ClinicalTrials.gov record
Active, not recruiting Phase 3 Interventional

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

ClinicalTrials.gov ID: NCT04747613

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.

ClinicalTrials.gov public records Last synced May 9, 2026, 6:41 AM EDT

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

Study identification

NCT ID
NCT04747613
Recruitment status
Active, not recruiting
Study type
Interventional
Phase
Phase 3
Lead sponsor
Novartis Pharmaceuticals
Industry
Enrollment
208 participants

Conditions and interventions

Interventions

  • Iptacopan Drug

Drug

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 26, 2021
Primary completion
Oct 17, 2027
Completion
Oct 17, 2027
Last update posted
May 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 May 7, 2026 · Synced May 9, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04747613 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →