JRCT ID: jRCT2021210059
Registered date:17/12/2021
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
Basic Information
Recruitment status | Recruiting |
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Health condition(s) or Problem(s) studied | Paroxysmal Nocturnal Hemoglobinuria |
Date of first enrollment | 09/12/2021 |
Target sample size | 19 |
Countries of recruitment | Italy,Japan,Republic of Korea,Japan,Taiwan,Japan |
Study type | Interventional |
Intervention(s) | Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral |
Outcome(s)
Primary Outcome | Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit |
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Secondary Outcome |
Key inclusion & exclusion criteria
Age minimum | >= 18age old |
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Age maximum | Not applicable |
Gender | Both |
Include criteria | - Male and female participants >=18 years of age with a diagnosis of PNH who have completed Phase 2 or Phase 3 clinical studies - 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 |
Exclude criteria | - Participants who in the parent PNH studies either screen or baseline failed, or prematurely withdrew from the study for any reason - 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 |
Related Information
Primary Sponsor | Suzuki Kazuyuki |
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Secondary Sponsor | |
Source(s) of Monetary Support | |
Secondary ID(s) | NCT04747613 |
Contact
Public contact | |
Name | Kazuyuki Suzuki |
Address | Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan Tokyo Japan 105-6333 |
Telephone | +81-120-003-293 |
rinshoshiken.toroku2@novartis.com | |
Affiliation | Novartis Pharma. K.K. |
Scientific contact | |
Name | Kazuyuki Suzuki |
Address | Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan Tokyo Japan 105-6333 |
Telephone | +81-120-003-293 |
rinshoshiken.toroku2@novartis.com | |
Affiliation | Novartis Pharma. K.K. |