Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

Overview

About this study

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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Main 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

Main 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 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 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 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

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 6/8/23. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Hatem Amer, M.D.

Contact us for the latest status

Contact information:

Adam Miller

(507) 266-8147

Miller.Adam@mayo.edu

Jacksonville, Fla.

Mayo Clinic principal investigator

Hani Wadei, M.D.

Contact us for the latest status

Contact information:

Clinical Studies Unit

(904) 953-2255

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Hasan Khamash, M.D.

Contact us for the latest status

Contact information:

Carla Owen R.N.

(480) 301-6198

Owen.Carla@mayo.edu

More information

Publications

Publications are currently not available