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

March 5, 2024 updated by: Novartis Pharmaceuticals

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

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.

Study Overview

Status

Active, not recruiting

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

19

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

  • Name: Novartis Pharmaceuticals
  • Phone Number: +41613241111

Study Locations

      • Essen, Germany, 45147
        • Novartis Investigative Site
    • MI
      • Milano, MI, Italy, 20122
        • Novartis Investigative Site
      • Seoul, Korea, Republic of, 06351
        • Novartis Investigative Site
      • Madrid, Spain, 28009
        • Novartis Investigative Site
      • Murcia, Spain, 30008
        • Novartis Investigative Site
    • Catalunya
      • Barcelona, Catalunya, Spain, 08035
        • Novartis Investigative Site
      • London, United Kingdom, W12 0HS
        • Novartis Investigative Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

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
Experimental: Iptacopan 200 mg BID
Iptacopan 200 mg BID
Other Names:
  • Generic name: iptacopan
  • Investigational new drug
  • company code: LNP023

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Day 1 to Day 85
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
Day 1 to Day 85

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Platelet count
Time Frame: Day 1 to Day 85
Time to first response
Day 1 to Day 85
Hemoglobin levels
Time Frame: Day 1 to Day 85
Time to first response
Day 1 to Day 85
Platelet count
Time Frame: Day 1 to Day 85
Duration of response
Day 1 to Day 85
Hemoglobin levels
Time Frame: Day 1 to Day 85
Duration of response
Day 1 to Day 85
Platelet count
Time Frame: Day 1 to Day 85
Magnitude of response
Day 1 to Day 85
Hemoglobin levels
Time Frame: Day 1 to Day 85
Magnitude of response
Day 1 to Day 85
Number of patients who use rescue therapy
Time Frame: Day 1 to Day 85
Need for rescue therapy
Day 1 to Day 85
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
Screening, Day 15, Day 29, Day 85, Day 113
Total billirubin
Time Frame: Screening, Day 15, Day 29, Day 85, Day 113
Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers
Screening, Day 15, Day 29, Day 85, Day 113
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
Screening, Baseline, Day 1, Day 15, Day 29, Day 85, Day 99, Day 113
Haptoglobin
Time Frame: Screening, Day 15, Day 29, Day 85, Day 113
Cohort 2 (CAD) only: Effect of iptacopan on relevant disease biomarkers
Screening, Day 15, Day 29, Day 85, Day 113
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
Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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
Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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
Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
Pharmcokinetic parameter: Ctrough
Time Frame: Day 15, Day 29 and Day 57: 0 hours/predose
Pharmacokinetics (PK) of iptacopan
Day 15, Day 29 and Day 57: 0 hours/predose
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
Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours
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
Up to end of study (Day 757) in Part B

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 21, 2021

Primary Completion (Actual)

September 20, 2023

Study Completion (Estimated)

April 30, 2024

Study Registration Dates

First Submitted

September 28, 2021

First Submitted That Met QC Criteria

October 8, 2021

First Posted (Actual)

October 21, 2021

Study Record Updates

Last Update Posted (Estimated)

March 6, 2024

Last Update Submitted That Met QC Criteria

March 5, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

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

Yes

Studies a U.S. FDA-regulated device product

No

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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