NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

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

NCT04889430

CompletedPhase 3

Sponsor: Novartis Pharmaceuticals

Conditions: Atypical Hemolytic Uremic Syndrome

Interventions: Iptacopan

Countries: United States, Brazil, China, Czechia, India, Japan, South Korea, Taiwan

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.

Eligibility overview

Sex: ALL

Age: 18 Years to 100 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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

Locations (36)

Los Angeles, California, United States
Los Angeles, California, United States
Orange, California, United States
Sacramento, California, United States
Torrance, California, United States
Washington D.C., District of Columbia, United States
Miami, Florida, United States
Minneapolis, Minnesota, United States
The Bronx, New York, United States
The Bronx, New York, United States
Durham, North Carolina, United States
Cincinnati, Ohio, United States
Cleveland, Ohio, United States
Columbus, Ohio, United States
Dallas, Texas, United States
Richmond, Virginia, United States
Fortaleza, Ceará, Brazil
Brasília, Federal District, Brazil
Belo Horizonte, Minas Gerais, Brazil
Recife, Pernambuco, Brazil
Porto Alegre, Rio Grande do Sul, Brazil
São Paulo, São Paulo, Brazil
São Paulo, São Paulo, Brazil
São Paulo, São Paulo, Brazil
Rio de Janeiro, Brazil
Salvador, Brazil
Beijing, China
Ostrava, Poruba, Czechia
Nagpur, Maharashtra, India
Pune, Maharashtra, India
+ 6 more on CT.gov