Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Interventions
Product Name: iptacopan
Product Code: LNP023
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Iptacopan
Current Sponsor code: LNP023
Other descriptive name: LNP023 HYDROCHLORIDE SALT
Concentrat
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male and female participants = 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201),Period 4 of CLFGX2201 or completed any Phase III (eg. CLNP023C12302, CLNP023C12301,CLNP023C12303) clinical study at the time point of enrollment visit in this roll over extension study. - Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections - Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 238 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 12
Exclusion criteria
Exclusion criteria: - Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data. - History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae -Female participants who are pregnant or breastfeeding, or intending to conceive during the course of the study. -Women of childbearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping investigational drug.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit;Secondary Objective: 1. Proportion of participants achieving sustained hemoglobin levels = 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals 2. Proportion of participants who remain free from transfusions evaluated over yearly intervals 3. Rate of breakthrough hemolysis (BTH) 4. Proportion of participants with Major Adverse Vascular Events MAVEs (incl. thrombosis) evaluated over yearly intervals;Primary end point(s): Proportion of participants with adverse events ;Timepoint(s) of evaluation of this end point: Time Frame: 60 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Proportion of participants achieving sustained hemoglobin levels = 12 g/dL in the absence of red blood cell transfusions 2. Proportion of participants who remain free from transfusions 3. Rate of breakthrough hemolysis (BTH) 4. Proportion of participants with Major Adverse Vascular Events MAVEs ;Timepoint(s) of evaluation of this end point: Time Frame: 60 months | — |
Countries
Brazil, China, Czech Republic, France, Italy, Japan, Korea, Republic of, Lithuania, Malaysia, Netherlands, Singapore, Spain, Taiwan, United States
Contacts
Public ContactClinical Trial Information Desk
SIA Novartis Baltics Lithuania Branch
Outcome results
None listed