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A multicenter, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult atypical hemolytic uremic syndrome (aHUS) patients who are naive to complement inhibitor therapy

Status: Recruiting
To evaluate the efficacy and safety of iptacopan at a dose of 200 mg twice a day for 52 weeks in patients with atypical hemolytic uremic syndrome (aHUS).
Sex: Male or Female
Age Group: 18 years and over
Inclusion Criteria:
• at least 18 years old
• evidence of active Thrombotic Microangiopathy (TMA)
Exclusion Criteria:
• previous or ongoing treatment with complement inhibitors, including anti-C5 antibody
• ADAMTS13 deficiency
• positive test for Shiga toxin * direct Coombs test
• had a bone marrow transplant or hematopoietic stem cell transplant, or a heart, lung, small bowel, pancreas or live transplant
Conditions:
Blood Disorders, Kidney, Prostate & Urinary, Rare Diseases
Keywords:
Clinics and Surgery Center (CSC)
Study Contact: Nattawat Klomjit - klomj001@umn.edu
Principal Investigator: Nattawat Klomjit
Phase: PHASE3
IRB Number: STUDY00020503
See this study on ClinicalTrials.gov