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NCT05876351 | Recruiting | Atypical Hemolytic Uremic

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

Alexion Pharmaceuticals, Inc.

Brief 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.

Condition or disease

Atypical Hemolytic Uremic

Intervention/treatment

Eculizumab

Phase

Phase 3

Detailed Description:

This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with aHUS in China. The study will be conducted in participants of any age who weigh ≥ 5 kg and who previously have not been treated with complement inhibitors. The study consists of an up to 7-day Screening Period and a 26-week Treatment Period. An 8-week Safety Follow-up Phone Call will be required only for participants who discontinue eculizumab treatment during the study or for participants who will not receive continued access to eculizumab after completing study treatment. Approximately 25 eligible participants in China will be enrolled.}}

Study Type : Interventional
Estimated Enrollment : 25 participants
Masking : None (Open Label)
Primary Purpose : Treatment
Official Title : Prospective, Single-Arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment-Naïve Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China
Actual Study Start Date : July 14, 2023
Estimated Primary Completion Date : March 31, 2026
Estimated Study Completion Date : March 31, 2026
Arm Intervention/treatment

Experimental: Eculizumab

Participants will receive Eculizumab in a single dose vial.

Drug: Eculizumab
Ages Eligible for Study:
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
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.
Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

Location Details

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

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Recruiting

China,

Research Site

Beijing, China, 100034

Recruiting

China,

Research Site

Beijing, China, 100045

Recruiting

China,

Research Site

Changsha, China, 410007

Recruiting

China,

Research Site

Chengdu, China, 610041

Recruiting

China,

Research Site

Guangzhou, China, 510062

Recruiting

China,

Research Site

Nanchang, China, 330006

Recruiting

China,

Research Site

Nanjing, China, 210002

Recruiting

China,

Research Site

Qingdao, China, 110016

Recruiting

China,

Research Site

Shenzhen, China, 518036

Recruiting

China,

Research Site

Suzhou, China, 215002

Recruiting

China,

Research Site

Taiyuan, China, 030012

Recruiting

China,

Research Site

Wuhan, China, 430030

Recruiting

China,

Research Site

Y Antai, China, 264000

Recruiting

China,

Research Site

Zhengzhou, China, 450018

Recruiting

China,

Research Site

Zhengzhou, China, 450052