Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
Public ClinicalTrials.gov record NCT04747613. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT04747613
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 208 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 100 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 27, 2021 (Actual)
- Primary completion
- Oct 18, 2027 (Estimated)
- Completion
- Oct 18, 2027 (Estimated)
- Last update posted
- Aug 7, 2026
2021 – 2027
United States locations
- U.S. sites
- 11
- U.S. states
- 6
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| City Of Hope National Med Center | Duarte | California | 91010 | — |
| City Of Hope | Duarte | California | 91010 | — |
| USC Norris Cancer Center | Los Angeles | California | 90033 | — |
| Univ Cali Irvine ALS Neuromuscular | Orange | California | 92868 | — |
| Univ of California Irvine (Chao Family Comprehensive Cancer Center) | Orange | California | 92868 | — |
| Lakes Research | Miami Lakes | Florida | 33014 | — |
| Augusta University Georgia | Augusta | Georgia | 30912 | — |
| Augusta University | Augusta | Georgia | 30912 | — |
| Montefiore Medical Center | The Bronx | New York | 10461 | — |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | — |
| Prisma Health Upstate | Greenville | South Carolina | 29615 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 49 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04747613, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 7, 2026 · Synced Oct 22, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04747613 live on ClinicalTrials.gov.