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

Last Update: Mar 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:

NCT04747613

Novartis Reference Number:CLNP023C12001B

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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.

Condition

Paroxysmal Nocturnal Hemoglobinuria

Phase

Phase 3

Overall Status

Recruiting

Number of Participants

250

Start Date

Jul 27, 2021

Completion Date

Feb 07, 2028

Gender

All

Age(s)

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

Recruiting

Duarte,(626-218-0247) -- Vinod Pullarkat,91010 - California,United States

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Phone: 1-888-669-6682

Email: [email protected]

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

Study Description

Interventions

Iptacopan

Eligibility Criteria

Study Location

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Cleveland Clinic Foundation

Univ of California Irvine (Chao Family Comprehensive Cancer Center)

Lakes Research

USC Norris Cancer Center

Montefiore Medical Center .

Augusta University

City Of Hope

Worldwide Contacts

Novartis Pharmaceuticals

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