Study to Evaluate Safety, Tolerability and Pharmacodynamics of KP104 in Participants With Thrombotic Microangiopathy Secondary to Systemic Lupus Erythematosus

An Open-label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 in Subjects With Thrombotic Microangiopathy Secondary to Systemic Lupus Erythematosus

This study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KP104 in participants with systemic lupus erythematosus (SLE)-Thrombotic microangiopathy (TMA). The study consists of 2 parts: Part 1 (Dose Optimization) and Part 2 (Proof of Concept). All participants will receive KP104 in combination with standard of care (SOC) for SLE-TMA.

Study Overview

• Status: Not yet recruiting
• Conditions: Systemic Lupus Erythematosus
• Intervention / Treatment: Drug: KP104

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Study Director; Email: privacy@kirapharma.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Meets criteria for SLE per the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) criteria.
• Decrease in platelet count to less than (<)150,000/microliters (mcL).
• Abnormal renal function.
• Females of childbearing potential with negative pregnancy test and males must agree to practice effective contraception from Screening until 28 days after the End of study (EOS) visit.
• Willing and able to provide informed consent.
• Evidence of microangiopathic hemolytic anemia

Exclusion Criteria:

• Diagnosis of other TMA syndromes.
• A renal biopsy within 7 days of screening that shows exclusively chronic changes of TMA.
• Positive Coombs test at the time of TMA diagnosis.
• Active or unresolved Neisseria meningitidis infection at screening.

Only key inclusion and exclusion criteria have been included.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part 1: Dose Optimization Cohort 1, Dose 1; Participants will be administered with KP104 as a weekly maintenance dose for 24 Weeks. After the last participant completes 6 weeks of treatment, all available data, including safety, PK, PD, and modeling results, will be reviewed by the Internal Data Review Committee (IDRC) to determine Dosing Regimen 2	Drug: KP104; KP104 will be administered.
Experimental: Part 1: Dose Optimization Cohort 2, Dose 2; Participants will be administered with KP104 dose regimen 2 for 24 Weeks. After the last participant completes 6 weeks of treatment, all available data, including safety, PK, PD, and modeling results, will be reviewed by the IDRC to determine Dosing Regimen 3.	Drug: KP104; KP104 will be administered.
Experimental: Part 1: Dose Optimization Cohort 3, Dose 3; Participants will be administered with KP104 dose regimen 3 for 24 Weeks. After the last participant completes 6 weeks of treatment, all available data, including safety, PK, PD, and modeling results, will be reviewed by IDRC to determine the Optimal biologic dose (OBD) for Part 2.	Drug: KP104; KP104 will be administered.
Experimental: Part 2: OBD Cohort, Dose 4; Participants will be administered with KP104 OBD for 24 Weeks.	Drug: KP104; KP104 will be administered.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Part 2: Percent change from Baseline in platelet count	Baseline (Day 1) and up to Week 12
Part 2: Percent change from Baseline in serum lactate dehydrogenase (LDH) levels	Baseline (Day 1) and up to Week 12
Parts 1 and 2: Number of participants with Treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs) and Adverse events of special interest (AESIs)	Up to 24 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Parts 1 and 2: Number of participants with Treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs) and Adverse events of special interest (AESIs)	Up to 24 weeks
Parts 1 and 2: Number of participants with change in clinical laboratory values, Electrocardiograms (ECGs), physical examinations, and vital signs	Up to 24 weeks

Collaborators and Investigators

• Sponsor: Kira Pharmacenticals (US), LLC.

Study record dates

Study Major Dates

• Study Start (Estimated): March 1, 2025
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): April 1, 2027

Study Registration Dates

• First Submitted: August 15, 2022
• First Submitted That Met QC Criteria: August 15, 2022
• First Posted (Actual): August 17, 2022

Study Record Updates

• Last Update Posted (Actual): October 28, 2024
• Last Update Submitted That Met QC Criteria: October 25, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: Systemic lupus erythematosus; Proof of Concept; Dose Selection; Thrombotic microangiopathy; KP-104
• Additional Relevant MeSH Terms: Cytopenia; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Hematologic Diseases; Blood Platelet Disorders; Thrombocytopenia; Lupus Erythematosus, Systemic; Thrombotic Microangiopathies
• Other Study ID Numbers: KP104-202

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No