Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

Study Identifier:
D7413C00001
ClinicalTrials.gov Identifier:
NCT05876351
EudraCT Identifier:
2025-000162-29
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Atypical Hemolytic Uremic
Study Drug
  • Drug: Eculizumab
Date
Jul 2023 - May 2025
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 0 - 99 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Any age weighing ≥ 5 kg
  • Complement treatment naïve with evidence of TMA.
  • History of aHUS prior to kidney transplant,or persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen.
  • Among participants with onset of TMA postpartum, persistent evidence of TMA for \> 3 days after the day of childbirth
  • All participants must be vaccinated against N meningitidis if not already vaccinated within the time period of active coverage specified by the vaccine manufacturer.
  • Participants \< 18 years of age must have been vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to local vaccination schedule guidelines.
  • In participants receiving treatment with medications known to cause TMA, persistent evidence of TMA at least 4 days after modifying the excluded medication
Exclusion Criteria
  • Known familial or acquired ADAMTS13deficiency (activity \< 5%).
  • ST-HUS as demonstrated by local guidelines.
  • Positive direct Coombs test which is indicative of a clinically significant immune-mediated hemolysis not due to aHUS.
  • HIV infection, and /or unresolved meningococcal disease
  • Ongoing sepsis, and / or presence or suspicion of active and untreated systemic infection
  • Organ transplantation history, and/or Bone marrow transplant/hematopoietic stem cell transplant within 6 months prior to the start of Screening.
  • Among participants with a kidney transplant, acute kidney dysfunction within 4 weeks of transplant consistent with the diagnosis of acute antibody-mediated rejection.
  • Among participants without a kidney transplant, history of kidney disease other than aHUS
  • Identified drug exposure-related HUS, and / or HUS related to vitamin B12 deficiency and / or known genetic defects of cobalamin C metabolism.
  • History of malignancy within 5 years of Screening.
  • Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
  • Chronic dialysis.
  • Prior use of complement inhibitors.
  • Use of tranexamic acid within 7 days prior to the start of Screening.
  • Other immunosuppressive therapies.
  • Receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks prior to the start of Screening.
  • Received vasopressors or inotropes within 7 days prior to Screening.
  • Previously or currently treated with a complement inhibitor.
  • Has participated in another interventional treatment study or used any experimental therapy.
  • Hypersensitivity to any excipient in eculizumab.
  • Pregnant or breastfeeding.
Sex
Female & Male
Age
0 - 99 Years

Study Details

Medical Condition
  • Atypical Hemolytic Uremic
Study Drug
  • Drug: Eculizumab
Date
Jul 2023 - May 2025
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 0 - 99 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Any age weighing ≥ 5 kg
  • Complement treatment naïve with evidence of TMA.
  • History of aHUS prior to kidney transplant,or persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen.
  • Among participants with onset of TMA postpartum, persistent evidence of TMA for \> 3 days after the day of childbirth
  • All participants must be vaccinated against N meningitidis if not already vaccinated within the time period of active coverage specified by the vaccine manufacturer.
  • Participants \< 18 years of age must have been vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to local vaccination schedule guidelines.
  • In participants receiving treatment with medications known to cause TMA, persistent evidence of TMA at least 4 days after modifying the excluded medication
Exclusion Criteria
  • Known familial or acquired ADAMTS13deficiency (activity \< 5%).
  • ST-HUS as demonstrated by local guidelines.
  • Positive direct Coombs test which is indicative of a clinically significant immune-mediated hemolysis not due to aHUS.
  • HIV infection, and /or unresolved meningococcal disease
  • Ongoing sepsis, and / or presence or suspicion of active and untreated systemic infection
  • Organ transplantation history, and/or Bone marrow transplant/hematopoietic stem cell transplant within 6 months prior to the start of Screening.
  • Among participants with a kidney transplant, acute kidney dysfunction within 4 weeks of transplant consistent with the diagnosis of acute antibody-mediated rejection.
  • Among participants without a kidney transplant, history of kidney disease other than aHUS
  • Identified drug exposure-related HUS, and / or HUS related to vitamin B12 deficiency and / or known genetic defects of cobalamin C metabolism.
  • History of malignancy within 5 years of Screening.
  • Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
  • Chronic dialysis.
  • Prior use of complement inhibitors.
  • Use of tranexamic acid within 7 days prior to the start of Screening.
  • Other immunosuppressive therapies.
  • Receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks prior to the start of Screening.
  • Received vasopressors or inotropes within 7 days prior to Screening.
  • Previously or currently treated with a complement inhibitor.
  • Has participated in another interventional treatment study or used any experimental therapy.
  • Hypersensitivity to any excipient in eculizumab.
  • Pregnant or breastfeeding.

Protocol Summary

This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with atypical hemolytic uremic syndrome (aHUS) in China

Trial Locations

Location
Status
Location
Research Site
Beijing, China, 100034
Status
N/A
Location
Research Site
Beijing, China, 100045
Status
N/A
Location
Research Site
Changsha, China, 410007
Status
N/A
Location
Research Site
Qingdao, China, 110016
Status
N/A
Location
Research Site
Taiyuan, China, 030012
Status
N/A
Location
Research Site
Wuhan, China, 430030
Status
N/A