Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)

Study Identifier:
ALXN1210-aHUS-312
ClinicalTrials.gov Identifier:
NCT03131219
EudraCT Identifier:
2016-002499-29
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Available Documents

ProtocolStatistical Analysis Plan

Study Details

Medical Condition
  • Atypical Hemolytic Uremic Syndrome (aHUS)
Study Drug
  • Biological: Ravulizumab
Date
Aug 2017 - Dec 2022
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Complement Inhibitor Treatment Naïve:
  • Participants from birth up to \<18 years of age and weighing ≥5 kilograms (kg) at the time of consent.
  • Participants had not been previously treated with complement inhibitors.
  • Evidence of thrombotic microangiopathy (TMA), including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
  • Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae type b.
  • Female participants of childbearing potential and male participants with female partners of childbearing potential must have used highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
  • Eculizumab Experienced:
  • Participants between 12 and \<18 years of age (non-Japanese sites) or \<18 years of age (Japanese sites) who had been treated with eculizumab according to the labelled dosing recommendation for aHUS for at least 90 days prior to screening.
  • Participants with documented diagnosis of aHUS.
  • Participants with clinical evidence of response to eculizumab indicated by stable TMA parameters at screening.
  • Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae type b.
  • Females of childbearing potential and male participants with female partners of childbearing potential must have used highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
Exclusion Criteria
  • Known familial or acquired ADAMTS13 ("a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13") deficiency (activity \<5%).
  • Known Shiga toxin-related hemolytic uremic syndrome.
  • Positive direct Coombs test.
  • Females who planned to become pregnant during the study or were currently pregnancy or breastfeeding.
  • Identified drug exposure-related hemolytic uremic syndrome.
  • Bone marrow transplant/hematopoietic stem cell transplant within the last 6 months prior to the start of screening.
  • Hemolytic uremic syndrome related to known genetic defects of cobalamin C metabolism.
  • Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
  • Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage kidney disease.
  • For eculizumab-experienced participants, prior use of complement inhibitors other than eculizumab.
  • For eculizumab-experienced participants, any known abnormal TMA parameters within 90 days prior to screening.
Sex
Female & Male
Age
N/A

Study Details

Medical Condition
  • Atypical Hemolytic Uremic Syndrome (aHUS)
Study Drug
  • Biological: Ravulizumab
Date
Aug 2017 - Dec 2022
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Complement Inhibitor Treatment Naïve:
  • Participants from birth up to \<18 years of age and weighing ≥5 kilograms (kg) at the time of consent.
  • Participants had not been previously treated with complement inhibitors.
  • Evidence of thrombotic microangiopathy (TMA), including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
  • Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae type b.
  • Female participants of childbearing potential and male participants with female partners of childbearing potential must have used highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
  • Eculizumab Experienced:
  • Participants between 12 and \<18 years of age (non-Japanese sites) or \<18 years of age (Japanese sites) who had been treated with eculizumab according to the labelled dosing recommendation for aHUS for at least 90 days prior to screening.
  • Participants with documented diagnosis of aHUS.
  • Participants with clinical evidence of response to eculizumab indicated by stable TMA parameters at screening.
  • Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae type b.
  • Females of childbearing potential and male participants with female partners of childbearing potential must have used highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
Exclusion Criteria
  • Known familial or acquired ADAMTS13 ("a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13") deficiency (activity \<5%).
  • Known Shiga toxin-related hemolytic uremic syndrome.
  • Positive direct Coombs test.
  • Females who planned to become pregnant during the study or were currently pregnancy or breastfeeding.
  • Identified drug exposure-related hemolytic uremic syndrome.
  • Bone marrow transplant/hematopoietic stem cell transplant within the last 6 months prior to the start of screening.
  • Hemolytic uremic syndrome related to known genetic defects of cobalamin C metabolism.
  • Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
  • Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage kidney disease.
  • For eculizumab-experienced participants, prior use of complement inhibitors other than eculizumab.
  • For eculizumab-experienced participants, any known abnormal TMA parameters within 90 days prior to screening.

Protocol Summary

The purpose of the study is to assess the efficacy of ravulizumab to control disease activity in children and adolescents with aHUS who have not previously used a complement inhibitor (complement inhibitor treatment-naïve), as well as in complement inhibitor-experienced (eculizumab-experienced) adolescent participants.

Trial Locations

Location
Status
Location
Research Site
Hollywood, Florida, United States, 33021
Status
N/A
Location
Research Site
Atlanta, Georgia, United States, 30322
Status
N/A
Location
Research Site
Detroit, Michigan, United States, 48201
Status
N/A
Location
Research Site
Omaha, Nebraska, United States, 68198
Status
N/A
Location
Research Site
Hackensack, New Jersey, United States, 07601
Status
N/A
Location
Research Site
Charlotte, North Carolina, United States, 28203
Status
N/A
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