Myelodysplastic Syndrome Clinical Trial

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

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

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Full Description

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.

View Eligibility Criteria

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 is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

167

Study ID:

NCT04747613

Recruitment Status:

Recruiting

Sponsor:

Novartis Pharmaceuticals

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There are 42 Locations for this study

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Novartis Investigative Site
Orange California, 92868, United States
Novartis Investigative Site
Augusta Georgia, 30912, United States
Novartis Investigative Site
Santo Andre SP, 09090, Brazil
Novartis Investigative Site
Sao Paulo SP, 01323, Brazil
Novartis Investigative Site
Tianjin Tianjin, 30002, China
Novartis Investigative Site
Beijing , 10073, China
Novartis Investigative Site
Tianjin , 30005, China
Novartis Investigative Site
Ostrava Poruba, 708 5, Czechia
Novartis Investigative Site
Brno - Bohunice , 625 0, Czechia
Novartis Investigative Site
Lille Cedex , 59 03, France
Novartis Investigative Site
Paris Cedex 10 , 75475, France
Novartis Investigative Site
Toulouse , 31059, France
Novartis Investigative Site
Aachen , 52074, Germany
Novartis Investigative Site
Essen , 45147, Germany
Novartis Investigative Site
Hamburg , 20246, Germany
Novartis Investigative Site
Riesa , 01589, Germany
Novartis Investigative Site
Ulm , 89081, Germany
Novartis Investigative Site
Ascoli Piceno AP, 63100, Italy
Novartis Investigative Site
Avellino AV, 83100, Italy
Novartis Investigative Site
Firenze FI, 50134, Italy
Novartis Investigative Site
Milano MI, 20122, Italy
Novartis Investigative Site
Bassano Del Grappa VI, 36061, Italy
Novartis Investigative Site
Nagoya Aichi, 453-8, Japan
Novartis Investigative Site
Fukushima city Fukushima, 960 1, Japan
Novartis Investigative Site
Kanazawa-city Ishikawa, 920-8, Japan
Novartis Investigative Site
Isehara Kanagawa, 259-1, Japan
Novartis Investigative Site
Suwa Nagano, 392-8, Japan
Novartis Investigative Site
Suita Osaka, 565 0, Japan
Novartis Investigative Site
Shinjuku-ku Tokyo, 160-0, Japan
Novartis Investigative Site
Kyoto , 606 8, Japan
Novartis Investigative Site
Niigata , 951 8, Japan
Novartis Investigative Site
Seoul , 03080, Korea, Republic of
Novartis Investigative Site
Seoul , 06351, Korea, Republic of
Novartis Investigative Site
Vilnius , LT-08, Lithuania
Novartis Investigative Site
Kota Kinabalu Sabah, 88586, Malaysia
Novartis Investigative Site
Kuching Sarawak, 93586, Malaysia
Novartis Investigative Site
Nijmegen , 6500 , Netherlands
Novartis Investigative Site
Singapore , 11922, Singapore
Novartis Investigative Site
Barcelona Catalunya, 08036, Spain
Novartis Investigative Site
Hualien , 970, Taiwan
Novartis Investigative Site
Taipei , 10002, Taiwan
Novartis Investigative Site
London , SE5 9, United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

167

Study ID:

NCT04747613

Recruitment Status:

Recruiting

Sponsor:


Novartis Pharmaceuticals

How clear is this clinincal trial information?

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