Last Update: Oct 01, 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
Oct 19, 2027
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.

Novartis Investigative Site

Recruiting

Paris 10,75475,France

Novartis Investigative Site

Recruiting

Toulouse,31059,France

Novartis Investigative Site

Recruiting

Lille,59037,France

Novartis Investigative Site

Recruiting

Nantes Cedex 1,44093,France

Novartis Investigative Site

Recruiting

Nice,06202,France

Novartis Investigative Site

Recruiting

Aachen,52074,Germany

Novartis Investigative Site

Recruiting

Dresden,01307,Germany

Novartis Investigative Site

Recruiting

Ulm,89081,Germany

Novartis Investigative Site

Recruiting

Firenze,FI,50134,Italy

Novartis Investigative Site

Recruiting

Milano,MI,20122,Italy

Novartis Investigative Site

Recruiting

Roma,RM,00161,Italy

Novartis Investigative Site

Recruiting

Bassano Del Grappa,Vicenza,36061,Italy

Novartis Investigative Site

Recruiting

Ascoli Piceno,AP,63100,Italy

Novartis Investigative Site

Recruiting

Avellino,AV,83100,Italy

Novartis Investigative Site

Recruiting

Barcelona,Catalunya,08036,Spain

Novartis Investigative Site

Recruiting

London,Se5 9rs,United Kingdom

Novartis Investigative Site

Recruiting

Leeds,West Yorkshire,Ls9 7tf,United Kingdom

Cleveland Clinic Foundation

Recruiting

Cleveland,Ohio,44195,United States

Jaroslaw Maciejewski

Univ of California Irvine (Chao Family Comprehensive Cancer Center)

Recruiting

Orange,California,92868,United States

Zahra Pakbaz

The University of Utah

Recruiting

Salt Lake City,Utah,84132,United States

June Lee
Srinivas Tantravahi

Augusta University

Recruiting

Augusta,Georgia,30912,United States

Abdullah Kutlar
Latanya Bowman

Novartis Investigative Site

Recruiting

Boston,Massachusetts,02114,United States

City Of Hope

Recruiting

Duarte,California,91010,United States

Vinod Pullarkat

Worldwide Contacts

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Novartis Pharmaceuticals

Novartis Pharmaceuticals