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Trial record 2 of 68 for:    replagal | Fabry Disease

Safety Study of Replagal® Therapy in Children 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: NCT01363492
Recruitment Status : Completed
First Posted : June 1, 2011
Results First Posted : May 20, 2014
Last Update Posted : May 20, 2014
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fabry Disease
Intervention Biological: Replagal (agalsidase alfa)
Enrollment 15
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Replagal® (0.2 mg/kg)
Hide Arm/Group Description 0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
Period Title: Overall Study
Started 14
Completed 14
Not Completed 0
Arm/Group Title Replagal (0.2 mg/kg)
Hide Arm/Group Description 0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Baseline Participants 14
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 14 participants
12.16  (2.992)
Age, Customized   [1] 
Measure Type: Number
Unit of measure:  Participants
<=18 years Number Analyzed 14 participants
14
[1]
Measure Description: Age at informed consent
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants
Female
9
  64.3%
Male
5
  35.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
UNITED STATES Number Analyzed 14 participants
14
Heart rate variability parameter SDNN  
Mean (Standard Deviation)
Unit of measure:  Msec
Number Analyzed 14 participants
103.46  (32.928)
Heart rate variability parameter rMSSD  
Mean (Standard Deviation)
Unit of measure:  Msec
Number Analyzed 14 participants
75.92  (45.747)
Heart rate variability parameter pNN50  
Mean (Standard Deviation)
Unit of measure:  Msec
Number Analyzed 14 participants
32.79  (19.997)
Left Ventricular Mass Index (LVMI)  
Mean (Standard Deviation)
Unit of measure:  (g/m^2.7)
Number Analyzed 14 participants
35.37  (10.129)
Midwall Fractional Shortening (MFS)  
Mean (Standard Deviation)
Unit of measure:  (%)
Number Analyzed 14 participants
18.63  (2.891)
Plasma Gb3  
Mean (Standard Deviation)
Unit of measure:  (nmol/mL)
Number Analyzed 14 participants
14.79  (12.228)
Urine Gb3  
Mean (Standard Deviation)
Unit of measure:  (nmol/g creatinine)
Number Analyzed 14 participants
1775.08  (3691.087)
1.Primary Outcome
Title Number of Serious Adverse Event (SAE)
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal (0.2 mg/kg)
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: events
0
2.Primary Outcome
Title Number of Treatment Emergent Adverse Event (TEAE)
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal (0.2 mg/kg)
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: events
166
3.Primary Outcome
Title Development of IgG Anti-Agalsidase Alfa Antibody
Hide Description Reflects development of Anti-Agalsidase antibodies post baseline
Time Frame Baseline to Week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal (0.2 mg/kg)
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: participants
1
4.Primary Outcome
Title Change From Baseline in Heart Rate Variability Parameter SDNN
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal 0.2 mg/kg
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: msec
10.46  (24.223)
5.Primary Outcome
Title Change From Baseline in Heart Rate Variability Parameter rMSSD
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal 0.2 mg/kg
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: msec
1.46  (53.502)
6.Primary Outcome
Title Change From Baseline in Heart Rate Variability Parameter pNN50
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal 0.2 mg/kg
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: msec
-4.13  (20.166)
7.Secondary Outcome
Title Change From Baseline in LVMI
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal (0.2 mg/kg)
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: (g/m^2.7)
0.16  (6.059)
8.Secondary Outcome
Title Change From Baseline in MFS
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal (0.2 mg/kg)
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: (%)
-0.62  (3.596)
9.Secondary Outcome
Title Change From Baseline in Plasma Gb3
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal (0.2 mg/kg)
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: (nmol/mL)
-5.71  (8.799)
10.Secondary Outcome
Title Change From Baseline in Urine Gb3
Hide Description [Not Specified]
Time Frame Baseline to week 55
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Replagal (0.2 mg/kg)
Hide Arm/Group Description:
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: (nmol/g creatinine)
-1403.25  (3636.711)
Time Frame Baseline to week 55
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Replagal® (0.2 mg/kg)
Hide Arm/Group Description 0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
All-Cause Mortality
Replagal® (0.2 mg/kg)
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Replagal® (0.2 mg/kg)
Affected / at Risk (%) # Events
Total   0/14 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Replagal® (0.2 mg/kg)
Affected / at Risk (%) # Events
Total   14/14 (100.00%)    
Blood and lymphatic system disorders   
Anaemia  1  1/14 (7.14%)  1
Iron deficiency anaemia  1  1/14 (7.14%)  1
Cardiac disorders   
Sinus bradycardia  1  1/14 (7.14%)  1
Ear and labyrinth disorders   
Ear pain  1  2/14 (14.29%)  2
Eye disorders   
Hordeolum  1  1/14 (7.14%)  1
Retinal vascular disorder  1  1/14 (7.14%)  1
Gastrointestinal disorders   
Abdominal pain  1  1/14 (7.14%)  2
Abdominal pain upper  1  2/14 (14.29%)  2
Diarrhoea  1  4/14 (28.57%)  5
Dyspepsia  1  1/14 (7.14%)  1
Mouth ulceration  1  1/14 (7.14%)  1
Nausea  1  4/14 (28.57%)  12
Tooth malformation  1  1/14 (7.14%)  1
Vomiting  1  4/14 (28.57%)  12
General disorders   
Chest discomfort  1  2/14 (14.29%)  2
Chills  1  2/14 (14.29%)  2
Fatigue  1  1/14 (7.14%)  1
Feeling cold  1  1/14 (7.14%)  2
Malaise  1  1/14 (7.14%)  1
Non-cardiac chest pain  1  1/14 (7.14%)  1
Pain  1  2/14 (14.29%)  2
Pyrexia  1  6/14 (42.86%)  8
Immune system disorders   
Seasonal allergy  1  1/14 (7.14%)  1
Infections and infestations   
Alveolar osteitis  1  1/14 (7.14%)  1
Gastroenteritis  1  2/14 (14.29%)  4
Gastroenteritis viral  1  2/14 (14.29%)  2
Infectious mononucleosis  1  1/14 (7.14%)  1
Nasopharyngitis  1  3/14 (21.43%)  3
Otitis media  1  1/14 (7.14%)  1
Pharyngitis streptococcal  1  2/14 (14.29%)  2
Rhinitis  1  2/14 (14.29%)  2
Sinusitis  1  3/14 (21.43%)  4
Upper respiratory tract infection  1  4/14 (28.57%)  5
Injury, poisoning and procedural complications   
Avulsion fracture  1  1/14 (7.14%)  1
Contusion  1  1/14 (7.14%)  1
Facial bones fracture  1  1/14 (7.14%)  1
Scratch  1  1/14 (7.14%)  1
Tooth fracture  1  1/14 (7.14%)  1
Investigations   
Body temperature increased  1  1/14 (7.14%)  1
Heart rate increased  1  1/14 (7.14%)  1
Liver palpable subcostal  1  1/14 (7.14%)  1
Metabolism and nutrition disorders   
Decreased appetite  1  1/14 (7.14%)  1
Hyperlipidaemia  1  1/14 (7.14%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia  1  2/14 (14.29%)  2
Muscular weakness  1  1/14 (7.14%)  1
Musculoskeletal pain  1  1/14 (7.14%)  1
Pain in extremity  1  4/14 (28.57%)  7
Nervous system disorders   
Dizziness  1  2/14 (14.29%)  2
Headache  1  4/14 (28.57%)  7
Paraesthesia  1  2/14 (14.29%)  2
Psychiatric disorders   
Restlessness  1  1/14 (7.14%)  1
Renal and urinary disorders   
Proteinuria  1  2/14 (14.29%)  2
Respiratory, thoracic and mediastinal disorders   
Asthma  1  1/14 (7.14%)  1
Cough  1  4/14 (28.57%)  8
Dyspnoea  1  3/14 (21.43%)  11
Nasal congestion  1  1/14 (7.14%)  1
Oropharyngeal pain  1  1/14 (7.14%)  1
Pharyngeal erythema  1  2/14 (14.29%)  2
Rhinorrhoea  1  1/14 (7.14%)  1
Throat irritation  1  2/14 (14.29%)  3
Throat tightness  1  1/14 (7.14%)  1
Skin and subcutaneous tissue disorders   
Acne  1  2/14 (14.29%)  2
Angioedema  1  1/14 (7.14%)  1
Erythema  1  1/14 (7.14%)  1
Petechiae  1  1/14 (7.14%)  1
Pityriasis rosea  1  1/14 (7.14%)  1
Pruritus  1  1/14 (7.14%)  1
Rash  1  1/14 (7.14%)  1
Urticaria  1  1/14 (7.14%)  1
Vascular disorders   
Flushing  1  1/14 (7.14%)  4
Hypotension  1  1/14 (7.14%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
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.
Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication
Results Point of Contact
Name/Title: Study Physician
Organization: Shire Development LLC
Phone: +1 866 842 5335
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01363492     History of Changes
Other Study ID Numbers: HGT-REP-084
First Submitted: March 31, 2011
First Posted: June 1, 2011
Results First Submitted: March 25, 2014
Results First Posted: May 20, 2014
Last Update Posted: May 20, 2014