Study Description
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. 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 treatment naive to complement inhibitor therapy (including anti-C5
antibody). The study will enroll approximately 50 participants and assess the effects of
iptacopan on a range of efficacy assessments relevant to aHUS including hematological and
kidney parameters, dialysis requirement, changes in chronic kidney disease (CKD) stage,
as well as patient reported outcomes (PRO) for fatigue and quality of life.
Interventions
Iptacopan
Eligibility Criteria
Main Inclusion Criteria:
- 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 Criteria:
- 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
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