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

Running, not enrolling · Phase 3

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria

In brief

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

Key facts

Study ID

NCT04747613

Run by

Novartis Pharmaceuticals

People needed

208

Starts

2021-07-27

Expected to finish

2027-10-18

Last updated by the study team

2026-08-07

Who can join

Age: 18 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• City Of Hope — Duarte, California, United States
• USC Norris Cancer Center — Los Angeles, California, United States
• Univ Cali Irvine ALS Neuromuscular — Orange, California, United States
• Univ of California Irvine (Chao Family Comprehensive Cancer Center) — Orange, California, United States
• Lakes Research — Miami Lakes, Florida, United States
• Augusta University Georgia — Augusta, Georgia, United States
• Augusta University — Augusta, Georgia, United States
• Montefiore Medical Center — The Bronx, New York, United States
• Cleveland Clinic Foundation — Cleveland, Ohio, United States
• Prisma Health Upstate — Greenville, South Carolina, United States
• Novartis Investigative Site — Santo André, São Paulo, Brazil
• Novartis Investigative Site — São Paulo, São Paulo, Brazil
• Novartis Investigative Site — Beijing, China
• Novartis Investigative Site — Tianjin, China
• Novartis Investigative Site — Tianjin, China
• Novartis Investigative Site — Ostrava, Poruba, Czechia
• Novartis Investigative Site — Brno, Czechia
• Novartis Investigative Site — Lille, France
• Novartis Investigative Site — Nantes, France
• Novartis Investigative Site — Nice, France
• Novartis Investigative Site — Paris, France
• Novartis Investigative Site — Toulouse, France
• Novartis Investigative Site — Dresden, Saxony, Germany
• Novartis Investigative Site — Aachen, Germany
• City Of Hope National Med Center — Duarte, California, United States

Full record on ClinicalTrials.gov

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