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Clinical Trials in the UK / NCT04747613
Active, not recruiting Phase 3

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

NCT04747613 · tracked via the Priya Life Science UK tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Started
2021-07-27
Last updated
2026-08-07

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Investigational drug(s) / intervention(s)

Iptacopan

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

Study summary

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

Eligibility

Sex
ALL
Min age
18 Years
Max age
100 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (60)

FacilityCityRegionStatus
City Of Hope National Med Center Duarte California
City Of Hope Duarte California
USC Norris Cancer Center Los Angeles California
Univ Cali Irvine ALS Neuromuscular Orange California
Univ of California Irvine (Chao Family Comprehensive Cancer Center) Orange California
Lakes Research Miami Lakes Florida
Augusta University Georgia Augusta Georgia
Augusta University Augusta Georgia
Montefiore Medical Center The Bronx New York
Cleveland Clinic Foundation Cleveland Ohio
Prisma Health Upstate Greenville South Carolina
Novartis Investigative Site Santo André São Paulo
Novartis Investigative Site São Paulo São Paulo
Novartis Investigative Site Beijing China
Novartis Investigative Site Tianjin China
Novartis Investigative Site Tianjin China
Novartis Investigative Site Ostrava Poruba
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
Novartis Investigative Site Aachen Germany
Novartis Investigative Site Essen Germany
Novartis Investigative Site Hamburg Germany
Novartis Investigative Site Riesa Germany
Novartis Investigative Site Ulm Germany
Novartis Investigative Site Ascoli Piceno AP
Novartis Investigative Site Avellino AV
Novartis Investigative Site Florence FI
Novartis Investigative Site Milan MI
Novartis Investigative Site Roma RM
Novartis Investigative Site Torino TO
Novartis Investigative Site Bassano del Grappa VI
Novartis Investigative Site Nagoya Aichi-ken
Novartis Investigative Site Isehara Kanagawa
Novartis Investigative Site Suwa Nagano
Novartis Investigative Site Suita Osaka

+ 20 more sites — see the full list on the official registry below.

More Novartis Pharmaceuticals trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04747613 on ClinicalTrials.gov ↗ ← All trials in the UK