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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04039477
Recruitment Status : Recruiting
First Posted : July 31, 2019
Last Update Posted : February 25, 2020
Sponsor:
Information provided by (Responsible Party):
Kezar Life Sciences, Inc.

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.

Condition or disease Intervention/treatment Phase
Autoimmune Hemolytic Anemia Immune Thrombocytopenia Drug: KZR-616 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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)
Estimated Study Start Date : March 2020
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : June 2021


Arm Intervention/treatment
Experimental: Arm A - KZR-616 30mg
KZR-616 30mg Subcutaneous (SC) injection weekly for 13 weeks
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

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




Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. 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 [ Time Frame: 13 weeks ]
  2. Mean change from Baseline over time in hematologic parameters of interest (Hgb for AIHA; PLT for ITP) [ Time Frame: Through study completion, up to 25 weeks ]
  3. Proportion of patients with a response at Week 13 [ Time Frame: 13 weeks ]
  4. Proportion of patients over time with a response [ Time Frame: Through study completion, up to 25 weeks ]
  5. Time to response [ Time Frame: Through study completion, up to 25 weeks ]
  6. Proportion of patients over time with loss of response [ Time Frame: Through study completion, up to 25 weeks ]
  7. Proportion of patients over time with sustained response [ Time Frame: Through study completion, up to 25 weeks ]
  8. Mean change from Baseline over time in Hct [ Time Frame: Through study completion, up to 25 weeks ]
  9. Mean change from Baseline over time in Lactate Dehydrogenase (LDH) [ Time Frame: Through study completion, up to 25 weeks ]
  10. Change from Baseline over time in Patient Global Assessment scores [ Time Frame: Baseline and every 4 weeks for 25 weeks ]
    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."

  11. For AIHA: Proportion of patients with an Hgb >12 g/dL or 2 g/dL higher than Baseline at W13 [ Time Frame: 13 weeks ]
  12. For AIHA: Number of blood transfusions and units of blood administered over time [ Time Frame: Through study completion, up to 25 weeks ]
  13. For ITP: Number of platelet transfusions and units of platelets administered over time [ Time Frame: Through study completion, up to 25 weeks ]
  14. Safety and tolerability of KZR-616 in patients with AIHA or ITP as assessed by monitoring incidence and severity of adverse events (AEs) [ Time Frame: Through study completion, up to 25 weeks ]
  15. Peak Plasma Concentration (Cmax) following KZR-616 injection [ Time Frame: Day 1 ]
  16. Peak Plasma Concentration (Cmax) following KZR-616 injection [ Time Frame: Day 29 ]
  17. Time to peak plasma concentration (Tmax) following KZR-616 injection [ Time Frame: Day 1 ]
  18. Time to peak plasma concentration (Tmax) following KZR-616 injection [ Time Frame: Day 29 ]
  19. Area under the plasma concentration versus time curve (AUC) following KZR-616 injection [ Time Frame: Day 1 ]
  20. Area under the plasma concentration versus time curve (AUC) following KZR-616 injection [ Time Frame: Day 29 ]
  21. Half-life (T1/2) following KZR-616 injection [ Time Frame: Day 1 ]
  22. Half-life (T1/2) following KZR-616 injection [ Time Frame: Day 29 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 AIHA, ITP, or Evans syndrome
  4. AIHA or ITP disease activity as follows::

    1. ITP: Per 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 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04039477


Contacts
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Contact: Clinical Operations (650)822-5600 clinicaltrials@kezarbio.com

Locations
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United States, California
KZR Research Site Not yet recruiting
Los Angeles, California, United States, 90007
KZR Research Site Not yet recruiting
San Francisco, California, United States, 94143
United States, Florida
KZR Research Site Recruiting
Miami Lakes, Florida, United States, 33014
KZR Research Site Recruiting
Tampa, Florida, United States, 33612
United States, Illinois
KZR Research Site Not yet recruiting
Peoria, Illinois, United States, 61615
United States, Massachusetts
KZR Research Site Recruiting
Boston, Massachusetts, United States, 02114
United States, Minnesota
KZR Research Site Not yet recruiting
Minneapolis, Minnesota, United States, 55454
KZR-616 Research Site Not yet recruiting
Rochester, Minnesota, United States, 55455
United States, New Jersey
KZR Research Site Not yet recruiting
Morristown, New Jersey, United States, 07960
United States, New York
KZR Research Site Recruiting
Bronx, New York, United States, 10467
United States, North Carolina
KZR Research Site Not yet recruiting
Greenville, North Carolina, United States, 27834
United States, Ohio
KZR Research Site Not yet recruiting
Cleveland, Ohio, United States, 44195
KZR Research Site Recruiting
Columbus, Ohio, United States, 43210
United States, Texas
KZR Research Site Recruiting
Webster, Texas, United States, 77598
Italy
KZR Research Site Not yet recruiting
Bologna, Italy
KZR Research Site Not yet recruiting
Genova, Italy
Poland
KZR Research Site Not yet recruiting
Kraków, Poland
KZR Research Site Not yet recruiting
Poznań, Poland
Russian Federation
KZR Research Site Not yet recruiting
Moscow, Russian Federation
KZR Research Site Not yet recruiting
Saint Petersburg, Russian Federation
Sponsors and Collaborators
Kezar Life Sciences, Inc.
Investigators
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Study Director: Kezar Kezar Life Sciences, Inc.

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Responsible Party: Kezar Life Sciences, Inc.
ClinicalTrials.gov Identifier: NCT04039477    
Other Study ID Numbers: KZR-616-005
First Posted: July 31, 2019    Key Record Dates
Last Update Posted: February 25, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Kezar Life Sciences, Inc.:
Autoimmune
Hemolytic
Anemia
Autoimmune Hemolytic Anemia
AIHA
Immune
Thrombocytopenia
Immune Thrombocytopenia
ITP
Blood disorders
Hematology
Additional relevant MeSH terms:
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Anemia
Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hemolysis
Hematologic Diseases
Blood Platelet Disorders
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms