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NCT04747613 · ClinicalTrials.gov registry record · Phase 3
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
A Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria, sponsored by Novartis Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 208
- Enrollment target
- 20
- Study locations
NCT04747613: Active Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria, sponsored by Novartis Pharmaceuticals.
NCT04747613 is a Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria that is active but no longer recruiting, run by Novartis Pharmaceuticals. The registered enrollment target is 208 participants, below the 250-participant average among 14 other Paroxysmal Nocturnal Hemoglobinuria trials with a reported enrollment target (17% lower). The trial reports 20 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04747613, a Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria, is active but no longer recruiting, sponsored by Novartis Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 208 participants
- Enrollment target
- 20
- Study locations
Study Summary
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan
Primary Outcome
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
Conditions Studied
Interventions
- DRUG Iptacopan
Study Locations (20)
Other
- Novartis Investigative Site - Beijing
- Novartis Investigative Site - Tianjin
- Novartis Investigative Site - Tianjin
- Novartis Investigative Site - Brno-Bohunice
- Novartis Investigative Site - Lille
- Novartis Investigative Site - Nantes
- Novartis Investigative Site - Nice
- Novartis Investigative Site - Paris
- Novartis Investigative Site - Toulouse
California
- City Of Hope - Duarte
- USC Norris Cancer Center - Los Angeles
- Univ of California Irvine (Chao Family Comprehensive Cancer Center) - Orange
São Paulo
- Novartis Investigative Site - Santo André
- Novartis Investigative Site - São Paulo
Florida
- Lakes Research - Miami Lakes
Georgia
- Augusta University - Augusta
New York
- Montefiore Medical Center - The Bronx
Ohio
- Cleveland Clinic Foundation - Cleveland
South Carolina
- Prisma Health Upstate - Greenville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 208 participants |
| Start Date | 2021-07-27 |
| Est. Completion | 2027-10-19 |
| Phase | Phase 3 |
What the registry record for NCT04747613 still lists
NCT04747613 is an interventional study that assigns participants to a tested intervention. The registered 208 participants enrollment target is mid-sized for trials with a published cap, below the 250-participant average among 14 other Paroxysmal Nocturnal Hemoglobinuria trials with a reported enrollment target (17% lower).
The record links to 1 condition, with Paroxysmal Nocturnal Hemoglobinuria appearing as the primary indexed condition, and to 1 intervention - of which Iptacopan is the first listed.
NCT04747613 names 20 study sites across 9 states, led by Other, California, São Paulo.
Frequently Asked Questions
What is clinical trial NCT04747613 about?
NCT04747613 is a clinical study titled "Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria". This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan
What is the current status of trial NCT04747613?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 208 participants. The study started on 2021-07-27. Estimated completion is 2027-10-19.
What conditions does trial NCT04747613 study?
This clinical trial studies the following conditions: Paroxysmal Nocturnal Hemoglobinuria.
What interventions are being tested in trial NCT04747613?
The interventions under investigation include: Iptacopan (DRUG).
Who is sponsoring clinical trial NCT04747613?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04747613 being conducted?
This trial has 20 study locations across California, Florida, Georgia, New York, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Paroxysmal Nocturnal Hemoglobinuria
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04747613's enrollment target sits among peer trials
208 3rd of 14 higher than 12 of 14 other Paroxysmal Nocturnal Hemoglobinuria trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Paroxysmal Nocturnal Hemoglobinuria trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
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-
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-
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-
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A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
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Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
COMPLETED · Phase 3
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