Status:
ACTIVE_NOT_RECRUITING
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
18-100 years
Phase:
PHASE3
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 ...
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...
Eligibility Criteria
Inclusion
- Inclusion criteria:
- Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension.
- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
- Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months
- Exclusion Criteria:
- 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.
Exclusion
Key Trial Info
Start Date :
July 27 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 19 2027
Estimated Enrollment :
208 Patients enrolled
Trial Details
Trial ID
NCT04747613
Start Date
July 27 2021
End Date
October 19 2027
Last Update
March 19 2025
Active Locations (57)
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1
City Of Hope
Duarte, California, United States, 91010
2
USC Norris Cancer Center
Los Angeles, California, United States, 90033
3
Univ of California Irvine (Chao Family Comprehensive Cancer Center)
Orange, California, United States, 92868
4
Lakes Research
Miami Lakes, Florida, United States, 33014