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 range18 Years – 120 Years
SexALL
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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…
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