Study of LNP023 (iptacopan)to access efficacy and safety in patients who are 18 or more years of age diagnosed with atypical hemolytic uremic syndrome who have never received treatment with complement inhibitors

Phase 3

• Conditions: Health Condition 1: D593- Hemolytic-uremic syndrome

• Registration Number: CTRI/2021/10/037662
• Lead Sponsor: ovartis Healthcare Pvt Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 26 June 2023

Recruitment & Eligibility

• Status: Open to Recruitment
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

1-Male and female patients >= 18 years of age with evidence of thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury, based on the following laboratory findings:

2-Platelet count <150x109/L during the Screening Period or within 28 days prior to the start of the Screening Period, and

3-LDH >=1.5 x upper limit of normal (ULN) during the Screening Period or within 28 days prior to the start of the Screening Period and hemoglobin

<= lower limit of normal (LLN) for age and gender during the Screening Period or within 28 days prior to the start of the Screening Period, and

•Serum creatinine >=ULN during the Screening Period (patients requiring dialysis for acute kidney injury are eligible)

•Vaccination against Neisseria meningitidis infection is 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

4-If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given according to local regulations. The vaccines should be given at least 2 weeks prior to first study drug administration. If iptacopan 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 medication and for at least 2 weeks after vaccination.

5-Among patients with a kidney transplant, (a) known history of aHUS prior to current kidney transplantation, or (b) no known history of aHUS, and persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen

Exclusion Criteria

1-Treatment with complement inhibitors, including anti-C5 antibody

2-ADAMTS13 deficiency, and/or Shiga toxin-related hemolytic uremic syndrome (STX-HUS), and/or Positive direct Coombs test

3-Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS

4-Receiving PE/PI, for 28 days or longer, prior to the start of screening for the current TMA

5-Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation

6-In patients with a kidney transplant, acute kidney dysfunction consistent with the diagnosis of transplantation failure due to acute/chronic active T-Cell mediated rejection (TCMR) and/or active/chronic active antibody-mediated rejection (ABMR) according to Banff 2017 criteria

7-Among patients with native kidney, history of any kidney disease other than aHUS, such as:

8-Known kidney biopsy finding suggestive of underlying disease other than aHUS

9-Kidney ultrasound finding demonstrating small kidneys suggestive of chronic kidney failure

10-Known family history and/or genetic diagnosis of non-complement mediated genetic kidney disease (eg, focal segmental glomerulosclerosis)

11-Liver disease or liver injury at screening

12-Patients with sepsis, severe systemic infection or COVID-19 infection

13-Presence of systemic infections (bacterial, viral, fungal or parasitic) that, in the opinion of the Investigator, confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease

14-Active infection, or history of recurrent invasive infections, caused by encapsulated bacteria (i.e. meningococcus, pneumococcus), or H. influenzae

15-Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome

16-Chronic hemo- or peritoneal dialysis

Study & Design

• Study Type: Interventional
• Study Design: Not specified