Last Update: Jun 27, 2024
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 Phase3 Studies With Iptacopan
ClinicalTrials.gov Identifier:
Novartis Reference Number:CLNP023C12001B
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

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 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.

Paroxysmal Nocturnal Hemoglobinuria
Phase3
Recruiting
250
Jul 27, 2021
Jan 10, 2028
All
18 Years - (Adult, Older Adult)

Interventions

Drug

Iptacopan

Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral

Eligibility 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 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.

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