Efficacy and safety of iptacopan (LNP023) in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor therapy

Phase 1

• Conditions: atypical hemolytic uremic syndrome; MedDRA version: 20.1Level: LLTClassification code 10079841Term: Atypical hemolytic uremic syndromeSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2020-005186-13-AT
• Lead Sponsor: ovartis Pharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-05-27
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

- Adult patients with evidence of thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury
- Vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections are required prior to the start of study treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local regulations, at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study treatment and for at least 2 weeks after vaccination
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 45
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

- Treatment with complement inhibitors, including anti-C5 antibody
- ADAMTS13 deficiency (<5% activity), and/or Shiga toxin-related hemolytic uremic syndrome (STx-HUS), and/or Positive Coombs test
- Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase e (DGKE) mediated aHUS
- Receiving PE/PI, for 28 days or longer, prior to the start of screening for the current TMA
- Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation
- Patients with sepsis, severe systemic infection, COVID-19 infection, systemic infection which confounds an accurate diagnosis or aHUS or impedes the ability to manage the aHUS disease, active infection (or history of recurrent invasive infections) caused by encapsulated bacteria
- Kidney disease suggestive of other disease than aHUS or of chronic kidney failure or family history of non-complement mediated genetic kidney disease
- Liver disease or liver injury at screening
- Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
- Chronic hemo- or peritoneal dialysis

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified