Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematol… (NCT05086744) | Clinical Trial Compass
TerminatedPhase 2
Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders
Stopped: Business decision
United States, Germany, Italy19 participantsStarted 2021-12-21
Plain-language summary
The main purpose of this study was to evaluate the efficacy and safety of iptacopan in participants with autoimmune benign hematological disorders such as primary immune thrombocytopenia and primary cold agglutinin disease.
Who can participate
Age range
18 Years – 120 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
All Cohorts:
* Written informed consent
* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections was required and vaccination against Haemophilus influenzae infection is recommended prior to the start of treatment.
* Weight of at least 35 kg
Cohort 1 specific inclusion criteria:
* Participants with a diagnosis of persistent or chronic primary ITP
* Participants must have received at least 1 unique prior therapy administered with the intention to treat ITP
* Sustained thrombocytopenia
Cohort 2 specific inclusion criteria:
* Participants with a diagnosis of primary CAD
* Participants must have received at least 1 unique prior therapy administered with the intention to treat CAD
* Laboratory evidence of ongoing hemolysis
* Sustained anemia
Exclusion Criteria:
All cohorts:
* Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 30 days, whichever is longer; or longer if required by local regulations
* Past or concomitant use of medications prohibited by the protocol
* Known or suspected hereditary or acquired complement deficiency
* History of primary or secondary immunodeficiency, including a positive HIV test result
* Chronic infection with Hepatitis B or C virus
* History of recurrent invasive infections caused by encapsulated organisms, including Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae
* Presence or suspicion of any active infect…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Cohort 1 (ITP): Number of Participants With a Clinically Meaningful Response
Timeframe: Up to 12 weeks (Part A)
2
Cohort 2 (CAD): Number of Participants With a Clinically Meaningful Response