General disorders       
Adverse event  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Chills  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Fatigue  1  1/11 (9.09%)  1/9 (11.11%)  2/20 (10.00%) 
Feeling hot  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Influenza like illness  1  1/11 (9.09%)  1/9 (11.11%)  2/20 (10.00%) 
Malaise  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Oedema peripheral  1  0/11 (0.00%)  3/9 (33.33%)  3/20 (15.00%) 
Pain  1  1/11 (9.09%)  1/9 (11.11%)  2/20 (10.00%) 
Pyrexia  1  2/11 (18.18%)  0/9 (0.00%)  2/20 (10.00%) 
Sluggishness  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Infections and infestations       
Arteriovenous fistula site infection  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Nasopharyngitis  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Upper respiratory tract infection  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Injury, poisoning and procedural complications       
Excoriation  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Joint sprain  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Muscle strain  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Investigations       
Blood bicarbonate decreased  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Blood calcium decreased  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Blood phosphorus increased  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Breath sounds abnormal  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Haematocrit decreased  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Haemoglobin decreased  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Red blood cell count decreased  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Metabolism and nutrition disorders       
Gout  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Hypokalaemia  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Hypovolaemia  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Musculoskeletal and connective tissue disorders       
Limb discomfort  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Muscle spasms  1  0/11 (0.00%)  2/9 (22.22%)  2/20 (10.00%) 
Musculoskeletal discomfort  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Pain in extremity  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Nervous system disorders       
Dizziness  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Headache  1  1/11 (9.09%)  2/9 (22.22%)  3/20 (15.00%) 
Restless legs syndrome  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Sinus headache  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Somnolence  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Speech disorder  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Transient ischaemic attack  1  2/11 (18.18%)  0/9 (0.00%)  2/20 (10.00%) 
Respiratory, thoracic and mediastinal disorders       
Cough  1  1/11 (9.09%)  1/9 (11.11%)  2/20 (10.00%) 
Epistaxis  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Nasal congestion  1  0/11 (0.00%)  1/9 (11.11%)  1/20 (5.00%) 
Pharyngolaryngeal pain  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Productive cough  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Skin and subcutaneous tissue disorders       
Night sweats  1  1/11 (9.09%)  0/9 (0.00%)  1/20 (5.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 8.1
The trial was terminated early due to inadequate study design, leading to small numbers of subjects analyzed.
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.
In multi-site studies, PI can publish after Genzyme publishes or 18 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 60 days upon notifying PI that it will file a patent application on inventions contained in the draft.
Results Point of Contact
Name/Title: Genzyme MedInfo
Organization: Genzyme Corporation
Phone: 800-745-4447
Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00837824     History of Changes
Other Study ID Numbers: AGAL-022-02
First Submitted: October 23, 2008
First Posted: February 5, 2009
Results First Submitted: December 5, 2008
Results First Posted: February 5, 2009
Last Update Posted: April 7, 2015