Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| ALX-PNH-502 | Registry Identifier | Alexion |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment. |
| |
| Cohort 2 | Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Danicopan | Drug | Participants treated with danicopan as an add-on therapy. |
| |
| Measure | Description | 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 |
| Measure | Description | 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 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
The study population will consist of 2 treatment cohorts:
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Research Site | Boston | Massachusetts | 02210 | United States |
Not provided
Not provided
Not provided
Not provided
| Soliris/Ultomiris |
| Drug |
Participants treated with Soliris/Ultomiris monotherapy. |
|
| 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 |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000718467 | danicopan |
| C481642 | eculizumab |
| C000629409 | ravulizumab |
Not provided
Not provided
Not provided