Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18+
SponsorAlexion Pharmaceuticals, Inc.
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
Participants with TTP, STEC-HUS, secondary TMA that is obviously unrelated to complement abnormality.
Participants with TMA caused by malignant tumors, abnormal Cobalamin C metabolism, Streptococcus pneumoniae, drugs, autoimmune diseases other than systemic lupus erythematosus and related diseases (e.g. scleroderma etc.), or hematopoietic stem cell transplantation
Participants with pathological complement gene variants (CFH, CFI , CD46 (MCP), C3, CFB, THBD, DGKE) associated with the development of aHUS at enrolment
Participants with positive anti-factor H antibodies
Trial design
Design model
Single group
Treatments tested in this trial
Ravulizumab
DrugParticipants will receive ravulizumab via IV infusion.
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: RavulizumabExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Percentage of Participants Showing Improvement in Platelet Count During the 26-week Ravulizumab Treatment
Time frame
Baseline up to Week 26Secondary outcomes
1
Percentage of Participants Showing Improvement in Renal Function During the 26-week Ravulizumab Treatment
Time frame
Baseline up to Week 262
Percentage of Participants Showing Improvement in Platelet Count
Time frame
Day 4 and on Weeks 1, 2, 10, 18, and 263
Percentage of Participants Showing Improvement in Renal Function
Time frame
Day 4 and on Weeks 1, 2, 10, 18, and 264
Percentage of Participants Showing Improvement in Complete Thrombotic Microangiopathy (TMA) Response or Partial TMA Response
Time frame
Day 4 and on Weeks 1, 2, 10, 18, and 26Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov