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

• Register: ClinicalTrials.gov
• Last refreshed on: 18 June 2024
• Main ID: NCT05086744
• Date of registration: 28/09/2021
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders
• Scientific 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
• Date of first enrolment: December 21, 2021
• Target sample size: 19
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT05086744
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

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

Key inclusion & exclusion 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.


Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cold Agglutinin Disease (CAD)

Immune Thrombocytopenia (ITP)

Intervention(s)

Drug: Iptacopan

Primary Outcome(s)

Platelet count [Time Frame: Day 1 to Day 85]

Hemoglobin levels [Time Frame: Day 1 to Day 85]

Secondary Outcome(s)

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

Pharmacokinetic parameter: Cmax [Time Frame: Day 15 and Day 57: 0 hours, 0.5 hours, 1 hour, 2 hours, 4 hours and 6 hours]

Platelet count [Time Frame: Day 1 to Day 85]

Haptoglobin [Time Frame: Baseline, Day 15, Day 29, Day 85]

Hemoglobin levels [Time Frame: Day 1 to Day 85]

Lactate dehydrogenase (LDH) [Time Frame: Baseline, Day 15, Day 29, Day 85]

Pharmcokinetic parameter: Tmax [Time Frame: 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]

Pharmcokinetic parameter: Ctrough [Time Frame: Day 15, Day 29 and Day 57: 0 hours/predose]

Total billirubin [Time Frame: Baseline, Day 15, Day 29, Day 85]

Number of adverse events and serious adverse events [Time Frame: Up to end of study (Day 757) in Part B]

Reticulocytes count [Time Frame: Baseline, Day 1, Day 15, Day 29, Day 85]

Secondary ID(s)

2021-002039-40

CLNP023L12201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors