Paroxysmal Nocturnal Hemoglobinuria, PNH
Conditions
Keywords
Paroxysmal Nocturnal Hemoglobinuria, PNH, Danicopan, Ravulizumab, Eculizumab
Brief summary
This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of danicopan as add-on therapy to ravulizumab/eculizumab in participants with PNH.
Interventions
DRUGDanicopan
Participants treated with danicopan as an add-on therapy.
DRUGSoliris/Ultomiris
Participants treated with Soliris/Ultomiris monotherapy.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult participants aged ≥ 18 years at treatment initiation. * Initiated treatment with Ultomiris, Soliris, and/or danicopan on or after IPIG or Alexion International PNH Registry enrollment.
Exclusion criteria
* Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years |
| Event Rate of Meningococcal Infections | From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years |
| Event Rate of Serious Infections | Up to approximately 5 years |
| Event Rate of Malignancies and Hematologic Abnormalities | Up to approximately 5 years |
Secondary
| Measure | Time frame |
|---|---|
| Number of Participants with AEs and SAEs in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding | Up to approximately 5 years |
| Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding | Up to 12 months |
| Number of Participants with PNH symptoms at Diagnosis | Baseline (Day 1) |
| Number of Participants with a History Bone Marrow Transplant | Baseline (Day 1) |
| Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis | Baseline (Day 1) |
| Number of Participants with Ongoing Severe Hepatic Impairment as Defined by Child-Pugh Class C at Enrollment | Baseline (Day 1) |
| Number of Participants Who Discontinue Danicopan Treatment | Up to approximately 5 years |
Countries
United States
Outcome results
None listed