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

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.

Conditions

Atypical Hemolytic Uremic Syndrome

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Study Overview

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Study Details

Study overview

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.

A Multicenter, Single-arm, Open Label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult aHUS Patients Who Are Naive to Complement Inhibitor Therapy

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

Condition
Atypical Hemolytic Uremic Syndrome
Intervention / Treatment

-

Contacts and Locations

Birmingham

Uni Of Alabama At Birmingham Division of Nephrology, Birmingham, Alabama, United States, 35233

Los Angeles

USC Norris Cancer Center, Los Angeles, California, United States, 90033

Orange

Univ Cali Irvine ALS Neuromuscular Nephrology Department, Orange, California, United States, 92868

Torrance

Harbor-UCLA Medical Center ., Torrance, California, United States, 90502

Washington

Georgetown University Lombardi Cancer Center, Washington, District of Columbia, United States, 20007 2197

Miami

University Of Miami, Miami, Florida, United States, 33136

Minneapolis

University of Minnesota, Minneapolis, Minnesota, United States, 55455

Saint Louis

WA Uni School Of Med ., Saint Louis, Missouri, United States, 63110

Albuquerque

University of New Mexico Clin and Transl Science Ctr, Albuquerque, New Mexico, United States, 87131-0001

Bronx

Montefiore Medical Center ., Bronx, New York, United States, 10461

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Novartis Pharmaceuticals,

Novartis Pharmaceuticals, STUDY_DIRECTOR, Novartis Pharmaceuticals

Study Record Dates

2026-01-06