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Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT04039477
Other study ID # KZR-616-005
Secondary ID
Status Withdrawn
Phase Phase 2
First received
Last updated
Start date July 2020
Est. completion date August 5, 2020

Study information

Verified date August 2020
Source Kezar Life Sciences, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Withdrawn
Enrollment 0
Est. completion date August 5, 2020
Est. primary completion date August 5, 2020
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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 <lower limit of normal (LLN) ii. Corrected reticulocyte count >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

Study Design


Intervention

Drug:
KZR-616
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

Locations

Country Name City State
Australia KZR Research Site Woolloongabba
Italy KZR Research Site Bologna
Italy KZR Research Site Genova
Poland KZR Research Site Kraków
Poland KZR Research Site Poznan
Russian Federation KZR Research Site Moscow
Russian Federation KZR Research Site Saint Petersburg
United States KZR Research Site Boston Massachusetts
United States KZR Research Site Bronx New York
United States KZR Research Site Cleveland Ohio
United States KZR Research Site Columbus Ohio
United States KZR Research Site Greenville North Carolina
United States KZR Research Site Jacksonville Florida
United States KZR Research Site Los Angeles California
United States KZR Research Site Miami Lakes Florida
United States KZR Research Site Minneapolis Minnesota
United States KZR Research Site Morristown New Jersey
United States KZR Research Site Peoria Illinois
United States KZR-616 Research Site Rochester Minnesota
United States KZR Research Site San Francisco California
United States KZR Research Site Tampa Florida
United States KZR Research Site Webster Texas

Sponsors (1)

Lead Sponsor Collaborator
Kezar Life Sciences, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Italy,  Poland,  Russian Federation, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 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
Secondary Mean change from Baseline over time in hematologic parameters of interest (Hgb for AIHA; PLT for ITP) Through study completion, up to 25 weeks
Secondary Proportion of patients with a response at Week 13 13 weeks
Secondary Proportion of patients over time with a response Through study completion, up to 25 weeks
Secondary Time to response Through study completion, up to 25 weeks
Secondary Proportion of patients over time with loss of response Through study completion, up to 25 weeks
Secondary Proportion of patients over time with sustained response Through study completion, up to 25 weeks
Secondary Mean change from Baseline over time in Hct Through study completion, up to 25 weeks
Secondary Mean change from Baseline over time in Lactate Dehydrogenase (LDH) Through study completion, up to 25 weeks
Secondary Change from Baseline over time in Patient Global Assessment scores The PtGA is a visual analog scale (VAS) ranging from 0 to 100. Patients will provide a global rating of their disease, for the day of the visit, in response to the statement "Considering all the ways that your disease affects you, please rate how you are feeling today by clicking or tapping on the line:" using a 100-point VAS where 0 is "very good, no symptoms" and 100 is "very poor, very severe symptoms." Baseline and every 4 weeks for 25 weeks
Secondary For AIHA: Proportion of patients with an Hgb >12 g/dL or 2 g/dL higher than Baseline at W13 13 weeks
Secondary For AIHA: Number of blood transfusions and units of blood administered over time Through study completion, up to 25 weeks
Secondary For ITP: Number of platelet transfusions and units of platelets administered over time Through study completion, up to 25 weeks
Secondary 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
Secondary Peak Plasma Concentration (Cmax) following KZR-616 injection Day 1
Secondary Peak Plasma Concentration (Cmax) following KZR-616 injection Day 29
Secondary Time to peak plasma concentration (Tmax) following KZR-616 injection Day 1
Secondary Time to peak plasma concentration (Tmax) following KZR-616 injection Day 29
Secondary Area under the plasma concentration versus time curve (AUC) following KZR-616 injection Day 1
Secondary Area under the plasma concentration versus time curve (AUC) following KZR-616 injection Day 29
Secondary Half-life (T1/2) following KZR-616 injection Day 1
Secondary Half-life (T1/2) following KZR-616 injection Day 29
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