Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 32 of 103 for:    Pompe Disease

A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe 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: NCT01526785
Recruitment Status : Terminated (Terminated due to approved label expansion)
First Posted : February 6, 2012
Results First Posted : October 27, 2015
Last Update Posted : December 8, 2015
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pompe Disease
Intervention Drug: Alglucosidase alfa
Enrollment 113
Recruitment Details  
Pre-assignment Details The study was conducted in the United States. A total of 113 participants were treated between 9 March 2012 and 4 June 2014.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Alglucosidase alfa (4000 litre [L] scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.
Period Title: Overall Study
Started 113
Completed 100
Not Completed 13
Reason Not Completed
Study Terminated by Sponsor             8
Death             2
Physician Decision             1
Withdrawal by Subject             2
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per physician's routine practice.
Overall Number of Baseline Participants 113
Hide Baseline Analysis Population Description
Full analysis population defined as all participants who receive at least 1 infusion of 4000 L alglucosidase alfa during the study.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 113 participants
4.8  (3.73)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 113 participants
Female
53
  46.9%
Male
60
  53.1%
1.Primary Outcome
Title Percentage of Participants Who Were Clinically Stable or Improved at Week 52
Hide Description Clinical stability was defined as absence of death due to disease progression or new dependency on invasive ventilation and; decline in cardiac status, motor function, and pulmonary function from baseline.
Time Frame Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population. Number of participants analyzed = participants with available data at Week 52 for this outcome.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per physician's routine practice.
Overall Number of Participants Analyzed 104
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
83.7
(75.1 to 90.2)
2.Secondary Outcome
Title Survival Rate at Week 52
Hide Description Percentage of participants who were alive at Week 52, were reported. Survival rate was calculated by Kaplan-Meier estimate.
Time Frame Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per physician's routine practice.
Overall Number of Participants Analyzed 113
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
98.1
(92.73 to 99.53)
3.Secondary Outcome
Title Invasive Ventilator-Free Survival Rate at Week 52
Hide Description Percentage of participants, who were invasive ventilator-free at week 52, are reported. Invasive ventilation was defined as mechanical ventilatory support applied with the use of an endotracheal tube or tracheostomy. Invasive ventilator-free survival rate was calculated by Kaplan-Meier estimate.
Time Frame Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population. Number of participants analyzed = participants with available data at Week 52 for this outcome.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per physician's routine practice.
Overall Number of Participants Analyzed 81
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
92.4
(83.89 to 96.53)
4.Secondary Outcome
Title Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52
Hide Description Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates an increase in LVM-Z score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population. Number of participants analyzed = participants with available data at Week 52 for this outcome.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per physician's routine practice.
Overall Number of Participants Analyzed 67
Mean (Standard Deviation)
Unit of Measure: Z score
-0.5  (1.71)
5.Secondary Outcome
Title Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52
Hide Description GMFM-88 (88-item measure to detect gross motor function) consists of 5 components, each measured on a 4-point Likert scale.The score for each dimension was expressed as a percentage of the maximum score for that dimension.Total score ranges from 0% to 100%, where higher scores indicate better motor functions.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population. Number of participants analysed = participants with baseline and Week 52 GMFM-88 data.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per physician's routine practice.
Overall Number of Participants Analyzed 90
Mean (Standard Deviation)
Unit of Measure: percentage of total score
3.7  (17.46)
6.Secondary Outcome
Title Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position
Hide Description Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population. Number of participants analysed = participants with baseline and Week 52 FVC data.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per physician's routine practice.
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: percent predicted FVC
3.2  (11.07)
7.Secondary Outcome
Title Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position
Hide Description Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population. Number of participants analysed = participants with baseline and Week 52 FVC data.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per physician's routine practice.
Overall Number of Participants Analyzed 22
Mean (Standard Deviation)
Unit of Measure: percent predicted FVC
2.3  (11.8)
Time Frame All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (Week 52) 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 infusion of study drug up to 30 days after last infusion of study drug). Analysis was performed on safety population which included all treated participants.
 
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Alglucosidase alfa (4000 L scale) IV infusion administered for 52 weeks as per participant's routine practice.
All-Cause Mortality
Alglucosidase Alfa
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Alglucosidase Alfa
Affected / at Risk (%)
Total   73/113 (64.60%) 
Cardiac disorders   
Bradycardia  1  2/113 (1.77%) 
Cardiac arrest  1  1/113 (0.88%) 
Cardiac failure congestive  1  1/113 (0.88%) 
Cardio-respiratory arrest  1  3/113 (2.65%) 
Cardiopulmonary failure  1  1/113 (0.88%) 
Cyanosis  1  1/113 (0.88%) 
Left ventricular hypertrophy  1  2/113 (1.77%) 
Mitral valve incompetence  1  1/113 (0.88%) 
Pulseless electrical activity  1  1/113 (0.88%) 
Sinus tachycardia  1  1/113 (0.88%) 
Supraventricular tachycardia  1  4/113 (3.54%) 
Tachycardia  1  1/113 (0.88%) 
Ear and labyrinth disorders   
Motion sickness  1  1/113 (0.88%) 
Eye disorders   
Eyelid ptosis  1  1/113 (0.88%) 
Gastrointestinal disorders   
Abdominal pain  1  2/113 (1.77%) 
Constipation  1  2/113 (1.77%) 
Dental caries  1  1/113 (0.88%) 
Diarrhoea  1  1/113 (0.88%) 
Dysphagia  1  1/113 (0.88%) 
Gastrointestinal disorder  1  1/113 (0.88%) 
Gastrooesophageal reflux disease  1  1/113 (0.88%) 
Intussusception  1  1/113 (0.88%) 
Nausea  1  1/113 (0.88%) 
Vomiting  1  2/113 (1.77%) 
General disorders   
Chest pain  1  2/113 (1.77%) 
Chills  1  2/113 (1.77%) 
Device breakage  1  1/113 (0.88%) 
Device malfunction  1  1/113 (0.88%) 
Hypothermia  1  1/113 (0.88%) 
Oedema peripheral  1  1/113 (0.88%) 
Pyrexia  1  10/113 (8.85%) 
Hepatobiliary disorders   
Cholecystitis acute  1  1/113 (0.88%) 
Infections and infestations   
Adenovirus infection  1  1/113 (0.88%) 
Bacteraemia  1  2/113 (1.77%) 
Bacterial tracheitis  1  2/113 (1.77%) 
Bronchiolitis  1  2/113 (1.77%) 
Cellulitis  1  1/113 (0.88%) 
Device related infection  1  6/113 (5.31%) 
Gastroenteritis  1  1/113 (0.88%) 
Gastroenteritis viral  1  3/113 (2.65%) 
Haemophilus bacteraemia  1  1/113 (0.88%) 
Influenza  1  2/113 (1.77%) 
Klebsiella bacteraemia  1  1/113 (0.88%) 
Lobar pneumonia  1  1/113 (0.88%) 
Lung infection  1  1/113 (0.88%) 
Metapneumovirus infection  1  3/113 (2.65%) 
Otitis media acute  1  1/113 (0.88%) 
Parainfluenzae virus infection  1  1/113 (0.88%) 
Periumbilical abscess  1  1/113 (0.88%) 
Pneumonia  1  25/113 (22.12%) 
Pneumonia parainfluenzae viral  1  1/113 (0.88%) 
Pneumonia pseudomonal  1  1/113 (0.88%) 
Pneumonia respiratory syncytial viral  1  2/113 (1.77%) 
Pneumonia viral  1  3/113 (2.65%) 
Respiratory syncytial virus bronchiolitis  1  1/113 (0.88%) 
Respiratory syncytial virus infection  1  1/113 (0.88%) 
Respiratory tract infection  1  4/113 (3.54%) 
Respiratory tract infection viral  1  1/113 (0.88%) 
Rhinovirus infection  1  3/113 (2.65%) 
Sepsis  1  2/113 (1.77%) 
Staphylococcal infection  1  1/113 (0.88%) 
Tracheitis  1  5/113 (4.42%) 
Tracheostomy infection  1  1/113 (0.88%) 
Upper respiratory tract infection  1  2/113 (1.77%) 
Urinary tract infection  1  3/113 (2.65%) 
Urinary tract infection bacterial  1  1/113 (0.88%) 
Urinary tract infection staphylococcal  1  1/113 (0.88%) 
Urosepsis  1  1/113 (0.88%) 
Viral infection  1  2/113 (1.77%) 
Injury, poisoning and procedural complications   
Feeding tube complication  1  1/113 (0.88%) 
Femur fracture  1  1/113 (0.88%) 
Foreign body  1  1/113 (0.88%) 
Foreign body aspiration  1  1/113 (0.88%) 
Tracheal obstruction  1  1/113 (0.88%) 
Traumatic haemothorax  1  1/113 (0.88%) 
Investigations   
Alanine aminotransferase increased  1  1/113 (0.88%) 
Aspartate aminotransferase increased  1  1/113 (0.88%) 
Blood albumin decreased  1  1/113 (0.88%) 
Gamma-glutamyltransferase increased  1  1/113 (0.88%) 
Oxygen saturation decreased  1  3/113 (2.65%) 
Metabolism and nutrition disorders   
Dehydration  1  3/113 (2.65%) 
Failure to thrive  1  1/113 (0.88%) 
Fluid overload  1  1/113 (0.88%) 
Hypoglycaemia  1  1/113 (0.88%) 
Musculoskeletal and connective tissue disorders   
Muscle contracture  1  1/113 (0.88%) 
Muscular weakness  1  2/113 (1.77%) 
Nervous system disorders   
Brain injury  1  1/113 (0.88%) 
Cerebral ischaemia  1  1/113 (0.88%) 
Convulsion  1  2/113 (1.77%) 
Gross motor delay  1  1/113 (0.88%) 
Loss of consciousness  1  1/113 (0.88%) 
Presyncope  1  1/113 (0.88%) 
Tremor  1  1/113 (0.88%) 
Psychiatric disorders   
Mental status changes  1  1/113 (0.88%) 
Self-injurious ideation  1  1/113 (0.88%) 
Renal and urinary disorders   
Urinary retention  1  3/113 (2.65%) 
Respiratory, thoracic and mediastinal disorders   
Acute respiratory distress syndrome  1  2/113 (1.77%) 
Acute respiratory failure  1  2/113 (1.77%) 
Apnoea  1  1/113 (0.88%) 
Aspiration  1  1/113 (0.88%) 
Atelectasis  1  3/113 (2.65%) 
Bronchial secretion retention  1  1/113 (0.88%) 
Bronchiectasis  1  2/113 (1.77%) 
Chronic respiratory failure  1  1/113 (0.88%) 
Cough  1  2/113 (1.77%) 
Dyspnoea  1  5/113 (4.42%) 
Hypoxia  1  3/113 (2.65%) 
Pneumonia aspiration  1  4/113 (3.54%) 
Pneumothorax  1  1/113 (0.88%) 
Pulmonary granuloma  1  1/113 (0.88%) 
Respiratory disorder  1  1/113 (0.88%) 
Respiratory distress  1  16/113 (14.16%) 
Respiratory failure  1  7/113 (6.19%) 
Respiratory tract congestion  1  1/113 (0.88%) 
Rhonchi  1  1/113 (0.88%) 
Skin and subcutaneous tissue disorders   
Hyperhidrosis  1  1/113 (0.88%) 
Purpura  1  1/113 (0.88%) 
Rash  1  1/113 (0.88%) 
Surgical and medical procedures   
Central venous catheterisation  1  1/113 (0.88%) 
Therapeutic procedure  1  1/113 (0.88%) 
Vascular disorders   
Hypotension  1  1/113 (0.88%) 
Jugular vein thrombosis  1  1/113 (0.88%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Alglucosidase Alfa
Affected / at Risk (%)
Total   113/113 (100.00%) 
Blood and lymphatic system disorders   
Anaemia  1  7/113 (6.19%) 
Coagulopathy  1  1/113 (0.88%) 
Eosinophilia  1  6/113 (5.31%) 
Iron deficiency anaemia  1  2/113 (1.77%) 
Lymphadenitis  1  1/113 (0.88%) 
Lymphadenopathy  1  1/113 (0.88%) 
Neutropenia  1  1/113 (0.88%) 
Cardiac disorders   
Arrhythmia  1  3/113 (2.65%) 
Bradycardia  1  2/113 (1.77%) 
Bundle branch block right  1  1/113 (0.88%) 
Cardiac failure  1  2/113 (1.77%) 
Cardiomyopathy  1  1/113 (0.88%) 
Defect conduction intraventricular  1  4/113 (3.54%) 
Diastolic dysfunction  1  1/113 (0.88%) 
Heart valve incompetence  1  1/113 (0.88%) 
Left ventricular dysfunction  1  1/113 (0.88%) 
Pericardial effusion  1  5/113 (4.42%) 
Right atrial dilatation  1  1/113 (0.88%) 
Right ventricular hypertrophy  1  1/113 (0.88%) 
Sinus bradycardia  1  1/113 (0.88%) 
Sinus tachycardia  1  2/113 (1.77%) 
Supraventricular tachycardia  1  3/113 (2.65%) 
Tachycardia  1  13/113 (11.50%) 
Tricuspid valve incompetence  1  1/113 (0.88%) 
Wolff-Parkinson-White syndrome  1  2/113 (1.77%) 
Congenital, familial and genetic disorders   
Cryptorchism  1  1/113 (0.88%) 
Ear and labyrinth disorders   
Cerumen impaction  1  3/113 (2.65%) 
Conductive deafness  1  1/113 (0.88%) 
Deafness  1  1/113 (0.88%) 
Deafness neurosensory  1  1/113 (0.88%) 
Deafness unilateral  1  1/113 (0.88%) 
Ear pain  1  5/113 (4.42%) 
Eustachian tube dysfunction  1  5/113 (4.42%) 
Middle ear effusion  1  5/113 (4.42%) 
Mixed deafness  1  1/113 (0.88%) 
Otorrhoea  1  3/113 (2.65%) 
Tympanic membrane disorder  1  1/113 (0.88%) 
Tympanic membrane perforation  1  1/113 (0.88%) 
Endocrine disorders   
Precocious puberty  1  2/113 (1.77%) 
Eye disorders   
Astigmatism  1  2/113 (1.77%) 
Blepharitis  1  1/113 (0.88%) 
Chalazion  1  1/113 (0.88%) 
Conjunctival hyperaemia  1  1/113 (0.88%) 
Dry eye  1  4/113 (3.54%) 
Eye discharge  1  1/113 (0.88%) 
Eye irritation  1  1/113 (0.88%) 
Eye pruritus  1  1/113 (0.88%) 
Eye swelling  1  1/113 (0.88%) 
Eyelid oedema  1  1/113 (0.88%) 
Eyelid ptosis  1  4/113 (3.54%) 
Hypermetropia  1  1/113 (0.88%) 
Lid sulcus deepened  1  1/113 (0.88%) 
Myopia  1  2/113 (1.77%) 
Ocular hyperaemia  1  1/113 (0.88%) 
Photophobia  1  1/113 (0.88%) 
Strabismus  1  1/113 (0.88%) 
Vision blurred  1  1/113 (0.88%) 
Gastrointestinal disorders   
Abdominal discomfort  1  3/113 (2.65%) 
Abdominal distension  1  1/113 (0.88%) 
Abdominal pain  1  10/113 (8.85%) 
Abdominal pain upper  1  11/113 (9.73%) 
Abnormal faeces  1  1/113 (0.88%) 
Anal fissure  1  1/113 (0.88%) 
Aphthous stomatitis  1  1/113 (0.88%) 
Constipation  1  20/113 (17.70%) 
Dental caries  1  3/113 (2.65%) 
Dental discomfort  1  1/113 (0.88%) 
Diarrhoea  1  55/113 (48.67%) 
Dysphagia  1  2/113 (1.77%) 
Faecal incontinence  1  1/113 (0.88%) 
Faeces discoloured  1  2/113 (1.77%) 
Flatulence  1  1/113 (0.88%) 
Frequent bowel movements  1  1/113 (0.88%) 
Gastric ulcer  1  1/113 (0.88%) 
Gastritis  1  2/113 (1.77%) 
Gastrointestinal disorder  1  4/113 (3.54%) 
Gastrooesophageal reflux disease  1  4/113 (3.54%) 
Gingival bleeding  1  1/113 (0.88%) 
Haematochezia  1  2/113 (1.77%) 
Haemorrhoids  1  1/113 (0.88%) 
Ileus  1  1/113 (0.88%) 
Infrequent bowel movements  1  1/113 (0.88%) 
Lip blister  1  1/113 (0.88%) 
Lip ulceration  1  1/113 (0.88%) 
Mouth ulceration  1  1/113 (0.88%) 
Nausea  1  12/113 (10.62%) 
Oral disorder  1  1/113 (0.88%) 
Rectal fissure  1  1/113 (0.88%) 
Regurgitation  1  1/113 (0.88%) 
Retained deciduous tooth  1  1/113 (0.88%) 
Retching  1  1/113 (0.88%) 
Salivary hypersecretion  1  2/113 (1.77%) 
Sensitivity of teeth  1  1/113 (0.88%) 
Teething  1  4/113 (3.54%) 
Tongue spasm  1  1/113 (0.88%) 
Tooth deposit  1  1/113 (0.88%) 
Tooth impacted  1  1/113 (0.88%) 
Toothache  1  2/113 (1.77%) 
Vomiting  1  42/113 (37.17%) 
General disorders   
Abasia  1  1/113 (0.88%) 
Asthenia  1  4/113 (3.54%) 
Catheter site erythema  1  1/113 (0.88%) 
Catheter site extravasation  1  4/113 (3.54%) 
Catheter site haemorrhage  1  1/113 (0.88%) 
Catheter site inflammation  1  1/113 (0.88%) 
Catheter site oedema  1  1/113 (0.88%) 
Catheter site pain  1  3/113 (2.65%) 
Catheter site rash  1  1/113 (0.88%) 
Catheter site related reaction  1  1/113 (0.88%) 
Catheter site swelling  1  1/113 (0.88%) 
Chest discomfort  1  3/113 (2.65%) 
Chest pain  1  6/113 (5.31%) 
Chills  1  2/113 (1.77%) 
Crepitations  1  1/113 (0.88%) 
Device breakage  1  1/113 (0.88%) 
Device dislocation  1  4/113 (3.54%) 
Device issue  1  1/113 (0.88%) 
Device malfunction  1  9/113 (7.96%) 
Device occlusion  1  13/113 (11.50%) 
Discomfort  1  2/113 (1.77%) 
Disease progression  1  1/113 (0.88%) 
Energy increased  1  1/113 (0.88%) 
Extravasation  1  2/113 (1.77%) 
Fatigue  1  4/113 (3.54%) 
Feeling hot  1  1/113 (0.88%) 
Gait disturbance  1  1/113 (0.88%) 
Granuloma  1  2/113 (1.77%) 
Gravitational oedema  1  1/113 (0.88%) 
Influenza like illness  1  1/113 (0.88%) 
Infusion site extravasation  1  1/113 (0.88%) 
Infusion site swelling  1  2/113 (1.77%) 
Injection site extravasation  1  1/113 (0.88%) 
Injection site swelling  1  1/113 (0.88%) 
Malaise  1  2/113 (1.77%) 
Oedema  1  3/113 (2.65%) 
Oedema peripheral  1  2/113 (1.77%) 
Pain  1  3/113 (2.65%) 
Peripheral swelling  1  4/113 (3.54%) 
Pyrexia  1  61/113 (53.98%) 
Secretion discharge  1  6/113 (5.31%) 
Thirst  1  1/113 (0.88%) 
Thrombosis in device  1  1/113 (0.88%) 
Hepatobiliary disorders   
Cholelithiasis  1  1/113 (0.88%) 
Hepatic fibrosis  1  1/113 (0.88%) 
Immune system disorders   
Drug hypersensitivity  1  2/113 (1.77%) 
Food allergy  1  1/113 (0.88%) 
Hypersensitivity  1  5/113 (4.42%) 
Multiple allergies  1  1/113 (0.88%) 
Seasonal allergy  1  2/113 (1.77%) 
Infections and infestations   
Abscess limb  1  1/113 (0.88%) 
Acarodermatitis  1  1/113 (0.88%) 
Adenovirus infection  1  1/113 (0.88%) 
Atypical pneumonia  1  1/113 (0.88%) 
Bacterial tracheitis  1  5/113 (4.42%) 
Beta haemolytic streptococcal infection  1  1/113 (0.88%) 
Body tinea  1  3/113 (2.65%) 
Bronchiolitis  1  2/113 (1.77%) 
Bronchitis  1  8/113 (7.08%) 
Candida infection  1  1/113 (0.88%) 
Cellulitis  1  1/113 (0.88%) 
Chronic sinusitis  1  1/113 (0.88%) 
Clostridium difficile colitis  1  2/113 (1.77%) 
Conjunctivitis  1  7/113 (6.19%) 
Croup infectious  1  2/113 (1.77%) 
Device related infection  1  1/113 (0.88%) 
Ear infection  1  12/113 (10.62%) 
Ear lobe infection  1  1/113 (0.88%) 
Enterovirus infection  1  1/113 (0.88%) 
Erythema infectiosum  1  1/113 (0.88%) 
Escherichia urinary tract infection  1  1/113 (0.88%) 
Eye infection  1  2/113 (1.77%) 
Folliculitis  1  1/113 (0.88%) 
Fungal infection  1  2/113 (1.77%) 
Fungal skin infection  1  1/113 (0.88%) 
Furuncle  1  1/113 (0.88%) 
Gastroenteritis  1  11/113 (9.73%) 
Gastroenteritis norovirus  1  1/113 (0.88%) 
Gastroenteritis viral  1  4/113 (3.54%) 
Gastrointestinal infection  1  1/113 (0.88%) 
Gingival abscess  1  1/113 (0.88%) 
Gingivitis  1  1/113 (0.88%) 
Haemophilus infection  1  1/113 (0.88%) 
Hand-foot-and-mouth disease  1  1/113 (0.88%) 
Herpes zoster  1  1/113 (0.88%) 
Hordeolum  1  3/113 (2.65%) 
Impetigo  1  1/113 (0.88%) 
Infection  1  1/113 (0.88%) 
Influenza  1  8/113 (7.08%) 
Labyrinthitis  1  1/113 (0.88%) 
Lice infestation  1  1/113 (0.88%) 
Lip infection  1  1/113 (0.88%) 
Lobar pneumonia  1  1/113 (0.88%) 
Nail infection  1  1/113 (0.88%) 
Nasopharyngitis  1  12/113 (10.62%) 
Oral candidiasis  1  3/113 (2.65%) 
Oral herpes  1  1/113 (0.88%) 
Otitis externa  1  2/113 (1.77%) 
Otitis externa fungal  1  1/113 (0.88%) 
Otitis media  1  23/113 (20.35%) 
Otitis media acute  1  6/113 (5.31%) 
Otitis media chronic  1  3/113 (2.65%) 
Pharyngitis  1  7/113 (6.19%) 
Pharyngitis streptococcal  1  13/113 (11.50%) 
Pneumonia  1  17/113 (15.04%) 
Pneumonia viral  1  1/113 (0.88%) 
Pseudomonas infection  1  4/113 (3.54%) 
Rash pustular  1  1/113 (0.88%) 
Respiratory syncytial virus infection  1  3/113 (2.65%) 
Respiratory tract infection  1  5/113 (4.42%) 
Rhinitis  1  6/113 (5.31%) 
Rhinovirus infection  1  3/113 (2.65%) 
Rotavirus infection  1  1/113 (0.88%) 
Sinusitis  1  5/113 (4.42%) 
Skin infection  1  1/113 (0.88%) 
Staphylococcal infection  1  3/113 (2.65%) 
Stoma site infection  1  2/113 (1.77%) 
Streptococcal infection  1  2/113 (1.77%) 
Streptococcal urinary tract infection  1  1/113 (0.88%) 
Subcutaneous abscess  1  1/113 (0.88%) 
Tinea infection  1  2/113 (1.77%) 
Tonsillitis  1  2/113 (1.77%) 
Tooth abscess  1  2/113 (1.77%) 
Tooth infection  1  1/113 (0.88%) 
Tracheitis  1  7/113 (6.19%) 
Tracheobronchitis  1  1/113 (0.88%) 
Tracheostomy infection  1  3/113 (2.65%) 
Upper respiratory tract infection  1  49/113 (43.36%) 
Upper respiratory tract infection bacterial  1  1/113 (0.88%) 
Urinary tract infection  1  19/113 (16.81%) 
Viral infection  1  15/113 (13.27%) 
Viral rash  1  2/113 (1.77%) 
Viral upper respiratory tract infection  1  5/113 (4.42%) 
Vulvovaginal mycotic infection  1  1/113 (0.88%) 
Injury, poisoning and procedural complications   
Agitation postoperative  1  1/113 (0.88%) 
Animal bite  1  1/113 (0.88%) 
Animal scratch  1  1/113 (0.88%) 
Ankle fracture  1  1/113 (0.88%) 
Arthropod bite  1  7/113 (6.19%) 
Arthropod sting  1  1/113 (0.88%) 
Concussion  1  1/113 (0.88%) 
Contusion  1  6/113 (5.31%) 
Exposure to communicable disease  1  1/113 (0.88%) 
Eye contusion  1  1/113 (0.88%) 
Eye injury  1  1/113 (0.88%) 
Face injury  1  1/113 (0.88%) 
Fall  1  11/113 (9.73%) 
Feeding tube complication  1  2/113 (1.77%) 
Femur fracture  1  2/113 (1.77%) 
Foreign body  1  1/113 (0.88%) 
Gastrointestinal anastomotic leak  1  1/113 (0.88%) 
Gastrointestinal stoma complication  1  1/113 (0.88%) 
Hand fracture  1  1/113 (0.88%) 
Head injury  1  3/113 (2.65%) 
Humerus fracture  1  1/113 (0.88%) 
Joint dislocation  1  2/113 (1.77%) 
Laceration  1  6/113 (5.31%) 
Laryngeal injury  1  1/113 (0.88%) 
Ligament sprain  1  5/113 (4.42%) 
Limb injury  1  2/113 (1.77%) 
Lower limb fracture  1  1/113 (0.88%) 
Overdose  1  1/113 (0.88%) 
Periorbital haemorrhage  1  1/113 (0.88%) 
Post procedural haemorrhage  1  2/113 (1.77%) 
Postoperative fever  1  1/113 (0.88%) 
Postoperative respiratory distress  1  1/113 (0.88%) 
Procedural nausea  1  1/113 (0.88%) 
Procedural pain  1  8/113 (7.08%) 
Procedural vomiting  1  1/113 (0.88%) 
Scratch  1  2/113 (1.77%) 
Skin abrasion  1  9/113 (7.96%) 
Skin wound  1  1/113 (0.88%) 
Soft tissue injury  1  1/113 (0.88%) 
Splinter  1  1/113 (0.88%) 
Stoma site erythema  1  2/113 (1.77%) 
Stoma site haemorrhage  1  2/113 (1.77%) 
Stoma site irritation  1  3/113 (2.65%) 
Stoma site rash  1  1/113 (0.88%) 
Stoma site reaction  1  1/113 (0.88%) 
Sunburn  1  1/113 (0.88%) 
Thermal burn  1  2/113 (1.77%) 
Tibia fracture  1  1/113 (0.88%) 
Tooth avulsion  1  1/113 (0.88%) 
Tooth fracture  1  1/113 (0.88%) 
Tooth injury  1  1/113 (0.88%) 
Tracheostomy malfunction  1  2/113 (1.77%) 
Vaccination complication  1  2/113 (1.77%) 
Wound  1  1/113 (0.88%) 
Wound dehiscence  1  1/113 (0.88%) 
Investigations   
Alanine aminotransferase abnormal  1  1/113 (0.88%) 
Alanine aminotransferase increased  1  3/113 (2.65%) 
Aspartate aminotransferase abnormal  1  1/113 (0.88%) 
Aspartate aminotransferase increased  1  4/113 (3.54%) 
Atrial pressure increased  1  1/113 (0.88%) 
Blood alkaline phosphatase increased  1  1/113 (0.88%) 
Blood creatine phosphokinase MB increased  1  7/113 (6.19%) 
Blood creatine phosphokinase abnormal  1  1/113 (0.88%) 
Blood creatine phosphokinase increased  1  7/113 (6.19%) 
Blood culture positive  1  1/113 (0.88%) 
Blood lactate dehydrogenase abnormal  1  1/113 (0.88%) 
Blood lactate dehydrogenase increased  1  2/113 (1.77%) 
Blood potassium decreased  1  2/113 (1.77%) 
Blood pressure decreased  1  2/113 (1.77%) 
Blood pressure increased  1  7/113 (6.19%) 
Blood pressure systolic increased  1  1/113 (0.88%) 
Blood uric acid decreased  1  1/113 (0.88%) 
Blood uric acid increased  1  1/113 (0.88%) 
Blood zinc decreased  1  1/113 (0.88%) 
Body temperature increased  1  5/113 (4.42%) 
Brain natriuretic peptide increased  1  5/113 (4.42%) 
Breath sounds abnormal  1  2/113 (1.77%) 
Creatine urine increased  1  1/113 (0.88%) 
Creatinine urine increased  1  1/113 (0.88%) 
Electrocardiogram PR shortened  1  1/113 (0.88%) 
Electrocardiogram QT prolonged  1  1/113 (0.88%) 
Electrocardiogram ST segment elevation  1  1/113 (0.88%) 
Electrocardiogram abnormal  1  2/113 (1.77%) 
Eosinophil count increased  1  1/113 (0.88%) 
Gamma-glutamyltransferase increased  1  1/113 (0.88%) 
Haematocrit decreased  1  1/113 (0.88%) 
Haemoglobin decreased  1  3/113 (2.65%) 
Heart rate increased  1  5/113 (4.42%) 
Liver function test abnormal  1  1/113 (0.88%) 
Neutrophil count abnormal  1  1/113 (0.88%) 
Neutrophil count increased  1  1/113 (0.88%) 
Neutrophil percentage increased  1  1/113 (0.88%) 
Occult blood positive  1  1/113 (0.88%) 
Oxygen saturation decreased  1  4/113 (3.54%) 
Platelet count increased  1  2/113 (1.77%) 
Protein urine  1  1/113 (0.88%) 
Protein urine present  1  1/113 (0.88%) 
Pulmonary function test decreased  1  2/113 (1.77%) 
Pulse pressure abnormal  1  1/113 (0.88%) 
Respiratory rate increased  1  1/113 (0.88%) 
Serum ferritin decreased  1  1/113 (0.88%) 
Specific gravity urine  1  1/113 (0.88%) 
Sputum culture positive  1  1/113 (0.88%) 
Staphylococcus test positive  1  1/113 (0.88%) 
Tuberculin test positive  1  1/113 (0.88%) 
Urine ketone body present  1  1/113 (0.88%) 
Urine output decreased  1  2/113 (1.77%) 
Weight decreased  1  2/113 (1.77%) 
Weight increased  1  3/113 (2.65%) 
White blood cell count abnormal  1  1/113 (0.88%) 
White blood cell count increased  1  1/113 (0.88%) 
Metabolism and nutrition disorders   
Abnormal weight gain  1  1/113 (0.88%) 
Decreased appetite  1  4/113 (3.54%) 
Dehydration  1  5/113 (4.42%) 
Electrolyte imbalance  1  1/113 (0.88%) 
Failure to thrive  1  1/113 (0.88%) 
Hypoalbuminaemia  1  1/113 (0.88%) 
Hypochloraemia  1  1/113 (0.88%) 
Hypoglycaemia  1  3/113 (2.65%) 
Hypokalaemia  1  2/113 (1.77%) 
Iron deficiency  1  1/113 (0.88%) 
Vitamin D deficiency  1  4/113 (3.54%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  5/113 (4.42%) 
Arthropathy  1  1/113 (0.88%) 
Axillary mass  1  1/113 (0.88%) 
Back pain  1  2/113 (1.77%) 
Deformity thorax  1  1/113 (0.88%) 
Foot deformity  1  2/113 (1.77%) 
Fracture pain  1  1/113 (0.88%) 
Hip deformity  1  1/113 (0.88%) 
Joint contracture  1  2/113 (1.77%) 
Joint stiffness  1  1/113 (0.88%) 
Joint swelling  1  3/113 (2.65%) 
Kyphosis  1  2/113 (1.77%) 
Limb discomfort  1  2/113 (1.77%) 
Muscle contracture  1  1/113 (0.88%) 
Muscle spasms  1  3/113 (2.65%) 
Muscle tightness  1  2/113 (1.77%) 
Muscular weakness  1  12/113 (10.62%) 
Musculoskeletal chest pain  1  2/113 (1.77%) 
Musculoskeletal pain  1  4/113 (3.54%) 
Musculoskeletal stiffness  1  1/113 (0.88%) 
Myalgia  1  1/113 (0.88%) 
Myopathy  1  2/113 (1.77%) 
Neck pain  1  3/113 (2.65%) 
Osteopenia  1  4/113 (3.54%) 
Pain in extremity  1  16/113 (14.16%) 
Pain in jaw  1  1/113 (0.88%) 
Scoliosis  1  6/113 (5.31%) 
Tendinous contracture  1  2/113 (1.77%) 
Tendon discomfort  1  3/113 (2.65%) 
Tendon disorder  1  1/113 (0.88%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Cholesteatoma  1  1/113 (0.88%) 
Nervous system disorders   
Allodynia  1  1/113 (0.88%) 
Areflexia  1  2/113 (1.77%) 
Burning sensation  1  1/113 (0.88%) 
Clumsiness  1  1/113 (0.88%) 
Convulsion  1  2/113 (1.77%) 
Dizziness  1  3/113 (2.65%) 
Facial paresis  1  1/113 (0.88%) 
Formication  1  1/113 (0.88%) 
Gross motor delay  1  2/113 (1.77%) 
Headache  1  14/113 (12.39%) 
Hypoaesthesia  1  1/113 (0.88%) 
Hyporeflexia  1  2/113 (1.77%) 
Hypotonia  1  2/113 (1.77%) 
Lethargy  1  4/113 (3.54%) 
Motor developmental delay  1  1/113 (0.88%) 
Motor dysfunction  1  2/113 (1.77%) 
Muscle contractions involuntary  1  1/113 (0.88%) 
Nystagmus  1  1/113 (0.88%) 
Paraesthesia  1  1/113 (0.88%) 
Paralysis flaccid  1  1/113 (0.88%) 
Poor quality sleep  1  1/113 (0.88%) 
Presyncope  1  1/113 (0.88%) 
Psychomotor hyperactivity  1  1/113 (0.88%) 
Somnolence  1  1/113 (0.88%) 
Speech disorder  1  1/113 (0.88%) 
Tremor  1  2/113 (1.77%) 
Psychiatric disorders   
Aggression  1  1/113 (0.88%) 
Agitation  1  4/113 (3.54%) 
Anxiety  1  5/113 (4.42%) 
Attention deficit/hyperactivity disorder  1  2/113 (1.77%) 
Bruxism  1  1/113 (0.88%) 
Insomnia  1  3/113 (2.65%) 
Irritability  1  1/113 (0.88%) 
Mental disorder  1  1/113 (0.88%) 
Mood swings  1  1/113 (0.88%) 
Oppositional defiant disorder  1  1/113 (0.88%) 
Sleep disorder  1  2/113 (1.77%) 
Renal and urinary disorders   
Dysuria  1  2/113 (1.77%) 
Haematuria  1  1/113 (0.88%) 
Incontinence  1  1/113 (0.88%) 
Nephrolithiasis  1  1/113 (0.88%) 
Pollakiuria  1  2/113 (1.77%) 
Proteinuria  1  1/113 (0.88%) 
Urinary hesitation  1  1/113 (0.88%) 
Urinary incontinence  1  4/113 (3.54%) 
Urinary retention  1  1/113 (0.88%) 
Reproductive system and breast disorders   
Genital discomfort  1  2/113 (1.77%) 
Vaginal prolapse  1  1/113 (0.88%) 
Vulvovaginal disorder  1  1/113 (0.88%) 
Vulvovaginal erythema  1  2/113 (1.77%) 
Vulvovaginal pruritus  1  1/113 (0.88%) 
Respiratory, thoracic and mediastinal disorders   
Adenoidal hypertrophy  1  1/113 (0.88%) 
Aspiration  1  2/113 (1.77%) 
Atelectasis  1  5/113 (4.42%) 
Bronchial hyperreactivity  1  3/113 (2.65%) 
Bronchial secretion retention  1  3/113 (2.65%) 
Bronchial wall thickening  1  1/113 (0.88%) 
Bronchomalacia  1  1/113 (0.88%) 
Bronchospasm  1  1/113 (0.88%) 
Choking  1  3/113 (2.65%) 
Cough  1  36/113 (31.86%) 
Dysphonia  1  1/113 (0.88%) 
Dyspnoea  1  6/113 (5.31%) 
Epistaxis  1  5/113 (4.42%) 
Haemoptysis  1  3/113 (2.65%) 
Hypercapnia  1  1/113 (0.88%) 
Hypoventilation  1  2/113 (1.77%) 
Hypoxia  1  1/113 (0.88%) 
Increased bronchial secretion  1  5/113 (4.42%) 
Increased upper airway secretion  1  7/113 (6.19%) 
Increased viscosity of bronchial secretion  1  3/113 (2.65%) 
Nasal congestion  1  16/113 (14.16%) 
Nasal discharge discolouration  1  1/113 (0.88%) 
Nasal dryness  1  1/113 (0.88%) 
Oropharyngeal pain  1  13/113 (11.50%) 
Pleural effusion  1  1/113 (0.88%) 
Pneumonia aspiration  1  3/113 (2.65%) 
Productive cough  1  2/113 (1.77%) 
Pulmonary artery dilatation  1  1/113 (0.88%) 
Pulmonary congestion  1  1/113 (0.88%) 
Pulmonary hypertension  1  1/113 (0.88%) 
Respiratory disorder  1  1/113 (0.88%) 
Respiratory distress  1  8/113 (7.08%) 
Respiratory failure  1  1/113 (0.88%) 
Respiratory muscle weakness  1  3/113 (2.65%) 
Respiratory tract congestion  1  7/113 (6.19%) 
Respiratory tract haemorrhage  1  2/113 (1.77%) 
Rhinitis allergic  1  2/113 (1.77%) 
Rhinorrhoea  1  24/113 (21.24%) 
Rhonchi  1  2/113 (1.77%) 
Sinus congestion  1  2/113 (1.77%) 
Sleep apnoea syndrome  1  4/113 (3.54%) 
Sneezing  1  2/113 (1.77%) 
Snoring  1  1/113 (0.88%) 
Sputum discoloured  1  1/113 (0.88%) 
Sputum increased  1  1/113 (0.88%) 
Tachypnoea  1  1/113 (0.88%) 
Throat irritation  1  3/113 (2.65%) 
Throat tightness  1  1/113 (0.88%) 
Tonsillar hypertrophy  1  2/113 (1.77%) 
Tracheal ulcer  1  1/113 (0.88%) 
Upper respiratory tract congestion  1  8/113 (7.08%) 
Upper-airway cough syndrome  1  1/113 (0.88%) 
Use of accessory respiratory muscles  1  1/113 (0.88%) 
Wheezing  1  5/113 (4.42%) 
Skin and subcutaneous tissue disorders   
Acne  1  3/113 (2.65%) 
Alopecia  1  2/113 (1.77%) 
Blister  1  1/113 (0.88%) 
Cold sweat  1  1/113 (0.88%) 
Decubitus ulcer  1  3/113 (2.65%) 
Dermatitis  1  3/113 (2.65%) 
Dermatitis allergic  1  1/113 (0.88%) 
Dermatitis contact  1  2/113 (1.77%) 
Dermatitis diaper  1  4/113 (3.54%) 
Dry skin  1  2/113 (1.77%) 
Ecchymosis  1  1/113 (0.88%) 
Eczema  1  2/113 (1.77%) 
Erythema  1  5/113 (4.42%) 
Excessive granulation tissue  1  4/113 (3.54%) 
Granuloma skin  1  1/113 (0.88%) 
Hyperhidrosis  1  1/113 (0.88%) 
Hyperkeratosis  1  1/113 (0.88%) 
Ingrowing nail  1  1/113 (0.88%) 
Ingrown hair  1  1/113 (0.88%) 
Macule  1  2/113 (1.77%) 
Mechanical urticaria  1  1/113 (0.88%) 
Papule  1  1/113 (0.88%) 
Petechiae  1  1/113 (0.88%) 
Photosensitivity reaction  1  1/113 (0.88%) 
Pruritus  1  5/113 (4.42%) 
Pruritus generalised  1  1/113 (0.88%) 
Rash  1  31/113 (27.43%) 
Rash erythematous  1  2/113 (1.77%) 
Rash macular  1  1/113 (0.88%) 
Rash pruritic  1  1/113 (0.88%) 
Red man syndrome  1  1/113 (0.88%) 
Seborrhoeic dermatitis  1  1/113 (0.88%) 
Skin disorder  1  2/113 (1.77%) 
Skin exfoliation  1  2/113 (1.77%) 
Skin hyperpigmentation  1  1/113 (0.88%) 
Skin irritation  1  4/113 (3.54%) 
Skin lesion  1  2/113 (1.77%) 
Skin ulcer  1  2/113 (1.77%) 
Swelling face  1  1/113 (0.88%) 
Urticaria  1  9/113 (7.96%) 
Surgical and medical procedures   
Central venous catheterisation  1  1/113 (0.88%) 
Ear tube removal  1  1/113 (0.88%) 
Gastrostomy tube removal  1  1/113 (0.88%) 
Vascular disorders   
Aortic dilatation  1  1/113 (0.88%) 
Flushing  1  7/113 (6.19%) 
Haematoma  1  2/113 (1.77%) 
Hypertension  1  5/113 (4.42%) 
Hypotension  1  2/113 (1.77%) 
Pallor  1  1/113 (0.88%) 
Peripheral coldness  1  1/113 (0.88%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17.1
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.
If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
Results Point of Contact
Name/Title: Trial Transparency Team
Organization: Sanofi
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01526785     History of Changes
Other Study ID Numbers: AGLU09411
EFC12720 ( Other Identifier: Sanofi )
First Submitted: February 2, 2012
First Posted: February 6, 2012
Results First Submitted: September 29, 2015
Results First Posted: October 27, 2015
Last Update Posted: December 8, 2015