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

View Full Description

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:

250

Study ID:

NCT04747613

Recruitment Status:

Recruiting

Sponsor:

Novartis Pharmaceuticals

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There are 55 Locations for this study

See Locations Near You

City Of Hope
Duarte California, 91010, United States More Info
Contact
626-218-0247
Vinod Pullarkat
Principal Investigator
USC Norris Cancer Center
Los Angeles California, 90033, United States More Info
Aliya Anvery
Contact
323-865-3900
[email protected]
Ilene Weitz
Principal Investigator
Univ of California Irvine (Chao Family Comprehensive Cancer Center)
Orange California, 92868, United States More Info
Contact
714-509-6558
Zahra Pakbaz
Principal Investigator
Lakes Research
Miami Lakes Florida, 33014, United States More Info
Contact
786-362-5763
Eloy Roman
Principal Investigator
Augusta University
Augusta Georgia, 30912, United States More Info
Latanya Bowman
Contact
706-721-2941
[email protected]
Abdullah Kutlar
Principal Investigator
Montefiore Medical Center .
Bronx New York, 10461, United States More Info
Contact
718-920-2680
Irina Murakhovskaya
Principal Investigator
Cleveland Clinic Foundation
Cleveland Ohio, 44195, United States More Info
Contact
216-444-0441
Jaroslaw Maciejewski
Principal Investigator
Prisma Health Upstate
Greenville South Carolina, 29615, United States More Info
Emmi Chopp
Contact
864-455-9831
[email protected]
Bhumika Patel
Principal Investigator
Novartis Investigative Site
Santo Andre SP, 09090, Brazil
Novartis Investigative Site
Sao Paulo SP, 01323, Brazil
Novartis Investigative Site
Beijing , 10073, China
Novartis Investigative Site
Tianjin , 30002, 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
Nice , 06202, France
Novartis Investigative Site
Paris 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
Roma RM, 00161, Italy
Novartis Investigative Site
Torino TO, 10126, 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
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Suwa Nagano, 392-8, Japan
Novartis Investigative Site
Suita Osaka, 565 0, Japan
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Shinjuku-ku Tokyo, 160-0, Japan
Novartis Investigative Site
Kyoto , 606 8, Japan
Novartis Investigative Site
Niigata , 951 8, Japan
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Seoul , 03080, Korea, Republic of
Novartis Investigative Site
Seoul , 06351, Korea, Republic of
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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
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Singapore , 11922, Singapore
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Barcelona Catalunya, 08036, Spain
Novartis Investigative Site
Santiago De Compostela Galicia, 15706, Spain
Novartis Investigative Site
San Sebastian Pais Vasco, 20080, Spain
Novartis Investigative Site
Hualien , 970, Taiwan
Novartis Investigative Site
Taipei , 10002, Taiwan
Novartis Investigative Site
Istanbul , 34093, Turkey
Novartis Investigative Site
Leeds , LS9 7, United Kingdom
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:

250

Study ID:

NCT04747613

Recruitment Status:

Recruiting

Sponsor:


Novartis Pharmaceuticals

How clear is this clinincal trial information?

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