Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria

• Register: EUCTR
• Last refreshed on: 18 June 2024
• Main ID: EUCTR2020-004385-19-FR
• Date of registration: 15/07/2021
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma AG
• Public title: Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria
• Scientific title: An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan.
• Date of first enrolment: 31/08/2021
• Target sample size: 165
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-004385-19
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Brazil, China, Czech Republic, France, Italy, Japan, Korea, Republic of, Lithuania, Malaysia, Netherlands, Singapore, Spain, Taiwan, United States

Contacts

• Name: Information&Communication Médicales
• Affiliation: Novartis Pharma S.A.S

• Name: Information&Communication Médicales
• Affiliation: Novartis Pharma S.A.S

Key inclusion & exclusion criteria

Inclusion criteria:
- Male and female participants = 18 years of age with a diagnosis of PNH who have completed Phase 2 or Phase 3 clinical studies
- 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 157
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8

Exclusion criteria:
- Participants who in the parent PNH studies either screen or baseline failed, or prematurely withdrew from the study for any reason
- 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
- History of hematopoietic stem cell transplantation
Other protocol-defined inclusion/exclusion criteria may apply.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

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

Intervention(s)

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
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-

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
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

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

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

Timepoint(s) of evaluation of this end point: Time Frame: 60 months

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Time Frame: 60 months

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

Secondary ID(s)

2020-004385-19-LT

CLNP023C12001B

Source(s) of Monetary Support

Novartis Pharma AG

Ethics review

Status: Approved
Approval date: 31/08/2021
Contact: