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Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

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ClinicalTrials.gov Identifier: NCT01205152
Recruitment Status : Completed
First Posted : September 20, 2010
Results First Posted : November 17, 2017
Last Update Posted : March 13, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hypophosphatasia
Intervention Biological: asfotase alfa
Enrollment 10
Recruitment Details The main criteria for inclusion in Study ENB-002-08 were male and female patients </= 36 months of age, with severe infantile-onset HPP (symptom onset before 6 months of age) who were medically stable (ventilator support was allowed). To enter extension Study ENB-003-08, parent/guardian had to consent and patient had to complete Study ENB-002-08.
Pre-assignment Details  
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
Period Title: Overall Study
Started 10
Completed 9
Not Completed 1
Reason Not Completed
Death             1
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
Of the 11 patients who were enrolled in Study ENB-002-08, 10 patients completed Study ENB-002-08 and were enrolled in extension study ENB-003-08.
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Weeks
Number Analyzed 10 participants
56.44  (61.888)
[1]
Measure Description: Mean (SD) age at time of first dose in ENB-002-08 (NCT00744042)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
6
  60.0%
Male
4
  40.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Hispanic or Latino
1
  10.0%
Not Hispanic or Latino
9
  90.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
10
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
1.Primary Outcome
Title Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa
Hide Description Outcome measure is the number of patients with 1 or more treatment-emergent adverse event. The time period is from Baseline in the ENB-003-08 study to the end of the ENB-003-08 study.
Time Frame 84 months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (All 10 patients enrolled in Study ENB-003-08)
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description:
An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
Overall Number of Participants Analyzed 10
Measure Type: Count of Participants
Unit of Measure: Participants
10
 100.0%
2.Primary Outcome
Title Long-term Efficacy of Asfotase Alfa in Treating Rickets in Infants and Young Children With Hypophosphatasia (HPP).
Hide Description

Outcome measure is the evaluation of radiographic change in rickets severity using a qualitative Radiographic Global Impression of Change (RGI-C) Scale. Skeletal radiographs obtained at the patient's last assessment were compared with skeletal radiographs obtained before initiation of treatment (Baseline in Study ENB-002-08 [NCT00744042]). The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP-associated rickets) to +3 (indicative of complete or near complete healing of HPP-associated rickets).

The time period is pre-dose (Baseline from ENB-002-08 study) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies.

Time Frame Up to 90 Months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (All 10 patients enrolled in Study ENB-003-08)
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description:
An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
Overall Number of Participants Analyzed 10
Median (Full Range)
Unit of Measure: scores on a scale
2.00
(2.00 to 3.00)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Asfotase Alfa
Comments Change is relative to Baseline in Study ENB-002-08 (NCT00744042). The RGI-C score represents evaluation of skeletal X-rays at each post-treatment study timepoint compared with pre-treatment X-rays from Study ENB-002-08 using an ordinal scale. Therefore, an RGI-C score is not applicable for radiographs obtained at Baseline.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0020
Comments Two-sided with p-value threshold <0.05 for statistical significance.
Method Wilcoxon signed-rank test
Comments [Not Specified]
3.Secondary Outcome
Title Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Plasma Inorganic Pyrophosphate (PPi) Levels
Hide Description Outcome measure is the change from Baseline in plasma inorganic pyrophosphate (PPi) levels. The time period is pre-dose (Baseline from the ENB-002-08 study [NCT00744042]) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies.
Time Frame Up to 90 Months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (All 10 patients enrolled in Study ENB-003-08). Change from Baseline could not be calculated for 3 patients due to non-evaluable samples at Baseline.
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description:
An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
Overall Number of Participants Analyzed 7
Median (Full Range)
Unit of Measure: uM
-2.460
(-9.73 to 2.72)
4.Secondary Outcome
Title Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Pyridoxal-5-phosphate (PLP) Levels
Hide Description Outcome measure is the change from Baseline in pyridoxal-5-phosphate (PLP) levels. The time period is pre-dose (Baseline from the ENB-002-08 study [NCT00744042]) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies.
Time Frame Up to 90 Months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (All 10 patients enrolled in Study ENB-003-08). Change from Baseline could not be calculated for 2 patients because of non-evaluable samples at Baseline.
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description:
An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
Overall Number of Participants Analyzed 8
Median (Full Range)
Unit of Measure: ng/mL
-266.200
(-844.30 to 184.00)
5.Secondary Outcome
Title Effect of SC Asfotase Alfa on Growth: Weight Z-scores
Hide Description Outcome measure is the change from Baseline in Z-scores for weight. The time period is pre-dose (Baseline from the ENB-002-08 study [NCT00744042]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies.
Time Frame Up to 90 Months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (All 10 patients enrolled in Study ENB-003-08)
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description:
An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
Overall Number of Participants Analyzed 10
Median (Full Range)
Unit of Measure: Z-score
2.43
(-2.9 to 5.2)
6.Secondary Outcome
Title Effect of SC Asfotase Alfa on Growth: Height/Length Z-scores
Hide Description Outcome measure is the change from Baseline in Z-scores for height/length. The time period is pre-dose (Baseline from the ENB-002-08 study [NCT00744042]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies.
Time Frame Up to 90 Months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (All 10 patients enrolled in Study ENB-003-08)
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description:
An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
Overall Number of Participants Analyzed 10
Median (Full Range)
Unit of Measure: Z-score
1.93
(-3.2 to 4.6)
7.Secondary Outcome
Title Effect of SC Asfotase Alfa on Respiratory Function
Hide Description Outcome measure is the shift in the proportion of patients requiring respiratory support at their last assessment in Study ENB-003-08 compared with Baseline. The time period is pre-dose (Baseline from the ENB-002-08 study [NCT00744042]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies.
Time Frame Up to 90 Months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (All 10 patients enrolled in Study ENB-003-08)
Arm/Group Title Asfotase Alfa (Baseline ENB-002-08) Asfotase Alfa (Last Assessment)
Hide Arm/Group Description:
Baseline results prior to treatment with asfotase alfa
Results at the last assessment in the ENB-003-08 study.
Overall Number of Participants Analyzed 11 10
Measure Type: Count of Participants
Unit of Measure: Participants
No respiratory support
6
  54.5%
9
  90.0%
Supplemental oxygen
0
   0.0%
0
   0.0%
Continuous positive airway pressure
1
   9.1%
0
   0.0%
Mechanical ventilation
3
  27.3%
1
  10.0%
Biphasic positive airway pressure
0
   0.0%
0
   0.0%
Other
1
   9.1%
0
   0.0%
Time Frame Adverse events were recorded from the time the parents/legal guardians signed the ICF for study ENB-003-08 through completion of the patient's participation in the study ENB-003-08.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Asfotase Alfa
Hide Arm/Group Description An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
All-Cause Mortality
Asfotase Alfa
Affected / at Risk (%)
Total   1/10 (10.00%)    
Hide Serious Adverse Events
Asfotase Alfa
Affected / at Risk (%) # Events
Total   9/10 (90.00%)    
Cardiac disorders   
Cyanosis  1  1/10 (10.00%)  1
Congenital, familial and genetic disorders   
Congenital bowing of long bones  1  1/10 (10.00%)  1
Craniosynostosis  1  4/10 (40.00%)  5
Talipes  1  1/10 (10.00%)  1
Ear and labyrinth disorders   
Conductive deafness  1  1/10 (10.00%)  1
Eye disorders   
Papilloedema  1  1/10 (10.00%)  1
General disorders   
Immediate post-injection reaction  1  1/10 (10.00%)  1
Medical device complication  1  1/10 (10.00%)  1
Hepatobiliary disorders   
Chronic hepatitis  1  1/10 (10.00%)  1
Infections and infestations   
Bacterial tracheitis  1  1/10 (10.00%)  1
Croup infectious  1  1/10 (10.00%)  1
Pneumonia  1  3/10 (30.00%)  3
Respiratory syncytial virus bronchiolitis  1  1/10 (10.00%)  1
Septic shock  1  1/10 (10.00%)  1
Tracheitis  1  1/10 (10.00%)  1
Injury, poisoning and procedural complications   
Femur fracture  1  1/10 (10.00%)  2
Stress fracture  1  1/10 (10.00%)  1
Investigations   
Blood urea increased  1  1/10 (10.00%)  1
CSF pressure  1  1/10 (10.00%)  1
Investigation  1  1/10 (10.00%)  2
Metabolism and nutrition disorders   
Feeding disorder of infancy or early childhood  1  1/10 (10.00%)  1
Weight gain poor  1  1/10 (10.00%)  1
Musculoskeletal and connective tissue disorders   
Scoliosis  1  1/10 (10.00%)  2
Nervous system disorders   
Intracranial pressure increased  1  1/10 (10.00%)  1
Renal and urinary disorders   
Nephrolithiasis  1  1/10 (10.00%)  1
Urinary tract obstruction  1  1/10 (10.00%)  1
Respiratory, thoracic and mediastinal disorders   
Adenoidal disorder  1  1/10 (10.00%)  1
Apnoeic attack  1  1/10 (10.00%)  1
Asthma  1  1/10 (10.00%)  1
Respiratory failure  1  1/10 (10.00%)  2
Restrictive pulmonary disease  1  1/10 (10.00%)  1
Sleep apnoea syndrome  1  1/10 (10.00%)  1
Tonsillar disorder  1  1/10 (10.00%)  1
Skin and subcutaneous tissue disorders   
Urticaria  1  1/10 (10.00%)  1
Surgical and medical procedures   
Central venous catheter removal  1  1/10 (10.00%)  1
Tracheal fistula repair  1  1/10 (10.00%)  1
Tracheostomy tube removal  1  2/10 (20.00%)  2
Vascular disorders   
Deep vein thrombosis  1  1/10 (10.00%)  1
1
Term from vocabulary, MedDRA version 13.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Asfotase Alfa
Affected / at Risk (%) # Events
Total   10/10 (100.00%)    
Blood and lymphatic system disorders   
Anaemia  1  1/10 (10.00%)  1
Leukopenia  1  1/10 (10.00%)  1
Microcytosis  1  1/10 (10.00%)  1
Neutropenia  1  1/10 (10.00%)  1
Congenital, familial and genetic disorders   
Craniosynostosis  1  2/10 (20.00%)  5
Ear and labyrinth disorders   
Deafness  1  1/10 (10.00%)  1
Eustachian tube dysfunction  1  1/10 (10.00%)  1
Middle ear disorder  1  1/10 (10.00%)  1
Otorrhoea  1  1/10 (10.00%)  1
Tympanic membrane perforation  1  1/10 (10.00%)  1
Vertigo  1  1/10 (10.00%)  1
Endocrine disorders   
Precocious puberty  1  1/10 (10.00%)  1
Eye disorders   
Astigmatism  1  1/10 (10.00%)  1
Conjunctival deposit  1  1/10 (10.00%)  2
Conjunctival hyperaemia  1  1/10 (10.00%)  1
Conjunctivitis  1  2/10 (20.00%)  3
Eye pain  1  1/10 (10.00%)  2
Lacrimation increased  1  1/10 (10.00%)  1
Myopia  1  1/10 (10.00%)  1
Optic disc drusen  1  1/10 (10.00%)  1
Optic nerve disorder  1  1/10 (10.00%)  1
Papilloedema  1  2/10 (20.00%)  2
Visual impairment  1  2/10 (20.00%)  4
Gastrointestinal disorders   
Abdominal discomfort  1  1/10 (10.00%)  1
Abdominal pain  1  1/10 (10.00%)  9
Constipation  1  2/10 (20.00%)  4
Dental caries  1  4/10 (40.00%)  5
Diarrhoea  1  3/10 (30.00%)  5
Dysphagia  1  1/10 (10.00%)  1
Gastritis  1  1/10 (10.00%)  1
Gingival swelling  1  1/10 (10.00%)  2
Gingivitis  1  2/10 (20.00%)  2
Ileus  1  1/10 (10.00%)  1
Impaired gastric emptying  1  2/10 (20.00%)  2
Mouth ulceration  1  1/10 (10.00%)  1
Nausea  1  2/10 (20.00%)  2
Stomatitis  1  1/10 (10.00%)  1
Teething  1  1/10 (10.00%)  1
Tooth loss  1  4/10 (40.00%)  8
Toothache  1  1/10 (10.00%)  1
Vomiting  1  5/10 (50.00%)  14
General disorders   
Catheter site rash  1  1/10 (10.00%)  1
Injection site atrophy  1  1/10 (10.00%)  2
Injection site calcification  1  1/10 (10.00%)  2
Injection site discolouration  1  1/10 (10.00%)  1
Injection site erythema  1  4/10 (40.00%)  18
Injection site haematoma  1  2/10 (20.00%)  2
Injection site hypertrophy  1  2/10 (20.00%)  8
Injection site inflammation  1  1/10 (10.00%)  2
Injection site nodule  1  1/10 (10.00%)  1
Injection site reaction  1  2/10 (20.00%)  3
Injection site swelling  1  2/10 (20.00%)  5
Injection site warmth  1  1/10 (10.00%)  1
Pain  1  3/10 (30.00%)  4
Pyrexia  1  7/10 (70.00%)  21
Vaccination site inflammation  1  1/10 (10.00%)  1
Hepatobiliary disorders   
Cholelithiasis  1  1/10 (10.00%)  1
Immune system disorders   
Drug hypersensitivity  1  2/10 (20.00%)  2
Immunisation reaction  1  1/10 (10.00%)  1
Seasonal allergy  1  1/10 (10.00%)  1
Infections and infestations   
Abscess jaw  1  1/10 (10.00%)  2
Acute sinusitis  1  3/10 (30.00%)  4
Bronchitis  1  2/10 (20.00%)  2
Cellulitis  1  1/10 (10.00%)  1
Conjunctivitis infective  1  1/10 (10.00%)  1
Croup infectious  1  1/10 (10.00%)  3
Cystitis  1  1/10 (10.00%)  1
Gastroenteritis  1  3/10 (30.00%)  9
Gastroenteritis viral  1  1/10 (10.00%)  2
H1N1 influenza  1  1/10 (10.00%)  1
Hordeolum  1  1/10 (10.00%)  1
Influenza  1  3/10 (30.00%)  7
Injection site cellulitis  1  1/10 (10.00%)  3
Laryngitis  1  1/10 (10.00%)  1
Lice infestation  1  1/10 (10.00%)  1
Lower respiratory tract infection  1  2/10 (20.00%)  2
Molluscum contagiosum  1  1/10 (10.00%)  2
Nasopharyngitis  1  2/10 (20.00%)  2
Otitis externa  1  2/10 (20.00%)  2
Otitis media  1  6/10 (60.00%)  23
Otitis media acute  1  1/10 (10.00%)  1
Otitis media chronic  1  2/10 (20.00%)  2
Pharyngitis  1  3/10 (30.00%)  8
Pneumonia  1  5/10 (50.00%)  7
Pneumonia bacterial  1  1/10 (10.00%)  1
Pneumonia primary atypical  1  1/10 (10.00%)  1
Rhinitis  1  2/10 (20.00%)  5
Sinusitis  1  2/10 (20.00%)  8
Skin infection  1  1/10 (10.00%)  1
Tonsillitis  1  2/10 (20.00%)  6
Tooth abscess  1  2/10 (20.00%)  2
Tracheitis  1  2/10 (20.00%)  5
Upper respiratory tract infection  1  8/10 (80.00%)  77
Varicella  1  2/10 (20.00%)  2
Viral infection  1  3/10 (30.00%)  3
Viral pharyngitis  1  1/10 (10.00%)  1
Viral upper respiratory tract infection  1  2/10 (20.00%)  5
Injury, poisoning and procedural complications   
Adverse event following immunisation  1  1/10 (10.00%)  1
Arthropod sting  1  1/10 (10.00%)  1
Clavicle fracture  1  1/10 (10.00%)  2
Contusion  1  2/10 (20.00%)  2
Face injury  1  1/10 (10.00%)  1
Foreign body  1  1/10 (10.00%)  1
Hand fracture  1  2/10 (20.00%)  2
Head injury  1  1/10 (10.00%)  1
Joint sprain  1  1/10 (10.00%)  2
Limb injury  1  1/10 (10.00%)  1
Lower limb fracture  1  1/10 (10.00%)  2
Mouth injury  1  1/10 (10.00%)  1
Periorbital haematoma  1  1/10 (10.00%)  1
Procedural complication  1  1/10 (10.00%)  1
Procedural pain  1  2/10 (20.00%)  2
Procedural site reaction  1  1/10 (10.00%)  2
Rib fracture  1  2/10 (20.00%)  2
Scapula fracture  1  1/10 (10.00%)  1
Skin laceration  1  2/10 (20.00%)  2
Tibia fracture  1  2/10 (20.00%)  2
Tracheal haemorrhage  1  1/10 (10.00%)  1
Upper limb fracture  1  1/10 (10.00%)  2
Investigations   
Alanine aminotransferase increased  1  1/10 (10.00%)  1
Blood alkaline phosphatase abnormal  1  1/10 (10.00%)  1
Oxygen saturation decreased  1  2/10 (20.00%)  3
Staphylococcus test positive  1  1/10 (10.00%)  1
Urine calcium/creatinine ratio increased  1  2/10 (20.00%)  3
Vitamin D decreased  1  1/10 (10.00%)  2
Vitamin D increased  1  1/10 (10.00%)  1
Metabolism and nutrition disorders   
Abnormal weight gain  1  1/10 (10.00%)  1
Fluid overload  1  1/10 (10.00%)  1
Hypophosphataemic rickets  1  1/10 (10.00%)  1
Weight gain poor  1  1/10 (10.00%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/10 (10.00%)  2
Back pain  1  1/10 (10.00%)  2
Foot deformity  1  1/10 (10.00%)  1
Mobility decreased  1  1/10 (10.00%)  1
Osteopenia  1  1/10 (10.00%)  1
Pain in extremity  1  4/10 (40.00%)  19
Scoliosis  1  2/10 (20.00%)  2
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Melanocytic naevus  1  1/10 (10.00%)  1
Skin papilloma  1  1/10 (10.00%)  1
Nervous system disorders   
Headache  1  5/10 (50.00%)  14
Speech disorder developmental  1  2/10 (20.00%)  2
Psychiatric disorders   
Abnormal behaviour  1  1/10 (10.00%)  1
Agitation  1  1/10 (10.00%)  1
Anticipatory anxiety  1  1/10 (10.00%)  1
Anxiety  1  1/10 (10.00%)  1
Fear of needles  1  1/10 (10.00%)  1
Sleep disorder  1  1/10 (10.00%)  1
Renal and urinary disorders   
Dysuria  1  1/10 (10.00%)  2
Haematuria  1  1/10 (10.00%)  1
Hydronephrosis  1  2/10 (20.00%)  3
Nephrocalcinosis  1  2/10 (20.00%)  2
Renal cyst  1  1/10 (10.00%)  2
Renal pain  1  1/10 (10.00%)  2
Nephrolithiasis  1  2/10 (20.00%)  2
Reproductive system and breast disorders   
Breast enlargement  1  1/10 (10.00%)  1
Respiratory, thoracic and mediastinal disorders   
Adenoidal disorder  1  1/10 (10.00%)  1
Bronchial hyperreactivity  1  1/10 (10.00%)  1
Cough  1  2/10 (20.00%)  6
Epistaxis  1  1/10 (10.00%)  4
Increased upper airway secretion  1  1/10 (10.00%)  2
Nasal congestion  1  1/10 (10.00%)  1
Nasal polyps  1  1/10 (10.00%)  1
Oropharyngeal pain  1  1/10 (10.00%)  1
Rales  1  1/10 (10.00%)  1
Respiratory distress  1  1/10 (10.00%)  1
Rhinitis allergic  1  3/10 (30.00%)  4
Rhinorrhoea  1  1/10 (10.00%)  4
Sinus congestion  1  1/10 (10.00%)  1
Sleep apnea syndrome  1  2/10 (20.00%)  2
Tonsillar disorder  1  1/10 (10.00%)  1
Tonsillar hypertrophy  1  1/10 (10.00%)  2
Wheezing  1  3/10 (30.00%)  5
Skin and subcutaneous tissue disorders   
Acne  1  1/10 (10.00%)  1
Alopecia  1  1/10 (10.00%)  1
Dermatitis allergic  1  1/10 (10.00%)  1
Dry skin  1  1/10 (10.00%)  1
Excessive granulation tissue  1  1/10 (10.00%)  1
Lipohypertrophy  1  1/10 (10.00%)  1
Rash  1  4/10 (40.00%)  5
Rash papular  1  1/10 (10.00%)  3
Skin discolouration  1  1/10 (10.00%)  2
Skin irritation  1  2/10 (20.00%)  2
Urticaria  1  1/10 (10.00%)  1
Urticaria contact  1  1/10 (10.00%)  1
1
Term from vocabulary, MedDRA (13.0)
Indicates events were collected by systematic assessment
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Director of Clinical Trials
Organization: Alexion Pharmaceuticals, Inc.
Phone: 475-230-2596
EMail: ClinicalTrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01205152    
Other Study ID Numbers: ENB-003-08
FD-R-003745-03 ( Other Grant/Funding Number: Office of Orphan Product Development )
First Submitted: September 17, 2010
First Posted: September 20, 2010
Results First Submitted: August 14, 2017
Results First Posted: November 17, 2017
Last Update Posted: March 13, 2019