ACTIVE_NOT_RECRUITING

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

Conditions

Atypical Hemolytic Uremic Syndrome LNP023 iptacopan aHUS thrombotic microangiopathy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2022-01-17

Study Completion:2029-03-29

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04889430

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.

Ages Eligible for Study:18 Years to 100 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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

Inclusion Criteria

Exclusion 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

Contacts and Locations

Principal Investigator

Novartis Pharmaceuticals

STUDY_DIRECTOR

Novartis Pharmaceuticals

Study Locations (Sites)

Uni Of Alabama At Birmingham

Birmingham, Alabama, 35233

United States

USC Norris Cancer Center

Los Angeles, California, 90033

United States

Univ of California at Los Angeles

Los Angeles, California, 90095

United States

Univ Cali Irvine ALS Neuromuscular

Orange, California, 92868

United States

Univ of California at Sacramento

Sacramento, California, 95817

United States

Harbor-UCLA Medical Center .

Torrance, California, 90502

United States

Georgetown University Lombardi Cancer Center

Washington D.C., District of Columbia, 20007 2197

United States

University Of Miami

Miami, Florida, 33136

United States

Brigham and Womens Hospital

Boston, Massachusetts, 02115

United States

University of Minnesota

Minneapolis, Minnesota, 55455

United States

St Barnabas Hospital

Livingston, New Jersey, 07039

United States

Rut Univ for Translational Med Scie

New Brunswick, New Jersey, 08901

United States

Montefiore Medical Center .

The Bronx, New York, 10461

United States

Montefiore Medical Center

The Bronx, New York, 10461

United States

Duke University Medical Center

Durham, North Carolina, 27710

United States

University of Cincinnati

Cincinnati, Ohio, 45267-0585

United States

Cleveland Clinic Foundation

Cleveland, Ohio, 44195

United States

Comprehensive Transplant Ctr at OSU

Columbus, Ohio, 43210

United States

UT Southwestern Medical Center

Dallas, Texas, 75390

United States

Virginia Commonwealth University

Richmond, Virginia, 23298

United States

Collaborators and Investigators

Sponsor: Novartis Pharmaceuticals

Novartis Pharmaceuticals, STUDY_DIRECTOR, Novartis Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-01-17

Study Completion Date2029-03-29

Study Record Updates

Study Start Date2022-01-17

Study Completion Date2029-03-29

Terms related to this study

Keywords Provided by Researchers

LNP023
iptacopan
aHUS
atypical hemolytic uremic syndrome
thrombotic microangiopathy

Additional Relevant MeSH Terms

Atypical Hemolytic Uremic Syndrome