Status:
ACTIVE_NOT_RECRUITING
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Atypical Hemolytic Uremic Syndrome
Eligibility:
All Genders
18-100 years
Phase:
PHASE3
Brief Summary
The purpose of this Phase 3 study is to determine whether iptacopan (LNP023) is efficacious and safe for the treatment of aHUS in adult patients who are treatment naive to complement inhibitor therapy...
Detailed Description
The study is designed as a multicenter, single-arm, open label study to demonstrate the efficacy and safety of LNP023 (iptacopan) at a dose of 200 mg b.i.d. in adult patients with aHUS who are treatme...
Eligibility Criteria
Inclusion
- Main
- Adult patients with evidence of active 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
- Main
Exclusion
- Treatment with complement inhibitors, including anti-C5 antibody
- ADAMTS13 deficiency (\<10% activity or \<0.1U/ml), and/or Shiga toxin-related hemolytic uremic syndrome (STx-HUS), and/or Positive direct Coombs test
- Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS
- Receiving PE/PI, for 14 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 or active severe systemic bacterial, viral (including COVID-19) or fungal infection, systemic infection which confounds an accurate diagnosis of 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
- Other protocol-defined inclusion/exclusion criteria may apply
Key Trial Info
Start Date :
January 17 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 29 2029
Estimated Enrollment :
34 Patients enrolled
Trial Details
Trial ID
NCT04889430
Start Date
January 17 2022
End Date
March 29 2029
Last Update
December 11 2025
Active Locations (59)
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1
Uni Of Alabama At Birmingham
Birmingham, Alabama, United States, 35233
2
USC Norris Cancer Center
Los Angeles, California, United States, 90033
3
Univ of California at Los Angeles
Los Angeles, California, United States, 90095
4
Univ Cali Irvine ALS Neuromuscular
Orange, California, United States, 92868