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SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa (VITAL)

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ClinicalTrials.gov Identifier: NCT01371825
Recruitment Status : Active, not recruiting
First Posted : June 13, 2011
Results First Posted : April 18, 2016
Last Update Posted : November 14, 2017
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Lysosomal Acid Lipase Deficiency
Wolman Disease
Intervention Drug: Sebelipase alfa (SBC-102)
Enrollment 9

Recruitment Details A total of 12 Principal Investigators at 12 centers participated in this study, including 9 primary centers in the United Kingdom (UK), United States (US), France, Turkey, Saudia Arabia, Taiwan, Italy, and Egypt.
Pre-assignment Details To assess eligibility, subjects were screened for a period of up to 3 weeks prior to enrollment in the study. A total of 11 subjects were screened; 2 subjects died during screening. The other 9 subjects, all of whom were ≤ 8 months of age on the date of enrollment, met all eligibility criteria and were enrolled, treated and analysed.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Period Title: Primary Efficacy Analysis Period
Started 9
Completed 6
Not Completed 3
Reason Not Completed
Death             3
Period Title: Long-term Treatment Period
Started 6
Completed 6 [1]
Not Completed 0
[1]
This period is currently ongoing.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
<=18 years
9
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Months
Number Analyzed 9 participants
3
(1.1 to 5.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
4
  44.4%
Male
5
  55.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
6
  66.7%
Unknown or Not Reported
3
  33.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
  11.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
  11.1%
White
4
  44.4%
More than one race
0
   0.0%
Unknown or Not Reported
3
  33.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 9 participants
Saudi Arabia 0
Turkey 1
United States 1
Ireland 1
Taiwan 0
Egypt 1
United Kingdom 2
Italy 0
France 3
Germany 0
India 0
1.Primary Outcome
Title Percentage of Subjects in the PES Surviving to 12 Months of Age
Hide Description The primary efficacy endpoint was the percentage of subjects (%) in the Primary Efficacy Analysis Set (PES) who survived to at least 12 months of age.
Time Frame From week 0 to data cut-off (27 to 164 weeks of treatment)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Evaluable subjects in the Primary Efficacy Analysis Set (PES), which included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa. All 9 subjects were evaluable.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 9
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: % of subjects
67
(29.93 to 92.51)
2.Secondary Outcome
Title Percentage of Subjects in the PES Surviving at 18 Months of Age
Hide Description The percentage of subjects in the Primary Efficacy Analysis Set (PES) who survived to at least 18 months of age.
Time Frame from week 0 to data cut-off (27 to 164 weeks of treatment)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Evaluable subjects in the Primary Efficacy Analysis Set (PES), which included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa. Non-evaluable subjects (n=3) were defined as subjects who were alive, still in the study, and had not yet reached 18 months of age.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: percentage of subjects
50
3.Secondary Outcome
Title Percentage of Subjects in the PES Surviving at 24 Months of Age
Hide Description The percentage of subjects in the Primary Efficacy Analysis Set (PES) who survived to at least 24 months of age.
Time Frame from week 0 to data cut-off (27 to 164 weeks of treatment)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Evaluable subjects in the Primary Efficacy Analysis Set (PES), which included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa. Non-evaluable subjects (n=4) were defined as subjects who were alive, still in the study, and had not yet reached 24 months of age.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: percentage of subjects
40
4.Secondary Outcome
Title Median Age at Death
Hide Description [Not Specified]
Time Frame from week 0 to data cut-off (27 to 164 weeks of treatment)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects in the Primary Efficacy Ananlysis Set (PES) who died. The PES included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 3
Median (Full Range)
Unit of Measure: months
2.9
(2.8 to 4.3)
5.Secondary Outcome
Title Effect on Growth Parameters (Weight-for-age)
Hide Description Changes from baseline in percentiles for weight-for-age (WFA)
Time Frame from week 0 to data cut-off (27 to 164 weeks of treatment)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects in the Primary Efficacy Analysis Set (PES) who had growth failure at baseline and who survived beyond week 4 of treatment. The PES included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: subjects
WFA increases across 2 major percentiles 1
WFA increases across 3 major percentiles 2
WFA increases across 4 major percentiles 1
WFA increases across 5 major percentiles 1
6.Secondary Outcome
Title Dichotomous Growth Status Indicators
Hide Description The percentages of subjects meeting criteria for each dichotomous indicator of under nutrition, i.e., underweight (at least 2 SD below median for weight-for-age [WFA]), wasting (at least 2 SD below median for weight-for-length or -height [WFL/WFH]), and stunting (at least 2 SD below median for length- or height-for-age [LFA/HFA])
Time Frame Month 12 of treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects in the Primary Efficacy Analysis Set (PES) with available anthropometric data at Month 12 of treatment as of the data cut-off date (10 June 2014). The PES included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: percentage of subjects
Underweight (at least 2 SD below median for WFA) 0
Wasting (at least 2 SD below median for WFL/WFH) 0
Stunting (at least 2 SD below median for LFA/HFA) 25
7.Secondary Outcome
Title Changes in Serum Transaminases
Hide Description Change from baseline for alanine aminotransferase (ALT) and aspartate aminotransferase (AST)
Time Frame from week 0 to weeks 1 and 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects in the Primary Efficacy Analysis Set (PES) with available data at both baseline and week 1 (or week 4). The PES included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 9
Median (Full Range)
Unit of Measure: Change from baseline (U/L)
ALT at week 1 (n=7)
-23
(-161 to 28)
ALT at week 4 (n=5)
-33
(-226 to -4)
AST at week 1 (n=6)
-43
(-327 to -12)
AST at week 4 (n=4)
-55.5
(-427 to -20)
8.Secondary Outcome
Title Change in Serum Ferritin
Hide Description Change from baseline in serum ferritin
Time Frame from week 0 to week 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects in the Primary Efficacy Analysis Set (PES) with available data at both baseline and week 1. The PES included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 4
Median (Full Range)
Unit of Measure: change from baseline (µg/L)
-122
(-6934 to -72)
9.Secondary Outcome
Title Percentage of Subjects Achieving Transfusion-free Hemoglobin Normalization
Hide Description The percentage of subjects achieving transfusion-free hemoglobin normalization (TFHN) of ≥ 4 weeks at any time during the study (also referred to as short-term TFHN), and the percentage of subjects who maintained TFHN for ≥ 13 weeks beginning at Week 6 (also referred to as sustained early TFHN). A subject was considered to have achieved short-term TFHN if the/she had two post-baseline measurements of hemoglobin, obtained at least 4 weeks apart, that were above the age-adjusted lower limit of normal (LLN), and had no additional hemoglobin measurements below LLN during this minimum 4-week period and no transfusions administered during the minimum 4-week period or for 2 weeks prior to the start of this period.
Time Frame from week 0 to data cut-off (27 to 164 weeks of treatment)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects in the Primary Efficacy Analysis Set (PES) who received treatment with sebelipase alfa for at least 4 weeks (and could therefore be assessed for short-term TFHN). The PES included subjects who received any amount of sebelipase alfa and who were ≤ 8 months of age on the date of their first infusion of sebelipase alfa.
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description:
Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: percentage of subjects
TFHN for at least 4 weeks 83
TFHN by Week 6 and continuing at least 13 weeks 33
Time Frame From week 0 to the data cut-off (27 to 164 weeks of treatment)
Adverse Event Reporting Description Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the subject's parent or legal guardian.
 
Arm/Group Title Open-Label Sebelipase Alfa
Hide Arm/Group Description Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
All-Cause Mortality
Open-Label Sebelipase Alfa
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Open-Label Sebelipase Alfa
Affected / at Risk (%)
Total   8/9 (88.89%) 
Blood and lymphatic system disorders   
Lypmadenopathy  1  1/9 (11.11%) 
Cardiac disorders   
Tachycardia  1 [1]  1/9 (11.11%) 
Cardiac arrest  1  1/9 (11.11%) 
Gastrointestinal disorders   
Diarrhoea  1  1/9 (11.11%) 
Peritoneal haemorrhage  1  1/9 (11.11%) 
General disorders   
Chills  1 [1]  1/9 (11.11%) 
Pyrexia  1 [1]  2/9 (22.22%) 
Hyperthermia  1  1/9 (11.11%) 
Hepatobiliary disorders   
Hepatic failure  1  1/9 (11.11%) 
Infections and infestations   
Bacterial pyelonephritis  1  1/9 (11.11%) 
Catheter site infection  1  2/9 (22.22%) 
Device related infection  1  2/9 (22.22%) 
Device related sepsis  1  1/9 (11.11%) 
Respiratory tract infection  1  1/9 (11.11%) 
Roseola  1  1/9 (11.11%) 
Staphylococcal bacteremia  1  1/9 (11.11%) 
Staphylococcal sepsis  1  1/9 (11.11%) 
Upper respiratory tract infection  1  1/9 (11.11%) 
Viral infection  1  1/9 (11.11%) 
Investigations   
Weight decreased  1  1/9 (11.11%) 
Metabolism and nutrition disorders   
Dehydration  1  1/9 (11.11%) 
Failure to thrive  1  1/9 (11.11%) 
Food intolerance  1  1/9 (11.11%) 
Metabolic acidosis  1  1/9 (11.11%) 
Vascular disorders   
Pallor  1 [1]  1/9 (11.11%) 
Poor venous access  1  1/9 (11.11%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.1
[1]
Infusion-related reaction (1 event each of Grade 3 tachycardia and pallor + Grade 2 chills and pyrexia) at 1 infusion on Wk 12 (3 mg/kg qw); resolved same day after infusion interruption and administration of antihistamine, antipyretic, and IV saline
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Open-Label Sebelipase Alfa
Affected / at Risk (%)
Total   9/9 (100.00%) 
Blood and lymphatic system disorders   
Anaemia  1  4/9 (44.44%) 
Thrombocytopenia  1  1/9 (11.11%) 
Cardiac disorders   
Tachycardia  1  2/9 (22.22%) 
Bradycardia  1  2/9 (22.22%) 
Cardiovascular disorders  1  1/9 (11.11%) 
Congenital, familial and genetic disorders   
Hydrocele  1  2/9 (22.22%) 
Ear and labyrinth disorders   
Ear pain  1  1/9 (11.11%) 
Middle ear inflammation  1  1/9 (11.11%) 
Endocrine disorders   
Adrenal insufficiency  1  1/9 (11.11%) 
Eye disorders   
Conjunctivitis  1  1/9 (11.11%) 
Pupillary disorder  1  1/9 (11.11%) 
Gastrointestinal disorders   
Vomiting  1  6/9 (66.67%) 
Diarrhoea  1  6/9 (66.67%) 
Abdominal distension  1  1/9 (11.11%) 
Nausea  1  1/9 (11.11%) 
Teething  1  2/9 (22.22%) 
Ascites  1  1/9 (11.11%) 
Faeces discoloured  1  1/9 (11.11%) 
Gastroesophageal reflux disease  1  1/9 (11.11%) 
Haematochezia  1  1/9 (11.11%) 
Post-tusssive vomiting  1  1/9 (11.11%) 
Retching  1  1/9 (11.11%) 
Tongue dicolouration  1  1/9 (11.11%) 
Umbilical hernia  1  1/9 (11.11%) 
General disorders   
Pyrexia  1  5/9 (55.56%) 
Chills  1  1/9 (11.11%) 
Hyperthemia  1  1/9 (11.11%) 
Catheter site rash  1  1/9 (11.11%) 
Device dislocation  1  1/9 (11.11%) 
Disease progression  1  1/9 (11.11%) 
Extravasation  1  1/9 (11.11%) 
Feeling abnomal  1  1/9 (11.11%) 
Gait disturbance  1  1/9 (11.11%) 
Granuloma  1  1/9 (11.11%) 
Infusion site oedema  1  1/9 (11.11%) 
Irritabiliy  1  1/9 (11.11%) 
Oedema peripheral  1  1/9 (11.11%) 
Pain  1  1/9 (11.11%) 
Hepatobiliary disorders   
Cholelithiasis  1  1/9 (11.11%) 
Hyperbilirubianaemia  1  1/9 (11.11%) 
Infections and infestations   
Rhinitis  1  5/9 (55.56%) 
Nasopharyngitis  1  3/9 (33.33%) 
Device related infection  1  2/9 (22.22%) 
Ear infection viral  1  2/9 (22.22%) 
Bronchiolitis  1  2/9 (22.22%) 
Catheter site infection  1  2/9 (22.22%) 
Gastroenteritis  1  1/9 (11.11%) 
Pharyngitis  1  1/9 (11.11%) 
Varicella  1  2/9 (22.22%) 
Bacterial infection  1  1/9 (11.11%) 
Bronchitis  1  1/9 (11.11%) 
Candida nappy rash  1  1/9 (11.11%) 
Escherichia urinary tract infection  1  1/9 (11.11%) 
Eyelid infection  1  1/9 (11.11%) 
Fungal infection  1  1/9 (11.11%) 
Metapneumovirus infection  1  1/9 (11.11%) 
Oral candidiasis  1  1/9 (11.11%) 
Oral fungal infection  1  1/9 (11.11%) 
Oral herpes  1  1/9 (11.11%) 
Otitis media  1  1/9 (11.11%) 
Post procedural infection  1  1/9 (11.11%) 
Rash pustular  1  1/9 (11.11%) 
Staphylococcal skin infection  1  1/9 (11.11%) 
Upper respiratory tract infection  1  1/9 (11.11%) 
Viral infection  1  1/9 (11.11%) 
Wound infection fungal  1  1/9 (11.11%) 
Injury, poisoning and procedural complications   
Contusion  1  1/9 (11.11%) 
Endotracheal intubation complication  1  1/9 (11.11%) 
Limb injury  1  1/9 (11.11%) 
Mouth injury  1  1/9 (11.11%) 
Post procedural discharge  1  1/9 (11.11%) 
Postoperative ileus  1  1/9 (11.11%) 
Procedural site reaction  1  1/9 (11.11%) 
Investigations   
Staphylococcus test positive  1  1/9 (11.11%) 
Blood immunoglobulin A abnormal  1  1/9 (11.11%) 
Blood immunoglobulin G abnormal  1  1/9 (11.11%) 
Blood lactate dehydrogenase increased  1  1/9 (11.11%) 
Blood potassium decreased  1  1/9 (11.11%) 
Blood urea increased  1  1/9 (11.11%) 
Body temperature increased  1  1/9 (11.11%) 
Lymphocyte count abnormal  1  1/9 (11.11%) 
Monocyte count increased  1  1/9 (11.11%) 
Neutrophil count increased  1  1/9 (11.11%) 
Norovirus test positive  1  1/9 (11.11%) 
Oxygen saturation decreased  1  1/9 (11.11%) 
Platelet count increased  1  1/9 (11.11%) 
Respiratory syncytial virus test positive  1  1/9 (11.11%) 
Serum ferritin increased  1  1/9 (11.11%) 
Transaminases increased  1  1/9 (11.11%) 
Urine albumin/creatinine ratio increased  1  1/9 (11.11%) 
Urine output decreased  1  1/9 (11.11%) 
Vitamin D decreased  1  1/9 (11.11%) 
Lymphocyte count increased  1  1/9 (11.11%) 
Metabolism and nutrition disorders   
Decreased appetite  1  1/9 (11.11%) 
Dehydration  1  1/9 (11.11%) 
Hypercalcaemia  1  1/9 (11.11%) 
Hyperglycaemia  1  1/9 (11.11%) 
Hypernatraemia  1  1/9 (11.11%) 
Hypocalcaemia  1  1/9 (11.11%) 
Hypophosphataemia  1  1/9 (11.11%) 
Hypopproteinaemia  1  1/9 (11.11%) 
Hypovolaemia  1  1/9 (11.11%) 
Iron deficiency  1  1/9 (11.11%) 
Metabolic acidosis  1  1/9 (11.11%) 
Vitamin D deficiency  1  1/9 (11.11%) 
Nervous system disorders   
Hypotonia  1  1/9 (11.11%) 
Psychiatric disorders   
Agitation  1  1/9 (11.11%) 
Reproductive system and breast disorders   
Penile blister  1  1/9 (11.11%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  3/9 (33.33%) 
Rhinorrhoea  1  2/9 (22.22%) 
Pharyngeal erythema  1  1/9 (11.11%) 
Respiratory failue  1  1/9 (11.11%) 
Sneezing  1  1/9 (11.11%) 
Tonsillar disorder  1  1/9 (11.11%) 
Use of accessory respiratory muscles  1  1/9 (11.11%) 
Wheezing  1  1/9 (11.11%) 
Skin and subcutaneous tissue disorders   
Dermatitis diaper  1  3/9 (33.33%) 
Rash  1  2/9 (22.22%) 
Urticaria  1  3/9 (33.33%) 
Eczema  1  2/9 (22.22%) 
Pruritus generalised  1  1/9 (11.11%) 
Umbilical erythema  1  1/9 (11.11%) 
Vascular disorders   
Hypotension  1  1/9 (11.11%) 
Hypertension  1  1/9 (11.11%) 
Pallor  1  1/9 (11.11%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.1
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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
Name/Title: Mark Friedman, Medical Director
Organization: Alexion Pharmaceuticals
Phone: 781-357-9953
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01371825     History of Changes
Other Study ID Numbers: LAL-CL03
First Submitted: June 9, 2011
First Posted: June 13, 2011
Results First Submitted: January 14, 2016
Results First Posted: April 18, 2016
Last Update Posted: November 14, 2017