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Sponsor decision
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This is a prospective, non-interventional, multicenter study to evaluate the efficacy and safety of iptacopan in patients with PNH in real-world settings in Russia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| iptacopan | Adults with PNH who started the iptacopan therapy |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of patients with an increase in Hb level ≥ 20 g/l | Number of patients who achieved an increase in Hb level ≥ 20 g/l over 6 months after iptacopan treatment initiation and were Red Blood Cells (RBC) transfusion independent. | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of patients with an increase in Hb level ≥ 120 g/L | Number of patients who achieved an increase in Hb level ≥ 120 g/L at 12 months after initiation of iptacopan treatment and were RBC transfusion independent | 12 months |
| Number of patients with an increase in Hb level ≥ 20 g/L |
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Inclusion Criteria:
Exclusion Criteria:
1. Any situations where iptacopan is contraindicated in accordance with the Summary of Product Characteristics.
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Enrollment of female and male patients with PNH
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Syktyvkar | Komi | 167904 | Russia | ||
| Novartis Investigative Site |
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| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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Number of patients who achieved an increase in Hb level ≥ 20 g/L at 12 months after initiation of iptacopan treatment and was RBC transfusion independent |
| 12 months |
| Change from baseline in reticulocyte count (10⁹/L) | Change from baseline in reticulocyte count (10⁹/L) over 12 months after initiation of iptacopan treatment | 12 months |
| Change from baseline in LDH levels (U/L) | Change from baseline in LDH levels (U/L) over 12 months after initiation of iptacopan treatment | 12 months |
| Number of patients with an absence of administration of packed-RBC transfusions | Number of patients with an absence of administration of packed-RBC transfusions at 12 months after initiation of iptacopan treatment | 12 months |
| Change from baseline in the Hb level (g/L) | Change from baseline in the Hb level (g/L) at 3, 6 and 12 months after initiation of iptacopan treatment | Baseline, month 3, month 6 and month 12 |
| Number of breakthrough hemolysis (BTH) | Number of breakthrough hemolysis (BTH) cases 12 months following the initiation of iptacopan therapy | 12 months |
| Percentage of patients by BMI | Percentage of patients by Body mass index (BMI) (<25, 25.1-29.9, >30) | 12 months |
| Grozny |
| 364047 |
| Russia |
| Novartis Investigative Site | Moscow | 129110 | Russia |
| Novartis Investigative Site | Murmansk | 183047 | Russia |
| Novartis Investigative Site | Yuzhno-Sakhalinsk | 693000 | Russia |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |