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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: JPRN
Last refreshed on: 17 October 2023
Main ID:  JPRN-jRCT2021210014
Date of registration: 28/06/2021
Prospective Registration: Yes
Primary sponsor: Kamata Takeo
Public title: Pivotal study of danicopan as add-on therapy to a C5 inhibitor (eculizumab or ravulizumab) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have Clinically Evident EVH
Scientific title: A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
Date of first enrolment: 28/06/2021
Target sample size: 84
Recruitment status: Recruiting
URL:  https://jrct.niph.go.jp/latest-detail/jRCT2021210014
Study type:  Interventional
Study design:  randomized controlled trial, double blind, placebo control, parallel assignment, treatment purpose  
Phase:  3
Countries of recruitment
America Japan
Contacts
Name: Takeo    Kamata
Address:  1-18-14 Ebisu Shibuya-ku 150-0013 Tokyo Japan
Telephone: +81-3-5795-0775
Email: Takeo.Kamata@alexion.com
Affiliation:  Alexion Pharma GK
Name: Takeo    Kamata
Address:  1-18-14 Ebisu Shibuya-ku 150-0013 Tokyo Japan
Telephone: +81-3-57950-0775
Email: Takeo.Kamata@alexion.com
Affiliation:  Alexion Pharma GK
Key inclusion & exclusion criteria
Inclusion criteria: 1 Diagnosis of PNH
2 Clinically Evident Extravascular hemolysis defined by:
Anemia, Hgb is 9.5 g per dL or less than 9.5, with absolute reticulocyte count 120000 per microL or more.
At least 1 packed RBC or whole blood transfusion within 6 months prior to the start of the study
3 Receiving a C5 inhibitor for at least 6 months prior to Day 1 in this study at an approved dose or higher and with no change in dose or interval for at least 24 weeks preceding Day 1. For those patients who recently switched from eculizumab to ravulizumab, they must have received at least the loading dose and 3 maintenance doses of ravulizumab preceding Day 1.
4 Platelet count 30,000 per microL or more than 30,000 per microL without the need for platelet transfusions
5 Absolute neutrophil counts 750 per microL or more
6 Documentation of vaccination for Neisseria meningitidis: All patients must be vaccinated against meningococcal infections within 3 years prior to, or at the time of initiating study
Age 18 years or older.

Exclusion criteria: 1 History of a major organ transplant or hematopoietic stem cell transplantation
2 Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and or immunosuppressants
4 Known or suspected complement deficiency
8 Laboratory abnormalities at screening, including:
Alanine aminotransferase more than 2 x ULN
Direct bilirubin more than 2 x ULN
11 Current evidence of biliary cholestasis
13 Estimated glomerular filtration rate less than 30 mL per min per 1.73 m2 and or are on dialysis.


Age minimum: >= 18age old
Age maximum: Not applicable
Gender: Both
Health Condition(s) or Problem(s) studied
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria
D006457
Paroxysmal Nocturnal Hemoglobinuria
Intervention(s)
Double blinded
Additional Danicopan (ALXN2040) given to patients diagnosed with paroxysmal nocturnal hemoglobinuria and receiving complement C5 inhibitors
Primary Outcome(s)
Change in hemoglobin (Hgb) relative to baseline after 12 weeks of treatment
Secondary Outcome(s)
Proportion of patients with transfusion avoidance
Secondary ID(s)
NCT04469465
Source(s) of Monetary Support
Secondary Sponsor(s)
Ethics review
Status: Approval
Approval date:
Contact:
chiken@fmu.ac.jp
Fukushima medical University Hospital
+81-24-547-1111
chiken@fmu.ac.jp
Results
Results available:
Date Posted:
Date Completed:
URL:
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