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
Overview
- Phase
- Phase 2
- Intervention
- KP104
- Conditions
- Systemic Lupus Erythematosus
- Sponsor
- Kira Pharmacenticals (US), LLC.
- Enrollment
- 24
- Primary Endpoint
- 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)
- Status
- Not yet recruiting
- Last Updated
- last year
Overview
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.
Investigators
Eligibility Criteria
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.
Arms & Interventions
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
Intervention: KP104
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.
Intervention: KP104
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.
Intervention: KP104
Part 2: OBD Cohort, Dose 4
Participants will be administered with KP104 OBD for 24 Weeks.
Intervention: KP104
Outcomes
Primary Outcomes
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)
Time Frame: Up to 24 weeks
Part 2: Percent change from Baseline in platelet count
Time Frame: Baseline (Day 1) and up to Week 12
Part 2: Percent change from Baseline in serum lactate dehydrogenase (LDH) levels
Time Frame: Baseline (Day 1) and up to Week 12