Study Stopped
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NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice
PRIORITY- PNH
A Prospective, Non-interventional Study to Evaluate the Efficacy and Safety of Iptacopan in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) in Routine Clinical Practice in the Russian Federation
1 other identifier
observational
6
1 country
5
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Jul 2025
Shorter than P25 for all trials
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 17, 2025
CompletedFirst Posted
Study publicly available on registry
June 25, 2025
CompletedStudy Start
First participant enrolled
July 29, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 25, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
December 25, 2025
CompletedApril 13, 2026
April 1, 2026
5 months
June 17, 2025
April 7, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Secondary Outcomes (8)
Number of patients with an increase in Hb level ≥ 120 g/L
12 months
Number of patients with an increase in Hb level ≥ 20 g/L
12 months
Change from baseline in reticulocyte count (10⁹/L)
12 months
Change from baseline in LDH levels (U/L)
12 months
Number of patients with an absence of administration of packed-RBC transfusions
12 months
- +3 more secondary outcomes
Study Arms (1)
iptacopan
Adults with PNH who started the iptacopan therapy
Eligibility Criteria
Enrollment of female and male patients with PNH
You may qualify if:
- Age ≥18 years at the start of iptacopan therapy.
- The patient is undergoing treatment with iptacopan.
- The treating physician decided to prescribe iptacopan based on the Summary of Product Characteristics during routine clinical practice, regardless of study participation.
- Provision of written informed consent.
You may not qualify if:
- \. Any situations where iptacopan is contraindicated in accordance with the Summary of Product Characteristics.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (5)
Novartis Investigative Site
Syktyvkar, Komi, 167904, Russia
Novartis Investigative Site
Grozny, 364047, Russia
Novartis Investigative Site
Moscow, 129110, Russia
Novartis Investigative Site
Murmansk, 183047, Russia
Novartis Investigative Site
Yuzhno-Sakhalinsk, 693000, Russia
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 17, 2025
First Posted
June 25, 2025
Study Start
July 29, 2025
Primary Completion
December 25, 2025
Study Completion
December 25, 2025
Last Updated
April 13, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will not share