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Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders

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ClinicalTrials.gov Identifier: NCT05086744
Recruitment Status : Recruiting
First Posted : October 21, 2021
Last Update Posted : February 13, 2023
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Immune Thrombocytopenia (ITP) Cold Agglutinin Disease (CAD) Drug: Iptacopan Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: This is a basket study with different Cohorts.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : December 21, 2021
Estimated Primary Completion Date : September 27, 2023
Estimated Study Completion Date : November 3, 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Blood Disorders

Arms and Interventions
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Arm Intervention/treatment
Experimental: Iptacopan 200 mg BID
Iptacopan 200 mg BID
 Drug: Iptacopan
Iptacopan 200 mg BID
Other Names:
  • Generic name: iptacopan
  • Investigational new drug
  • company code: LNP023


Outcome Measures
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Primary Outcome Measures :
  1. Platelet count [ Time Frame: Day 1 to Day 85 ]
    Cohort 1: Ability of iptacopan to induce a clinically meaningful increase in platelet count in participants with primary ITP

  2. Hemoglobin levels [ Time Frame: Day 1 to Day 85 ]
    Cohort 2: Ability of iptacopan to induce a clinically meaningful increase in hemoglobin levels in participants with primary CAD


Secondary Outcome Measures :
  1. Platelet count [ Time Frame: Day 1 to Day 85 ]
    Time to first response

  2. Hemoglobin levels [ Time Frame: Day 1 to Day 85 ]
    Time to first response

  3. Platelet count [ Time Frame: Day 1 to Day 85 ]
    Duration of response

  4. Hemoglobin levels [ Time Frame: Day 1 to Day 85 ]
    Duration of response

  5. Platelet count [ Time Frame: Day 1 to Day 85 ]
    Magnitude of response

  6. Hemoglobin levels [ Time Frame: Day 1 to Day 85 ]
    Magnitude of response

  7. Number of patients who use rescue therapy [ Time Frame: Day 1 to Day 85 ]
    Need for rescue therapy

  8. Lactate dehydrogenase (LDH) [ Time Frame: Screening, Day 15, Day 29, Day 85, Day 113 ]
    Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers

  9. Total billirubin [ Time Frame: Screening, Day 15, Day 29, Day 85, Day 113 ]
    Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers

  10. Reticulocytes count [ Time Frame: Screening, Baseline, Day 1, Day 15, Day 29, Day 85, Day 99, Day 113 ]
    Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers

  11. Haptoglobin [ Time Frame: Screening, Day 15, Day 29, Day 85, Day 113 ]
    Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers

  12. Pharmacokinetic parameter: Cmax [ Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours ]
    Pharmacokinetics (PK) of iptacopan

  13. Pharmcokinetic parameter: AUCtau [ Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours ]
    Pharmacokinetics (PK) of iptacopan

  14. Pharmcokinetic parameter: AUClast [ Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours ]
    Pharmacokinetics (PK) of iptacopan

  15. Pharmcokinetic parameter: Ctrough [ Time Frame: Day 15, Day 29 and Day 57: 0 hours/predose ]
    Pharmacokinetics (PK) of iptacopan

  16. Pharmcokinetic parameter: Tmax [ Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours ]
    Pharmacokinetics (PK) of iptacopan

  17. Number of adverse events and serious adverse events [ Time Frame: Up to end of study (Day 757) in Part B ]
    Safety and tolerability of iptacopan in participants with autoimmune benign hematological disorders


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05086744


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Locations
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United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Hanny Al-Samkari       hal-samkari@mgh.harvard.edu   
Principal Investigator: Hanny Al-Samkari         
Germany
Novartis Investigative Site Recruiting
Essen, Germany, 45147
Italy
Novartis Investigative Site Recruiting
Bologna, BO, Italy, 40138
Novartis Investigative Site Recruiting
Milano, MI, Italy, 20122
Novartis Investigative Site Recruiting
Trieste, TS, Italy, 34129
Korea, Republic of
Novartis Investigative Site Recruiting
Seoul, Korea, Republic of, 03080
Novartis Investigative Site Recruiting
Seoul, Korea, Republic of, 06351
Spain
Novartis Investigative Site Recruiting
Salamanca, Castilla Y Leon, Spain, 37007
Novartis Investigative Site Recruiting
Barcelona, Catalunya, Spain, 08035
Novartis Investigative Site Recruiting
Madrid, Spain, 28009
Novartis Investigative Site Recruiting
Murcia, Spain, 30008
United Kingdom
Novartis Investigative Site Recruiting
London, United Kingdom, E1 1BB
Novartis Investigative Site Recruiting
London, United Kingdom, W12 0HS
Sponsors and Collaborators
Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05086744    
Other Study ID Numbers: CLNP023L12201
2021-002039-40 ( EudraCT Number )
First Posted: October 21, 2021    Key Record Dates
Last Update Posted: February 13, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thrombocytopenia
Immune System Diseases
Purpura, Thrombocytopenic, Idiopathic
Hematologic Diseases
Anemia, Hemolytic, Autoimmune
Blood Platelet Disorders
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
 Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Anemia, Hemolytic
Anemia