Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

Novartis

Status and phase: Enrolling; Phase 3

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Treatments: Drug: Iptacopan

Study type: Interventional

Funder types: Industry

Identifiers

NCT04747613

CLNP023C12001B

Details and patient eligibility

About

This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan

Full description

The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.

Enrollment: 250 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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 LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension.
• 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

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
• History of hematopoietic stem cell transplantation

Other protocol-defined inclusion/exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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250 participants in 1 patient group

Iptacopan

Experimental group

Description:

Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy

Treatment:

Drug: Iptacopan