Paroxysmal Nocturnal Hemoglobinuria, PNH
Conditions
Keywords
Paroxysmal Nocturnal Hemoglobinuria, PNH, Danicopan
Brief summary
This is a noninterventional cohort study using primary and secondary data from the International PNH Interest Group (IPIG) PNH registry. It is designed to characterize the long-term safety and tolerability of danicopan as add-on therapy to eculizumab or ravulizumab in adult participants with PNH.
Interventions
DRUGDanicopan
Participants will receive Danicopan as an add-on therapy.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult participants aged ≥ 18 years at danicopan treatment initiation (baseline) as add-on therapy to ravulizumab/eculizumab. * Initiated treatment with danicopan on or after registry enrollment
Exclusion criteria
* Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and ravulizumab and/or eculizumab treatment status.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants with Serious Infections Caused by Encapsulated Bacteria | Up to approximately 5 years |
| Number of Participants with Serious Adverse Events (SAEs) | Up to approximately 5 years |
Secondary
| Measure | Time frame |
|---|---|
| Number of Participants Who Discontinue Study Treatment | Up to approximately 5 years |
| Frequency of Reasons for Discontinuation | Up to approximately 5 years |
Countries
United States
Outcome results
None listed