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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-07-14

Study Completion Date

2025-05-07

Brief Summary

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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

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Detailed Description

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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.

Conditions

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Atypical Hemolytic Uremic

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

open-label study

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Eculizumab

Participants will receive Eculizumab in a single dose vial.

Group Type EXPERIMENTAL

Eculizumab

Intervention Type DRUG

Weight-based doses of Eculizumab will be administered intravenously as an induction dose followed by maintenance dose at Day 8, 15, or 29 depending on weight; then every 2 or 3 weeks, depending upon weight.

Interventions

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Eculizumab

Weight-based doses of Eculizumab will be administered intravenously as an induction dose followed by maintenance dose at Day 8, 15, or 29 depending on weight; then every 2 or 3 weeks, depending upon weight.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Any age weighing ≥ 5 kg
2. Complement treatment naïve with evidence of TMA.
3. History of aHUS prior to kidney transplant,or persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen.
4. Among participants with onset of TMA postpartum, persistent evidence of TMA for \> 3 days after the day of childbirth
5. All participants must be vaccinated against N meningitidis if not already vaccinated within the time period of active coverage specified by the vaccine manufacturer.
6. Participants \< 18 years of age must have been vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to local vaccination schedule guidelines.
7. 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

1. Known familial or acquired ADAMTS13deficiency (activity \< 5%).
2. ST-HUS as demonstrated by local guidelines.
3. Positive direct Coombs test which is indicative of a clinically significant immune-mediated hemolysis not due to aHUS.
4. HIV infection, and /or unresolved meningococcal disease
5. Ongoing sepsis, and / or presence or suspicion of active and untreated systemic infection
6. Organ transplantation history, and/or Bone marrow transplant/hematopoietic stem cell transplant within 6 months prior to the start of Screening.
7. Among participants with a kidney transplant, acute kidney dysfunction within 4 weeks of transplant consistent with the diagnosis of acute antibody-mediated rejection.
8. Among participants without a kidney transplant, history of kidney disease other than aHUS
9. Identified drug exposure-related HUS, and / or HUS related to vitamin B12 deficiency and / or known genetic defects of cobalamin C metabolism.
10. History of malignancy within 5 years of Screening.
11. Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
12. Chronic dialysis.
13. Prior use of complement inhibitors.
14. Use of tranexamic acid within 7 days prior to the start of Screening.
15. Other immunosuppressive therapies.
16. Receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks prior to the start of Screening.
17. Received vasopressors or inotropes within 7 days prior to Screening.
18. Previously or currently treated with a complement inhibitor.
19. Has participated in another interventional treatment study or used any experimental therapy.
20. Hypersensitivity to any excipient in eculizumab.
21. Pregnant or breastfeeding.

Minimum Eligible Age

0 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No