A Phase 2 Randomized, Dose-blind, Multicenter Study to Evaluate the Safety and Efficacy of KZR-616 in the Treatment of Patients With Autoimmune Hemolytic Anemia (AIHA) and Immune Thrombocytopenia (ITP)

Kezar Life Sciences, Inc.2 sites in 2 countriesJuly 2020

ConditionsAutoimmune Hemolytic Anemia Immune Thrombocytopenia

InterventionsKZR-616

DrugsKZR-616

Overview

Phase: Phase 2
Intervention: KZR-616
Conditions: Autoimmune Hemolytic Anemia
Sponsor: Kezar Life Sciences, Inc.
Locations: 2
Primary Endpoint: Mean change from Baseline to Week 13 in hematologic parameters of interest in evaluable patients (Hgb for AIHA; Platelets [PLT] for ITP)
Status: Withdrawn
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a Phase 2 randomized, dose-blind, multicenter study designed to evaluate the safety, tolerability, efficacy, Pharmacokinetics (PK), and Pharmacodynamics (PD) of treatment with KZR-616 in patients with active Autoimmune Hemolytic Anemia or Immune Thrombocytopenia.

Registry

clinicaltrials.gov

Start Date

July 2020

End Date

August 5, 2020

Last Updated

5 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Kezar Life Sciences, Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Adult patients must be at least 18 years of age at the time of signing informed consent at Screening
•Body Mass Index (BMI) equal to or greater than 18 kg/m2
•Have a documented diagnosis of primary or secondary AIHA, ITP, or primary Evans syndrome
•AIHA or ITP disease activity as follows::
•ITP: Per central or local laboratory assessments on 2 separate occasions ≥7 days apart during Screening, a mean Platelet (PLT) ≤30×109/L with no individual PLT \>35×109/L; or for those patients receiving a constant dose of permitted treatments for ITP: a mean PLT \<50×109/L, with no count \>55×109/L
•AIHA: Hgb ≤10 g/dL and presence of any 2 of the following:
•i. Haptoglobin \<lower limit of normal (LLN) ii. Corrected reticulocyte count \>upper limit of normal (ULN) iii. LDH \>ULN iv. Indirect bilirubin \>ULN.
•Documented inadequate response on intolerance to ≥1 standard treatment approach for AIHA or ≥2 standard treatment approaches for ITP

Exclusion Criteria

•Systemic Lupus Erythematosus (SLE) with confirmed anti-phospholipid antibody syndrome, the presence of positive lupus anti-coagulant test, moderate-high titer anti-cardiolipin IgG or IgM or moderate-high titer anti-beta2-globuilin IgG or IgM or severe central nervous system involvement
•History of clinically significant coagulopathy, hereditary thrombocytopenia, anemia, or family history of thrombocytopenia
•History of primary immunodeficiency
•Use of nonpermitted medications within the specified washout periods prior to screening
•Recent serious or ongoing infection, or risk for serious infection
•Any of the following laboratory values at Screening:
•Estimated glomerular filtration rate (eGFR) \<45 ml/min
•Absolute neutrophil count (ANC) \<1.5×109/L (1500/mm3)
•Serum aspartate transaminase (AST), serum alanine transaminase (ALT) or serum alkaline phosphatase \>2.5×ULN
•Thyroid stimulating hormone if outside of the central laboratory normal range and considered clinically significant

Arms & Interventions

Arm A - KZR-616 30mg

KZR-616 30mg Subcutaneous (SC) injection weekly for 13 weeks

Intervention: KZR-616

Arm B - KZR-616 45mg

KZR-616 30 mg SC injection weekly for 1 dose then 45mg weekly for 12 weeks.

Intervention: KZR-616

Outcomes

Primary Outcomes

Mean change from Baseline to Week 13 in hematologic parameters of interest in evaluable patients (Hgb for AIHA; Platelets [PLT] for ITP)

Time Frame: 13 weeks

Secondary Outcomes

Mean change from Baseline to Week 13 in hematologic parameters of interest (Hgb for AIHA; PLT for ITP) in the intent to treat (ITT) population(13 weeks)
Proportion of patients over time with a response(Through study completion, up to 25 weeks)
Mean change from Baseline over time in hematologic parameters of interest (Hgb for AIHA; PLT for ITP)(Through study completion, up to 25 weeks)
Proportion of patients over time with loss of response(Through study completion, up to 25 weeks)
Mean change from Baseline over time in Hct(Through study completion, up to 25 weeks)
For AIHA: Proportion of patients with an Hgb >12 g/dL or 2 g/dL higher than Baseline at W13(13 weeks)
For AIHA: Number of blood transfusions and units of blood administered over time(Through study completion, up to 25 weeks)
Safety and tolerability of KZR-616 in patients with AIHA or ITP as assessed by monitoring incidence and severity of adverse events (AEs)(Through study completion, up to 25 weeks)
Time to peak plasma concentration (Tmax) following KZR-616 injection(Day 29)
Proportion of patients with a response at Week 13(13 weeks)
Time to response(Through study completion, up to 25 weeks)
Proportion of patients over time with sustained response(Through study completion, up to 25 weeks)
Mean change from Baseline over time in Lactate Dehydrogenase (LDH)(Through study completion, up to 25 weeks)
Change from Baseline over time in Patient Global Assessment scores(Baseline and every 4 weeks for 25 weeks)
For ITP: Number of platelet transfusions and units of platelets administered over time(Through study completion, up to 25 weeks)
Peak Plasma Concentration (Cmax) following KZR-616 injection(Day 29)
Area under the plasma concentration versus time curve (AUC) following KZR-616 injection(Day 29)
Half-life (T1/2) following KZR-616 injection(Day 29)