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Trial record 1 of 1 for:    NCT00074958
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A Study of Fabrazyme in Pediatric Patients With 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: NCT00074958
Recruitment Status : Completed
First Posted : December 25, 2003
Results First Posted : June 16, 2009
Last Update Posted : April 2, 2015
Sponsor:
Information provided by:
Sanofi

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fabry Disease
Intervention Biological: Fabrazyme (agalsidase beta)
Enrollment 16
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Fabrazyme
Hide Arm/Group Description 1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
Period Title: Overall Study
Started 16
Completed 15
Not Completed 1
Reason Not Completed
Adverse Event             1
Arm/Group Title Fabrazyme
Hide Arm/Group Description 1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
Overall Number of Baseline Participants 16
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants
12.1  (2.52)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
Female
2
  12.5%
Male
14
  87.5%
1.Primary Outcome
Title Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Hide Description Skin biopsies were taken at Baseline, Week 24 and Week 48 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
Time Frame Baseline, Week 24 and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat (ITT) population - male patients only. 14 patients had skin biopsies performed at Baseline and Week 24 but only 5 patients had skin biopsies performed at Week 48.
Arm/Group Title Fabrazyme
Hide Arm/Group Description:
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: patients
None/Trace (0) Score at Baseline 2
None/Trace (0) Score at Week 24 14
None/Trace (0) Score at Week 48 5
Mild (1) Score at Baseline 0
Mild (1) Score at Week 24 0
Mild (1) Score at Week 48 0
Moderate (2) Score at Baseline 11
Moderate (2) Score at Week 24 0
Moderate (2) Score at Week 48 0
Severe (3) Score at Baseline 1
Severe (3) Score at Week 24 0
Severe (3) Score at Week 48 0
2.Secondary Outcome
Title Plasma GL-3
Hide Description Plasma GL-3 values at Baseline, Week 24, and Week 48. Normal plasma GL-3 level is ≤ 7.03 µg/mL.
Time Frame Baseline, Week 24 and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. 16 male patients had plasma GL-3 values at Baseline and Week 24, while 15 male patients had plasma GL-3 values at Week 48. 2 female patients had plasma GL-3 values at Baseline, Week 24 and Week 48.
Arm/Group Title Fabrazyme
Hide Arm/Group Description:
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
Overall Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: µg/mL
Male Patients at Baseline 15.4  (4.10)
Female Patients at Baseline 4.9  (0.64)
Overall at Baseline 14.1  (5.25)
Males at Week 24 5.5  (0.95)
Females at Week 24 3.6  (0.57)
Overall at Week 24 5.2  (1.10)
Males at Week 48 5.0  (1.00)
Females at Week 48 4.4  (1.27)
Overall at Week 48 4.9  (1.01)
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
Hide Arm/Group Description 1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
All-Cause Mortality
Fabrazyme
Affected / at Risk (%)
Total   --/-- 
Hide Serious Adverse Events
Fabrazyme
Affected / at Risk (%)
Total   1/16 (6.25%) 
Immune system disorders   
Anaphylactic shock † 1  1/16 (6.25%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 8.1
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Fabrazyme
Affected / at Risk (%)
Total   16/16 (100.00%) 
Blood and lymphatic system disorders   
Anaemia † 1  1/16 (6.25%) 
Lymphadenopathy † 1  2/16 (12.50%) 
Cardiac disorders   
Angina pectoris † 1  1/16 (6.25%) 
Arrhythmia † 1  1/16 (6.25%) 
Tachycardia † 1  1/16 (6.25%) 
Ear and labyrinth disorders   
Ear pain † 1  1/16 (6.25%) 
Tinnitus † 1  1/16 (6.25%) 
Vertigo † 1  1/16 (6.25%) 
Eye disorders   
Eye oedema † 1  1/16 (6.25%) 
Lacrimation increased † 1  2/16 (12.50%) 
Photophobia † 1  1/16 (6.25%) 
Vision blurred † 1  2/16 (12.50%) 
Gastrointestinal disorders   
Abdominal pain † 1  7/16 (43.75%) 
Abdominal pain upper † 1  4/16 (25.00%) 
Chapped lips † 1  1/16 (6.25%) 
Constipation † 1  3/16 (18.75%) 
Diarrhoea † 1  5/16 (31.25%) 
Dyspepsia † 1  3/16 (18.75%) 
Nausea † 1  9/16 (56.25%) 
Toothache † 1  2/16 (12.50%) 
Vomiting † 1  9/16 (56.25%) 
General disorders   
Asthenia † 1  2/16 (12.50%) 
Catheter site pain † 1  1/16 (6.25%) 
Chest pain † 1  2/16 (12.50%) 
Chills † 1  3/16 (18.75%) 
Fatigue † 1  1/16 (6.25%) 
Feeling cold † 1  2/16 (12.50%) 
Feeling hot † 1  2/16 (12.50%) 
Hyperthermia † 1  1/16 (6.25%) 
Injection site pain † 1  1/16 (6.25%) 
Malaise † 1  1/16 (6.25%) 
Oedema peripheral † 1  1/16 (6.25%) 
Pain † 1  1/16 (6.25%) 
Pyrexia † 1  7/16 (43.75%) 
Immune system disorders   
Seasonal allergy † 1  1/16 (6.25%) 
Infections and infestations   
Gastroenteritis † 1  1/16 (6.25%) 
Herpes simplex † 1  1/16 (6.25%) 
Hordeolum † 1  1/16 (6.25%) 
Infection † 1  1/16 (6.25%) 
Nasopharyngitis † 1  4/16 (25.00%) 
Pharyngitis † 1  4/16 (25.00%) 
Tonsillitis † 1  1/16 (6.25%) 
Viral infection † 1  1/16 (6.25%) 
Wound infection † 1  1/16 (6.25%) 
Injury, poisoning and procedural complications   
Concussion † 1  1/16 (6.25%) 
Contusion † 1  1/16 (6.25%) 
Fall † 1  2/16 (12.50%) 
Head injury † 1  1/16 (6.25%) 
Joint injury † 1  1/16 (6.25%) 
Joint sprain † 1  2/16 (12.50%) 
Post-traumatic pain † 1  1/16 (6.25%) 
Procedural complication † 1  1/16 (6.25%) 
Procedural pain † 1  1/16 (6.25%) 
Testicular injury † 1  1/16 (6.25%) 
Tibia fracture † 1  1/16 (6.25%) 
Wound † 1  1/16 (6.25%) 
Investigations   
Albumin urine present † 1  1/16 (6.25%) 
Blood bilirubin increased † 1  1/16 (6.25%) 
Blood pressure increased † 1  1/16 (6.25%) 
Body temperature increased † 1  3/16 (18.75%) 
Creatinine renal clearance decreased † 1  1/16 (6.25%) 
Heart rate increased † 1  1/16 (6.25%) 
Metabolism and nutrition disorders   
Anorexia † 1  2/16 (12.50%) 
Musculoskeletal and connective tissue disorders   
Arthralgia † 1  4/16 (25.00%) 
Back pain † 1  2/16 (12.50%) 
Chest wall pain † 1  1/16 (6.25%) 
Flank pain † 1  1/16 (6.25%) 
Groin pain † 1  1/16 (6.25%) 
Muscle spasms † 1  1/16 (6.25%) 
Myalgia † 1  1/16 (6.25%) 
Pain in extremity † 1  4/16 (25.00%) 
Shoulder pain † 1  3/16 (18.75%) 
Nervous system disorders   
Cognitive disorder † 1  1/16 (6.25%) 
Coordination abnormal † 1  1/16 (6.25%) 
Dizziness † 1  4/16 (25.00%) 
Dyspraxia † 1  1/16 (6.25%) 
Headache † 1  10/16 (62.50%) 
Hyperreflexia † 1  1/16 (6.25%) 
Lethargy † 1  2/16 (12.50%) 
Paraesthesia † 1  6/16 (37.50%) 
Tremor † 1  1/16 (6.25%) 
Psychiatric disorders   
Insomnia † 1  1/16 (6.25%) 
Renal and urinary disorders   
Microalbuminuria † 1  1/16 (6.25%) 
Respiratory, thoracic and mediastinal disorders   
Cough † 1  3/16 (18.75%) 
Nasal congestion † 1  2/16 (12.50%) 
Pharyngolaryngeal pain † 1  4/16 (25.00%) 
Rhinitis allergic † 1  2/16 (12.50%) 
Rhinorrhoea † 1  4/16 (25.00%) 
Sneezing † 1  1/16 (6.25%) 
Throat irritation † 1  1/16 (6.25%) 
Skin and subcutaneous tissue disorders   
Angiokeratoma † 1  3/16 (18.75%) 
Hyperhidrosis † 1  1/16 (6.25%) 
Pruritus † 1  1/16 (6.25%) 
Rash † 1  1/16 (6.25%) 
Rash erythematous † 1  1/16 (6.25%) 
Rash generalised † 1  1/16 (6.25%) 
Urticaria localised † 1  1/16 (6.25%) 
Vascular disorders   
Hypotension † 1  1/16 (6.25%) 
Pallor † 1  1/16 (6.25%) 
Poor peripheral circulation † 1  1/16 (6.25%) 
Vasoconstriction † 1  1/16 (6.25%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 8.1
The trial was not powered to demonstrate clinical benefit.
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
Layout table for Results Point of Contact information
Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
Phone: 800-745-4447
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00074958    
Other Study ID Numbers: AGAL-016-01
First Submitted: December 24, 2003
First Posted: December 25, 2003
Results First Submitted: March 3, 2009
Results First Posted: June 16, 2009
Last Update Posted: April 2, 2015