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

Sponsor

Kira Pharmacenticals (US), LLC. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05504187

Collaborator

(none)

Enrollment

Arms

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Systemic Lupus Erythematosus	Drug: KP104	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

24 participants

Allocation:

Non-Randomized

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Nov 1, 2022

Anticipated Primary Completion Date :

Oct 1, 2023

Anticipated Study Completion Date :

Oct 1, 2023

Arms and Interventions

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 64 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 64 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 2 for 24 Weeks. After the last participant completes 64 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.

Outcome Measures

Primary Outcome Measures

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]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 (<)100,000/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.
Positive nasopharyngeal swab for Neisseria meningitidis at Screening or a prior history of meningitis.

Only key inclusion and exclusion criteria have been included.