Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Registration Number
- NCT04747613
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief 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
- Detailed Description
The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 208
Not provided
- Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
- History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- History of hematopoietic stem cell transplantation
Other protocol-defined inclusion/exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Iptacopan Iptacopan Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy
- Primary Outcome Measures
Name Time Method Proportion of participants with adverse events 60 months Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
- Secondary Outcome Measures
Name Time Method Proportion of participants who remain free from transfusions 60 months Proportion of participants who remain free from transfusions evaluated over yearly intervals
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions 60 months Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
Rate of breakthrough hemolysis (BTH) 60 months Rate of breakthrough hemolysis (BTH)
Proportion of participants with Major Adverse Vascular Events MAVEs 60 months MAVEs (incl. thrombosis) evaluated over yearly intervals
Trial Locations
- Locations (9)
City Of Hope
🇺🇸Duarte, California, United States
USC Norris Cancer Center
🇺🇸Los Angeles, California, United States
Univ of California Irvine (Chao Family Comprehensive Cancer Center)
🇺🇸Orange, California, United States
Lakes Research
🇺🇸Miami Lakes, Florida, United States
Augusta University
🇺🇸Augusta, Georgia, United States
Montefiore Medical Center
🇺🇸Bronx, New York, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
Prisma Health Upstate
🇺🇸Greenville, South Carolina, United States
Novartis Investigative Site
🇬🇧London, United Kingdom
City Of Hope🇺🇸Duarte, California, United States