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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT05086744
Other study ID # CLNP023L12201
Secondary ID 2021-002039-40
Status Terminated
Phase Phase 2
First received
Last updated
Start date December 21, 2021
Est. completion date May 17, 2024

Study information

Verified date June 2024
Source Novartis
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The main purpose of this study is 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.


Recruitment information / eligibility

Status Terminated
Enrollment 19
Est. completion date May 17, 2024
Est. primary completion date September 20, 2023
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: All Cohorts: - Written informed consent - Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is 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 infection within 14 days prior to first study drug administration. - Any medical condition deemed likely to interfere with the participant's participation in the study - Any malignant disease diagnosed within the past 5 years, with the exception of localized non-melanoma skin cancer, in situ cervical cancer, or, for CAD, a low-grade lymphoproliferative disorder. - History of bone marrow/hematopoietic stem cell or solid organ transplantation. - Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after last iptacopan dose - Active severe bleeding or history of intracranial hemorrhage. - Liver disease, or liver injury as indicated by abnormal liver function tests. - Severe concurrent comorbidities of unstable medical conditions. Cohort 1 specific exclusion criteria: - Secondary ITP, as may arise in the setting of certain autoimmune disorders, immunodeficiency syndromes, infections, malignancies, and drug treatments - No ITP-directed background therapy permitted, with the exception of either a thrombopoietin receptor agonist or low-dose corticosteroid, as long as stable dosage for at least 4 weeks prior to first iptacopan dose - Abnormal coagulation screening labs Cohort 2 specific exclusion criteria: - Secondary cold agglutinin syndrome, as may arise in the setting of certain infections, autoimmune disorders, and malignancies (with the exception of a low-grade lymphoproliferative disorder) - No CAD-directed background therapy permitted Additional protocol-defined inclusion / exclusion criteria may apply.

Study Design


Intervention

Drug:
Iptacopan
Iptacopan 200 mg BID

Locations

Country Name City State
Germany Novartis Investigative Site Essen
Italy Novartis Investigative Site Milano MI
Korea, Republic of Novartis Investigative Site Seoul
Spain Novartis Investigative Site Barcelona Catalunya
Spain Novartis Investigative Site Madrid
Spain Novartis Investigative Site Murcia
United Kingdom Novartis Investigative Site London
United States Massachusetts General Hospital Boston Massachusetts

Sponsors (1)

Lead Sponsor Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Germany,  Italy,  Korea, Republic of,  Spain,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Platelet count Cohort 1: Ability of iptacopan to induce a clinically meaningful increase in platelet count in participants with primary ITP Day 1 to Day 85
Primary Hemoglobin levels Cohort 2: Ability of iptacopan to induce a clinically meaningful increase in hemoglobin levels in participants with primary CAD Day 1 to Day 85
Secondary Platelet count Time to first response Day 1 to Day 85
Secondary Hemoglobin levels Time to first response Day 1 to Day 85
Secondary Platelet count Duration of response Day 1 to Day 85
Secondary Hemoglobin levels Duration of response Day 1 to Day 85
Secondary Platelet count Magnitude of response Day 1 to Day 85
Secondary Hemoglobin levels Magnitude of response Day 1 to Day 85
Secondary Number of patients who use rescue therapy Need for rescue therapy Day 1 to Day 85
Secondary Lactate dehydrogenase (LDH) Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers Baseline, Day 15, Day 29, Day 85
Secondary Total billirubin Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers Baseline, Day 15, Day 29, Day 85
Secondary Reticulocytes count Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers Baseline, Day 1, Day 15, Day 29, Day 85
Secondary Haptoglobin Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers Baseline, Day 15, Day 29, Day 85
Secondary Pharmacokinetic parameter: Cmax Pharmacokinetics (PK) of iptacopan Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
Secondary Pharmcokinetic parameter: AUClast Pharmacokinetics (PK) of iptacopan Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
Secondary Pharmcokinetic parameter: Ctrough Pharmacokinetics (PK) of iptacopan Day 15, Day 29 and Day 57: 0 hours/predose
Secondary Pharmcokinetic parameter: Tmax Pharmacokinetics (PK) of iptacopan Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
Secondary Number of adverse events and serious adverse events Safety and tolerability of iptacopan in participants with autoimmune benign hematological disorders Up to end of study (Day 757) in Part B
See also
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