Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome 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

ClinicalTrials.gov Identifier: NCT04889430

Novartis Reference Number: CLNP023F12301

Last Update: Nov 16, 2022

See if you pre-qualify

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

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.

Condition 
Atypical Hemolytic Uremic Syndrome
Phase 
Phase 3
Overall status 
Recruiting
Start date 
Jan 17, 2022
Completion date 
Jan 02, 2026
Gender 
All
Age(s)
18 Years and older (Adult, Older Adult)

Interventions

Drug
Iptacopan
Iptacopan 200mg twice daily oral

Eligibility Criteria

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

Study Locations

United States
Novartis Investigative Site
Recruiting
Torrance, 90502
California
United States
Novartis Investigative Site
Recruiting
Albuquerque, 87131
New Mexico
United States
Austria
Novartis Investigative Site
Recruiting
Innsbruck, 6020
Tyrol
Austria
Novartis Investigative Site
Recruiting
Wien, 1090
-
Austria
Brazil
Novartis Investigative Site
Recruiting
Sao Paulo, 05403 000
SP
Brazil
Novartis Investigative Site
Recruiting
São Paulo, 04038-002
SP
Brazil
China
Novartis Investigative Site
Recruiting
Beijing, 100034
-
China
Czech Republic
Novartis Investigative Site
Recruiting
Ostrava, 708 52
Poruba
Czech Republic
Novartis Investigative Site
Recruiting
Praha 4, 140 00
-
Czech Republic
Novartis Investigative Site
Recruiting
Praha, 12808
-
Czech Republic
Greece
Novartis Investigative Site
Recruiting
Thessaloniki, 570 10
GR
Greece
India
Novartis Investigative Site
Recruiting
Pune, 411011
Maharashtra
India
Novartis Investigative Site
Recruiting
Chandigarh, 160012
Punjab
India
Novartis Investigative Site
Recruiting
Vellore, 632004
Tamil Nadu
India
Novartis Investigative Site
Recruiting
Lucknow, 226014
Uttar Pradesh
India
Japan
Novartis Investigative Site
Recruiting
Iruma-gun, 350-0495
Saitama
Japan
Novartis Investigative Site
Recruiting
Izumo-city, 693 8501
Shimane
Japan
Novartis Investigative Site
Recruiting
Bunkyo ku, 113 8655
Tokyo
Japan
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 03080
-
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 03722
-
Korea, Republic of
Slovenia
Novartis Investigative Site
Recruiting
Ljubljana, 1000
-
Slovenia
Taiwan
Novartis Investigative Site
Recruiting
Taichung, 40447
-
Taiwan
Novartis Investigative Site
Recruiting
Taoyuan, 33305
-
Taiwan
United Kingdom
Novartis Investigative Site
Recruiting
Newcastle Upon Tyne, NE7 7DN
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
Name: 
Novartis Pharmaceuticals
Phone: 

Have a question?

Call 1-888-669-6682 or email [email protected]