- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05086744
Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders
An Open-label, Multi-center, Phase 2 Basket Study to Assess Efficacy, Safety and Pharmacokinetics of Iptacopan (LNP023) in Participants With Autoimmune Benign Hematological Disorders
Study Overview
Status
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Novartis Pharmaceuticals
- Phone Number: 1-888-669-6682
- Email: novartis.email@novartis.com
Study Contact Backup
- Name: Novartis Pharmaceuticals
- Phone Number: +41613241111
Study Locations
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Essen, Germany, 45147
- Novartis Investigative Site
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MI
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Milano, MI, Italy, 20122
- Novartis Investigative Site
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Seoul, Korea, Republic of, 06351
- Novartis Investigative Site
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Madrid, Spain, 28009
- Novartis Investigative Site
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Murcia, Spain, 30008
- Novartis Investigative Site
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Catalunya
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Barcelona, Catalunya, Spain, 08035
- Novartis Investigative Site
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London, United Kingdom, W12 0HS
- Novartis Investigative Site
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Massachusetts
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Boston, Massachusetts, United States, 02114
- Massachusetts General Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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 Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Iptacopan 200 mg BID
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Iptacopan 200 mg BID
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Platelet count
Time Frame: Day 1 to Day 85
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Cohort 1: Ability of iptacopan to induce a clinically meaningful increase in platelet count in participants with primary ITP
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Day 1 to Day 85
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Hemoglobin levels
Time Frame: Day 1 to Day 85
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Cohort 2: Ability of iptacopan to induce a clinically meaningful increase in hemoglobin levels in participants with primary CAD
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Day 1 to Day 85
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Platelet count
Time Frame: Day 1 to Day 85
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Time to first response
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Day 1 to Day 85
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Hemoglobin levels
Time Frame: Day 1 to Day 85
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Time to first response
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Day 1 to Day 85
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Platelet count
Time Frame: Day 1 to Day 85
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Duration of response
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Day 1 to Day 85
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Hemoglobin levels
Time Frame: Day 1 to Day 85
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Duration of response
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Day 1 to Day 85
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Platelet count
Time Frame: Day 1 to Day 85
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Magnitude of response
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Day 1 to Day 85
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Hemoglobin levels
Time Frame: Day 1 to Day 85
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Magnitude of response
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Day 1 to Day 85
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Number of patients who use rescue therapy
Time Frame: Day 1 to Day 85
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Need for rescue therapy
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Day 1 to Day 85
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Lactate dehydrogenase (LDH)
Time Frame: Screening, Day 15, Day 29, Day 85, Day 113
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Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers
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Screening, Day 15, Day 29, Day 85, Day 113
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Total billirubin
Time Frame: Screening, Day 15, Day 29, Day 85, Day 113
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Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers
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Screening, Day 15, Day 29, Day 85, Day 113
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Reticulocytes count
Time Frame: Screening, Baseline, Day 1, Day 15, Day 29, Day 85, Day 99, Day 113
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Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers
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Screening, Baseline, Day 1, Day 15, Day 29, Day 85, Day 99, Day 113
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Haptoglobin
Time Frame: Screening, Day 15, Day 29, Day 85, Day 113
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Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers
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Screening, Day 15, Day 29, Day 85, Day 113
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Pharmacokinetic parameter: Cmax
Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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Pharmacokinetics (PK) of iptacopan
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Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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Pharmcokinetic parameter: AUCtau
Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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Pharmacokinetics (PK) of iptacopan
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Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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Pharmcokinetic parameter: AUClast
Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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Pharmacokinetics (PK) of iptacopan
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Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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Pharmcokinetic parameter: Ctrough
Time Frame: Day 15, Day 29 and Day 57: 0 hours/predose
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Pharmacokinetics (PK) of iptacopan
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Day 15, Day 29 and Day 57: 0 hours/predose
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Pharmcokinetic parameter: Tmax
Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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Pharmacokinetics (PK) of iptacopan
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Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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Number of adverse events and serious adverse events
Time Frame: Up to end of study (Day 757) in Part B
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Safety and tolerability of iptacopan in participants with autoimmune benign hematological disorders
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Up to end of study (Day 757) in Part B
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Immune System Diseases
- Autoimmune Diseases
- Hemorrhage
- Hemorrhagic Disorders
- Anemia
- Blood Coagulation Disorders
- Skin Manifestations
- Blood Platelet Disorders
- Anemia, Hemolytic
- Thrombotic Microangiopathies
- Purpura, Thrombocytopenic
- Purpura
- Cytopenia
- Hematologic Diseases
- Purpura, Thrombocytopenic, Idiopathic
- Thrombocytopenia
- Anemia, Hemolytic, Autoimmune
Other Study ID Numbers
- CLNP023L12201
- 2021-002039-40 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient level data and supporting clinical documents from applicable studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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