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

NCT04039477 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: KZR-616-005
• Study Type: interventional
• Target: N/A
• Locations: 5 countries
• Sites: 22

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.

Conditions

Autoimmune Hemolytic Anemia; Immune Thrombocytopenia

Keywords

Autoimmune; Hemolytic; Anemia; Autoimmune Hemolytic Anemia; AIHA; Immune; Thrombocytopenia; Immune Thrombocytopenia; ITP; Blood disorders; Hematology

Outcome Measures

Primary Outcomes (1)

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

  13 weeks

Secondary Outcomes (22)

• 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

• 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 with a response at Week 13

  13 weeks

• Proportion of patients over time with a response

  Through study completion, up to 25 weeks

• Time to response

  Through study completion, up to 25 weeks

Study Arms (2)

Arm A - KZR-616 30mg

EXPERIMENTAL

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

Drug: KZR-616

Arm B - KZR-616 45mg

EXPERIMENTAL

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

Drug: KZR-616

Interventions

KZR-616 DRUG

Patients will receive KZR-616 SC once weekly. Patients assigned to Arm A will receive 30 mg for 13 weeks and patients assigned to Arm B will receive 30 mg for 1 week then 45 mg for 12 weeks

Arm A - KZR-616 30mg; Arm B - KZR-616 45mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• International normalized ratio (INR) or activated partial thromboplastin time (aPTT) \>1.5×ULN
• Immunoglobulin G (IgG) \<500 mg/dL
• For ITP patients only: total bilirubin \>1.5×ULN (3×ULN for patients with documented Gilbert's syndrome).
• Presence of New York Heart Association Class III or IV heart failure, or uncontrolled blood pressure, or prolonged QT interval
• Major surgery within 12 weeks before Screening or planned during the study period

Sponsors & Collaborators

• Kezar Life Sciences, Inc.: lead

Study Sites (22)

KZR Research Site

Los Angeles, California, 90007, United States

Location

KZR Research Site

San Francisco, California, 94143, United States

Location

KZR Research Site

Jacksonville, Florida, 32224, United States

Location

KZR Research Site

Miami Lakes, Florida, 33014, United States

Location

KZR Research Site

Tampa, Florida, 33612, United States

Location

KZR Research Site

Peoria, Illinois, 61615, United States

Location

KZR Research Site

Boston, Massachusetts, 02114, United States

Location

KZR Research Site

Minneapolis, Minnesota, 55454, United States

Location

KZR-616 Research Site

Rochester, Minnesota, 55455, United States

Location

KZR Research Site

Morristown, New Jersey, 07960, United States

Location

KZR Research Site

The Bronx, New York, 10467, United States

Location

KZR Research Site

Greenville, North Carolina, 27834, United States

Location

KZR Research Site

Cleveland, Ohio, 44195, United States

Location

KZR Research Site

Columbus, Ohio, 43210, United States

Location

KZR Research Site

Webster, Texas, 77598, United States

Location

KZR Research Site

Woolloongabba, Australia

Location

KZR Research Site

Bologna, Italy

Location

KZR Research Site

Genova, Italy

Location

KZR Research Site

Krakow, Poland

Location

KZR Research Site

Poznan, Poland

Location

KZR Research Site

Moscow, Russia

Location

KZR Research Site

Saint Petersburg, Russia

Location

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune; Purpura, Thrombocytopenic, Idiopathic; Hemolysis; Anemia; Thrombocytopenia; Hematologic Diseases

Interventions

KZR-616

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Kezar

  Kezar Life Sciences, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 26, 2019
• First Posted: July 31, 2019
• Study Start: July 1, 2020
• Primary Completion: August 5, 2020
• Study Completion: August 5, 2020
• Last Updated: August 7, 2020

Record last verified: 2020-08

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); RU (2); US (15); IT (2); PL (2)