Overview

A Phase 2 Study to Evaluate the Safety and Efficacy of KZR-616 in Patients With AIHA and ITP

Status:
Withdrawn

Trial end date:
2020-08-05

Target enrollment:
0

Participant gender:
All

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.

Phase:

Phase 2

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Kezar Life Sciences, Inc.

Criteria

Inclusion Criteria:

1. Adult patients must be at least 18 years of age at the time of signing informed
consent at Screening

2. Body Mass Index (BMI) equal to or greater than 18 kg/m2

3. Have a documented diagnosis of primary or secondary AIHA, ITP, or primary Evans
syndrome

4. AIHA or ITP disease activity as follows::

1. 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

2. AIHA: Hgb ≤10 g/dL and presence of any 2 of the following:

i. Haptoglobin upper
limit of normal (ULN) iii. LDH >ULN iv. Indirect bilirubin >ULN.

5. Documented inadequate response on intolerance to ≥1 standard treatment approach for
AIHA or ≥2 standard treatment approaches for ITP

Exclusion Criteria:

1. 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

2. History of clinically significant coagulopathy, hereditary thrombocytopenia, anemia,
or family history of thrombocytopenia

3. History of primary immunodeficiency

4. Use of nonpermitted medications within the specified washout periods prior to
screening

5. Recent serious or ongoing infection, or risk for serious infection

6. Any of the following laboratory values at Screening:

1. Estimated glomerular filtration rate (eGFR) <45 ml/min

2. Absolute neutrophil count (ANC) <1.5×109/L (1500/mm3)

3. Serum aspartate transaminase (AST), serum alanine transaminase (ALT) or serum
alkaline phosphatase >2.5×ULN

4. Thyroid stimulating hormone if outside of the central laboratory normal range and
considered clinically significant

5. International normalized ratio (INR) or activated partial thromboplastin time
(aPTT) >1.5×ULN

6. Immunoglobulin G (IgG) <500 mg/dL

7. For ITP patients only: total bilirubin >1.5×ULN (3×ULN for patients with
documented Gilbert's syndrome).

7. Presence of New York Heart Association Class III or IV heart failure, or uncontrolled
blood pressure, or prolonged QT interval

8. Major surgery within 12 weeks before Screening or planned during the study period

9. History of any thrombotic or embolic event within 12 months prior to Screening

10. Clinical evidence of significant unstable or uncontrolled diseases

11. Any active or suspected malignancy or history of documented malignancy within the last
5 years before Screening, except appropriately excised and cured cervical carcinoma in
situ or basal or squamous cell carcinoma of the skin, or non-muscle invasive bladder
cancer