Multicenter, Open-label, Single-arm, Post-Marketing Clinical Study to Evaluate the Efficacy and Safety of Ravulizumab in Participants Clinically Diagnosed as Atypical Hemolytic Uremic Syndrome

Status: Recruiting
Location: See all (12) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
SUMMARY

The primary objective of this study is to assess the platelet count response to ravulizumab in participants clinically diagnosed as atypical hemolytic uremic syndrome (aHUS).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• Body weight ≥20 kilograms (kg)

• Participants clinically diagnosed as aHUS who have any of diseases/conditions listed below (including participants in whom Thrombotic microangiopathy (TMA) has not been improved even after treatment for the pathogenesis of diagnosed secondary TMA and therefore, diagnosis of aHUS was made).

• Infection (except for pneumococcal infection and Siga toxin-producing Escherichia coli infection)

• During pregnancy or postpartum

• Post-renal transplantation

• Hypertensive crisis/malignant hypertension

• Systemic lupus erythematosus and related diseases (e.g. dermatomyositis, mixed connective tissue disease, etc.)

• Participants with the following three signs:

• Thrombocytopenia: Platelet count \<150,000/microliter (μL)

• Microangiopathic haemolytic anaemia: Hb \< 10 grams per deciliter (g/dL) (\*)

• Acute kidney injury: one of the following is fulfilled; 1. ΔsCr ≥ 0.3 milligrams per deciliter (mg/dL) (within 48 hours), 2. 1.5-fold increase from baseline sCr (within 7 days), 3. urinary output ≤ 0.5 mL/kg/hour for ≥ 6 hours.

• No prior treatment with complement inhibitors.

• The investigator plans to provide the participant with 26-week treatment with ravulizumab in accordance with the treatment policy in clinical practice.

• Ravulizumab treatment is planned to be initiated within 14 days after onset of the latest TMA episode.

• Participants consenting to meningococcal vaccine administration and appropriate antibiotic prophylaxis (if required).

Locations
Other Locations
Japan
Research Site
NOT_YET_RECRUITING
Bunkyō City
Research Site
NOT_YET_RECRUITING
Hirakata-shi
Research Site
NOT_YET_RECRUITING
Iruma-gun
Research Site
NOT_YET_RECRUITING
Kyoto
Research Site
NOT_YET_RECRUITING
Matsumoto-shi
Research Site
NOT_YET_RECRUITING
Miyazaki
Research Site
RECRUITING
Nagoya
Research Site
NOT_YET_RECRUITING
Nara
Research Site
NOT_YET_RECRUITING
Nerima-ku
Research Site
NOT_YET_RECRUITING
Sapporo
Research Site
NOT_YET_RECRUITING
Shinjuku-ku
Research Site
NOT_YET_RECRUITING
Tsu
Contact Information
Primary
Alexion Pharmaceuticals, Inc. (Sponsor)
clinicaltrials@alexion.com
1-855-752-2356
Time Frame
Start Date: 2025-12-19
Estimated Completion Date: 2027-06-25
Participants
Target number of participants: 20
Treatments
Experimental: Ravulizumab
Participants will receive a weight-based loading dose of ravulizumab, followed by a weight-based dose 2 weeks after loading dose administration, then weight-based maintenance doses every 8 weeks via intravenous (IV) infusion.
Sponsors
Leads: Alexion Pharmaceuticals, Inc.

This content was sourced from clinicaltrials.gov