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

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

Last Update: Nov 17, 2022

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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

Condition 
Paroxysmal Nocturnal Hemoglobinuria
Phase 
Phase 3
Overall status 
Recruiting
Start date 
Jul 27, 2021
Completion date 
Jun 26, 2026
Gender 
All
Age(s)
18 Years and older (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.

Study Locations

United States
Novartis Investigative Site
Recruiting
Orange, 92868
California
United States
Novartis Investigative Site
Recruiting
Augusta, 30912
Georgia
United States
Brazil
Novartis Investigative Site
Recruiting
Santo Andre, 09090-790
SP
Brazil
Novartis Investigative Site
Recruiting
Sao Paulo, 01323-900
SP
Brazil
China
Novartis Investigative Site
Recruiting
Tianjin, 300020
Tianjin
China
Novartis Investigative Site
Recruiting
Beijing, 100730
-
China
Novartis Investigative Site
Recruiting
Tianjin, 300052
-
China
Czech Republic
Novartis Investigative Site
Recruiting
Ostrava, 708 52
Poruba
Czech Republic
Novartis Investigative Site
Recruiting
Brno - Bohunice, 625 00
-
Czech Republic
France
Novartis Investigative Site
Recruiting
Lille Cedex, 59 037
-
France
Novartis Investigative Site
Recruiting
Paris Cedex 10, 75475
-
France
Novartis Investigative Site
Recruiting
Toulouse, 31059
-
France
Germany
Novartis Investigative Site
Recruiting
Aachen, 52074
-
Germany
Novartis Investigative Site
Recruiting
Essen, 45147
-
Germany
Novartis Investigative Site
Recruiting
Hamburg, 20246
-
Germany
Novartis Investigative Site
Recruiting
Riesa, 01589
-
Germany
Novartis Investigative Site
Recruiting
Ulm, 89081
-
Germany
Italy
Novartis Investigative Site
Recruiting
Ascoli Piceno, 63100
AP
Italy
Novartis Investigative Site
Recruiting
Avellino, 83100
AV
Italy
Novartis Investigative Site
Recruiting
Firenze, 50134
FI
Italy
Novartis Investigative Site
Recruiting
Milano, 20122
MI
Italy
Novartis Investigative Site
Recruiting
Bassano Del Grappa, 36061
VI
Italy
Japan
Novartis Investigative Site
Recruiting
Nagoya, 453-8511
Aichi
Japan
Novartis Investigative Site
Recruiting
Fukushima city, 960 1295
Fukushima
Japan
Novartis Investigative Site
Recruiting
Kanazawa-city, 920-8641
Ishikawa
Japan
Novartis Investigative Site
Recruiting
Isehara, 259-1193
Kanagawa
Japan
Novartis Investigative Site
Recruiting
Suwa, 392-8510
Nagano
Japan
Novartis Investigative Site
Recruiting
Suita, 565 0871
Osaka
Japan
Novartis Investigative Site
Recruiting
Shinjuku-ku, 160-0023
Tokyo
Japan
Novartis Investigative Site
Recruiting
Kyoto, 606 8507
-
Japan
Novartis Investigative Site
Recruiting
Niigata, 951 8520
-
Japan
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 03080
-
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 06351
-
Korea, Republic of
Lithuania
Novartis Investigative Site
Recruiting
Vilnius, LT-08661
-
Lithuania
Malaysia
Novartis Investigative Site
Recruiting
Kota Kinabalu, 88586
Sabah
Malaysia
Novartis Investigative Site
Recruiting
Kuching, 93586
Sarawak
Malaysia
Singapore
Novartis Investigative Site
Recruiting
Singapore, 119228
-
Singapore
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08036
Catalunya
Spain
Taiwan
Novartis Investigative Site
Recruiting
Hualien, 970
-
Taiwan
Novartis Investigative Site
Recruiting
Taipei, 10002
-
Taiwan
United Kingdom
Novartis Investigative Site
Recruiting
London, SE5 9RS
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
Name: 
Novartis Pharmaceuticals
Phone: 

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Call 1-888-669-6682 or email [email protected]