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Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

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: NCT02112994
Recruitment Status : Completed
First Posted : April 14, 2014
Results First Posted : January 15, 2019
Last Update Posted : December 4, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Lysosomal Acid Lipase Deficiency
Intervention Drug: Sebelipase Alfa
Enrollment 31
Recruitment Details A total of 21 study centers were initiated in 15 countries, including Australia, Belgium, Brazil, Canada, Croatia, Denmark, Germany, Italy, Mexico, Netherlands, Russia, Spain, Turkey, United Kingdom (UK), and the United States. Seventeen study centers screened at least 1 participant in all of these countries, except the UK and the Netherlands.
Pre-assignment Details All participants began treatment with sebelipase alfa at a dose of 1 milligram/kilogram (mg/kg) every other week (qow). Individual participants could escalate/decrease their dose at any time during the study and only at the discretion of the Investigator and in consultation with the Sponsor. All 5 different doses are presented as Milestones below.
Arm/Group Title 2-<4 Years 4-18 Years >18 Years
Hide Arm/Group Description This subgroup is part of the full analysis set and includes only participants between the ages of 2 and 4 years old who initiated intravenous (IV) treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg every week (qw) was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status. This subgroup is part of the full analysis set and includes only participants between the ages of 4 and 18 years old who initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status. This subgroup is part of the full analysis set and includes only participants greater than 18 years old. This subgroup is part of the full analysis set and includes only participants greater than 18 years old who initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
Period Title: Overall Study
Started 6 16 9
Received at Least 1 Dose of Study Drug 6 16 9
Received 0.35 mg/kg QOW 0 0 1
Received 1 mg/kg QOW [1] 6 16 9
Received 1 mg/kg QW 1 1 0
Received 3 mg/kg QOW 3 5 3
Received 3 mg/kg QW 2 1 1
Completed 96-week Treatment Period 6 14 8
Completed [2] 6 14 6
Not Completed 0 2 3
Reason Not Completed
Progressive Disease             0             0             1
Pregnancy             0             1             1
Liver Transplant             0             1             0
Withdrawal by Subject             0             0             1
[1]
All participants started the study at this dose.
[2]
Participants who transitioned to commercial product are displayed as having completed the study.
Arm/Group Title Sebelipase Alfa
Hide Arm/Group Description Pediatric and adult participants initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
Overall Number of Baseline Participants 31
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 31 participants
16.82  (14.674)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants
2 to <4 years
6
  19.4%
4 to 18 years
16
  51.6%
>18 years
9
  29.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants
Female
12
  38.7%
Male
19
  61.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants
Hispanic or Latino
6
  19.4%
Not Hispanic or Latino
25
  80.6%
Unknown or Not Reported
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants
White
27
  87.1%
Other
4
  12.9%
1.Primary Outcome
Title Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)
Hide Description The number of participants experiencing severe TEAEs is presented for participants who received sebelipase alfa in this open-label study. Adverse events (AEs) information was obtained at each scheduled contact with the participant (or participant's parent or legal guardian). An AE was defined as any untoward medical occurrence that did not require a causal relationship with study drug administration. An AE could have been any unfavorable and unintended sign, symptom, or disease temporally associated with the use of the study drug, whether or not considered related to the study drug. Pre-existing conditions that worsened in severity during the study were reported as AEs. A summary of all serious and other non-serious AEs regardless of causality is located in the Reported AE module. Severity assessed using Clinical Data Interchange Standards Consortium Study Data Tabulation Model standard terminology v3.1.1. Data presented only according to age group, not dose of study drug received.
Time Frame Screening, Week 144
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: All participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.
Arm/Group Title 2-<4 Years 4-18 Years >18 Years
Hide Arm/Group Description:
This subgroup is part of the full analysis set and includes only participants between the ages of 2 and 4 years old who initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
This subgroup is part of the full analysis set and includes only participants between the ages of 4 and 18 years old who initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
This subgroup is part of the full analysis set and includes only participants greater than 18 years old. This subgroup is part of the full analysis set and includes only participants greater than 18 years old who initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
Overall Number of Participants Analyzed 6 16 9
Measure Type: Count of Participants
Unit of Measure: Participants
1
  16.7%
1
   6.3%
2
  22.2%
2.Secondary Outcome
Title Percent Change In Serum Lipids From Baseline To Week 144
Hide Description The effect of sebelipase alfa on lipid metabolism was evaluated by measuring the change from baseline to Week 144 in 4 serum lipids: low-density lipoprotein cholesterol (LDL-C); high-density lipoprotein cholesterol (HDL-C); non-HDL-C; triglycerides. Blood samples for these clinical laboratory tests were collected at scheduled time points and analyzed by a central laboratory.
Time Frame Baseline, Week 144
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: All participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.
Arm/Group Title 2-<4 Years 4-18 Years >18 Years
Hide Arm/Group Description:
This subgroup is part of the full analysis set and includes only participants between the ages of 2 and 4 years old who initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
This subgroup is part of the full analysis set and includes only participants between the ages of 4 and 18 years old who initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
This subgroup is part of the full analysis set and includes only participants greater than 18 years old. This subgroup is part of the full analysis set and includes only participants greater than 18 years old who initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
Overall Number of Participants Analyzed 5 12 2
Median (Full Range)
Unit of Measure: Percent Change
LDL-C
-37.5
(-52 to -25)
-29.2
(-59 to 23)
-22.5
(-37 to -8)
HDL-C
76.5
(30 to 132)
24.2
(-4 to 90)
6.1
(-10 to 22)
Non-HDL-C
-39.1
(-53 to -29)
-26.7
(-62 to 19)
-22.1
(-33 to -11)
Triglycerides
-48.3
(-61 to -11)
-15.8
(-74 to 112)
-22.0
(-25 to -19)
3.Secondary Outcome
Title Participants Testing Positive For Anti-drug Antibodies (ADAs)
Hide Description The impact of ADAs on the safety and immunogenicity of sebelipase alfa was evaluated by testing for ADAs in participants who received sebelipase alfa in this open-label study. Blood samples for assessment were collected prior to study infusions at Week 2, Week 4, Week 8, Week 12, and every 12 weeks thereafter. Participants testing positive for ADAs were also tested for the presence of neutralizing antibodies that inhibited sebelipase alfa enzyme activity and/or cellular uptake. Any participant experiencing a moderate or severe infusion-associated reaction (IAR) was to have an additional assessment of ADAs at the next study visit (prior to study drug infusion); these participants were to also have serum samples collected at 1 to 2 hours after IAR onset and at the next study visit (prior to study drug infusion) for analysis of serum tryptase. The count of participants who became ADA positive and who tested positive for neutralizing antibodies are presented.
Time Frame Week 144
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: All participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.
Arm/Group Title Sebelipase Alfa
Hide Arm/Group Description:
Pediatric and adult participants initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
Overall Number of Participants Analyzed 31
Measure Type: Count of Participants
Unit of Measure: Participants
ADA Positive
2
   6.5%
Neutralizing Antibodies Positive
0
   0.0%
4.Secondary Outcome
Title Percent Change In Body Mass Index (BMI)-For-Age Percentile From Baseline To Week 144 In Pediatric Participants
Hide Description To evaluate the effects of sebelipase alfa on growth parameters in pediatric participants (≤18 years old) presenting with evidence of growth delay, the percent change in the anthropometric parameter of BMI-for-age percentile from Baseline to Week 144 is reported. Anthropometric parameters were plotted on standard growth curves. When possible, historical data on growth parameters was also incorporated into the analyses. Percentiles and Z-scores for BMI-for-age were determined using standard growth charts appropriate to a participant's age on the date of the assessment: the World Health Organization standard growth chart for participants ≤2 years of age and the Centers for Disease Control standard growth chart for participants >2 years of age.
Time Frame Baseline, Week 144
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: All pediatric participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.
Arm/Group Title Pediatric Participants
Hide Arm/Group Description:
Pediatric participants initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
Overall Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
26.45  (118.432)
5.Secondary Outcome
Title Shift In Child-Pugh Status From Baseline To Week 144
Hide Description In order to evaluate the effects of sebelipase alfa on liver function, the number of participants with a shift in Child-Pugh status from Baseline to Week 144 is reported. The status is based on the Child-Pugh score, which is used in clinical practice to assess prognosis in individuals with chronic liver disease. Laboratory data were used in derivation of the score by summing individual scores (scored 1-3, with 3 indicating most severe) from clinical laboratory test results and physical examinations, including total serum bilirubin, serum albumin, prothrombin time, ascites, and hepatic encephalopathy. The total score was used to determine the Child-Pugh status, reported as Class A (score of 5 or 6), Class B (score of 7 to 9), or Class C (score of 10 to 15). Higher scores and higher categories represented a worse outcome. Data reported as 1 of 2 types of shifts in class: No Change from Baseline; Decline from Baseline.
Time Frame Baseline, Week 144
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: All participants who received at least 1 infusion of sebelipase alfa. The full analysis set was used for analysis of safety and efficacy.
Arm/Group Title Sebelipase Alfa
Hide Arm/Group Description:
Pediatric and adult participants initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg qw was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.
Overall Number of Participants Analyzed 18
Measure Type: Count of Participants
Unit of Measure: Participants
No Change: A to A
16
  88.9%
No Change: B to B
1
   5.6%
Decline: A to B
1
   5.6%
Time Frame Screening (up to 45 days prior to start of treatment) to Week 144.
Adverse Event Reporting Description All serious and non-serious AE data are displayed by the study drug administered at the time of AE onset.
 
Arm/Group Title 0.35 mg/kg QOW 1.0 mg/kg QOW 1.0 mg/kg QW 3.0 mg/kg QOW 3.0 mg/kg QW
Hide Arm/Group Description This reporting group is based on the safety set and includes AEs with onset during the administration of IV treatment of sebelipase alfa at a dose of 0.35 mg/kg qow. This reporting group is based on the safety set and includes AEs with onset during the administration of IV treatment of sebelipase alfa at a dose of 1.0 mg/kg qow. This reporting group is based on the safety set and includes AEs with onset during the administration of IV treatment of sebelipase alfa at a dose of 1.0 mg/kg qw. This reporting group is based on the safety set and includes AEs with onset during the administration of IV treatment of sebelipase alfa at a dose of 3.0 mg/kg qow. This reporting group is based on the safety set and includes AEs with onset during the administration of IV treatment of sebelipase alfa at a dose of 3.0 mg/kg qw.
All-Cause Mortality
0.35 mg/kg QOW 1.0 mg/kg QOW 1.0 mg/kg QW 3.0 mg/kg QOW 3.0 mg/kg QW
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/1 (0.00%)   0/31 (0.00%)   0/2 (0.00%)   0/11 (0.00%)   0/4 (0.00%) 
Hide Serious Adverse Events
0.35 mg/kg QOW 1.0 mg/kg QOW 1.0 mg/kg QW 3.0 mg/kg QOW 3.0 mg/kg QW
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/1 (0.00%)   8/31 (25.81%)   0/2 (0.00%)   3/11 (27.27%)   0/4 (0.00%) 
Gastrointestinal disorders           
Abdominal pain  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Abdominal pain lower  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Gastrointestinal haemorrhage  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Hepatobiliary disorders           
Hepatic function abnormal  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Immune system disorders           
Anaphylactic reaction  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Infections and infestations           
Pneumonia  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Injury, poisoning and procedural complications           
Clavicle fracture  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Radius fracture  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Metabolism and nutrition disorders           
Fluid overload  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Product Issues           
Device breakage  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Respiratory, thoracic and mediastinal disorders           
Pneumothorax  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Surgical and medical procedures           
Liver transplant  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Vascular disorders           
Shock  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
1
Term from vocabulary, MedDRA 20.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
0.35 mg/kg QOW 1.0 mg/kg QOW 1.0 mg/kg QW 3.0 mg/kg QOW 3.0 mg/kg QW
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/1 (0.00%)   30/31 (96.77%)   0/2 (0.00%)   10/11 (90.91%)   4/4 (100.00%) 
Blood and lymphatic system disorders           
Iron deficiency anaemia  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Macrocytosis  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Cardiac disorders           
Left ventricular dilatation  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Left ventricular hypertrophy  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Endocrine disorders           
Delayed puberty  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Gastrointestinal disorders           
Abdominal pain  1  0/1 (0.00%)  11/31 (35.48%)  0/2 (0.00%)  2/11 (18.18%)  0/4 (0.00%) 
Abdominal pain upper  1  0/1 (0.00%)  5/31 (16.13%)  0/2 (0.00%)  2/11 (18.18%)  0/4 (0.00%) 
Constipation  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Dental caries  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Diarrhoea  1  0/1 (0.00%)  12/31 (38.71%)  0/2 (0.00%)  2/11 (18.18%)  1/4 (25.00%) 
Gastritis  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  2/11 (18.18%)  0/4 (0.00%) 
Gingival bleeding  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Nausea  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  2/11 (18.18%)  1/4 (25.00%) 
Oral contusion  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Tongue eruption  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Toothache  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Vomiting  1  0/1 (0.00%)  9/31 (29.03%)  0/2 (0.00%)  2/11 (18.18%)  1/4 (25.00%) 
General disorders           
Fatigue  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Pyrexia  1  0/1 (0.00%)  15/31 (48.39%)  0/2 (0.00%)  4/11 (36.36%)  1/4 (25.00%) 
Vaccination site erythema  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Hepatobiliary disorders           
Cholelithiasis  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Immune system disorders           
Allergy to arthropod bite  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Infections and infestations           
Acute sinusitis  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Bronchitis  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Conjunctivitis  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Ear infection  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Eye infection  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Gastritis viral  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Gastroenteritis  1  0/1 (0.00%)  6/31 (19.35%)  0/2 (0.00%)  1/11 (9.09%)  2/4 (50.00%) 
Gastroenteritis viral  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Hordeolum  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Influenza  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Molluscum contagiosum  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Nasopharyngitis  1  0/1 (0.00%)  13/31 (41.94%)  0/2 (0.00%)  4/11 (36.36%)  3/4 (75.00%) 
Oral herpes  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  2/11 (18.18%)  0/4 (0.00%) 
Otitis media acute  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Pharyngitis  1  0/1 (0.00%)  6/31 (19.35%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Pharyngotonsillitis  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Respiratory tract infection  1  0/1 (0.00%)  7/31 (22.58%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Respiratory tract infection viral  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Rhinitis  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Sinusitis  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Tonsillitis  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Tracheitis  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Upper respiratory tract infection  1  0/1 (0.00%)  7/31 (22.58%)  0/2 (0.00%)  2/11 (18.18%)  2/4 (50.00%) 
Urinary tract infection  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Varicella zoster virus infection  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Viral infection  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Viral upper respiratory tract infection  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Injury, poisoning and procedural complications           
Arthropod bite  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Bone contusion  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Concussion  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Contusion  1  0/1 (0.00%)  4/31 (12.90%)  0/2 (0.00%)  3/11 (27.27%)  0/4 (0.00%) 
Face injury  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Forearm fracture  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Limb injury  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Procedural pain  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Scratch  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Skin abrasion  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Wound  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Investigations           
Activated partial thromboplastin time prolonged  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Alanine aminotransferase increased  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Aspartate aminotransferase increased  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Blood alkaline phosphatase increased  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Blood cholesterol increased  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Body temperature increased  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
C-reactive protein increased  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Gamma-glutamyltransferase increased  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Low density lipoprotein increased  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Protein total decreased  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Metabolism and nutrition disorders           
Hypomagnesaemia  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Iron deficiency  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Vitamin D deficiency  1  0/1 (0.00%)  4/31 (12.90%)  0/2 (0.00%)  2/11 (18.18%)  0/4 (0.00%) 
Musculoskeletal and connective tissue disorders           
Arthralgia  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Back pain  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Musculoskeletal pain  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)           
Skin papilloma  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Nervous system disorders           
Dizziness  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Headache  1  0/1 (0.00%)  10/31 (32.26%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Hypoaesthesia  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Migraine  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Renal and urinary disorders           
Urinary incontinence  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Reproductive system and breast disorders           
Balanoposthitis  1 [1]  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  0/11 (0.00%)  1/3 (33.33%) 
Respiratory, thoracic and mediastinal disorders           
Bronchospasm  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Catarrh  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Cough  1  0/1 (0.00%)  7/31 (22.58%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Dysphonia  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Dyspnoea  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Epistaxis  1  0/1 (0.00%)  5/31 (16.13%)  0/2 (0.00%)  3/11 (27.27%)  1/4 (25.00%) 
Oropharyngeal pain  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Rhinitis allergic  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Rhinorrhoea  1  0/1 (0.00%)  3/31 (9.68%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Tonsillar hypertrophy  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Skin and subcutaneous tissue disorders           
Dermatitis  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Dry skin  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Ecchymosis  1  0/1 (0.00%)  4/31 (12.90%)  0/2 (0.00%)  2/11 (18.18%)  0/4 (0.00%) 
Eczema  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Erythema  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Petechiae  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Rash  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  1/11 (9.09%)  1/4 (25.00%) 
Rash papular  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
Skin lesion  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Urticaria  1  0/1 (0.00%)  2/31 (6.45%)  0/2 (0.00%)  0/11 (0.00%)  0/4 (0.00%) 
Vascular disorders           
Haematoma  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  2/11 (18.18%)  0/4 (0.00%) 
Hypotension  1  0/1 (0.00%)  1/31 (3.23%)  0/2 (0.00%)  0/11 (0.00%)  1/4 (25.00%) 
Orthostatic hypotension  1  0/1 (0.00%)  0/31 (0.00%)  0/2 (0.00%)  1/11 (9.09%)  0/4 (0.00%) 
1
Term from vocabulary, MedDRA 20.1
Indicates events were collected by systematic assessment
[1]
Only male participants were exposed to this adverse event.
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
Layout table for Results Point of Contact information
Name/Title: Alexion Pharmaceuticals Inc.
Organization: Alexion Pharmaceuticals Inc.
Phone: 855-752-2356
EMail: clinicaltrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02112994    
Other Study ID Numbers: LAL-CL06
2011-004287-30 ( EudraCT Number )
First Submitted: March 20, 2014
First Posted: April 14, 2014
Results First Submitted: December 26, 2018
Results First Posted: January 15, 2019
Last Update Posted: December 4, 2019