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
Trial Snapshot
- Phase
- Phase 2
- Status
- Not yet recruiting
- 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)
Study 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.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 65 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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 (Drug)
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 (Drug)
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 (Drug)
Part 2: OBD Cohort, Dose 4
Participants will be administered with KP104 OBD for 24 Weeks.
Intervention: KP104 (Drug)
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
Secondary Outcomes
No secondary outcomes reported