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

August 5, 2020 updated by: Kezar Life Sciences, Inc.

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)

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Woolloongabba, Australia
        • KZR Research Site
  • Italy
      • Bologna, Italy
        • KZR Research Site
      • Genova, Italy
        • KZR Research Site
  • Poland
      • Kraków, Poland
        • KZR Research Site
      • Poznań, Poland
        • KZR Research Site
  • Russian Federation
      • Moscow, Russian Federation
        • KZR Research Site
      • Saint Petersburg, Russian Federation
        • KZR Research Site
  • United States
    • California
      • Los Angeles, California, United States, 90007
        • KZR Research Site
      • San Francisco, California, United States, 94143
        • KZR Research Site
    • Florida
      • Jacksonville, Florida, United States, 32224
        • KZR Research Site
      • Miami Lakes, Florida, United States, 33014
        • KZR Research Site
      • Tampa, Florida, United States, 33612
        • KZR Research Site
    • Illinois
      • Peoria, Illinois, United States, 61615
        • KZR Research Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • KZR Research Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • KZR Research Site
      • Rochester, Minnesota, United States, 55455
        • KZR-616 Research Site
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • KZR Research Site
    • New York
      • Bronx, New York, United States, 10467
        • KZR Research Site
    • North Carolina
      • Greenville, North Carolina, United States, 27834
        • KZR Research Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • KZR Research Site
      • Columbus, Ohio, United States, 43210
        • KZR Research Site
    • Texas
      • Webster, Texas, United States, 77598
        • KZR Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: QUADRUPLE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
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
13 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Time to peak plasma concentration (Tmax) following KZR-616 injection
Time Frame: Day 1
Day 1
Area under the plasma concentration versus time curve (AUC) following KZR-616 injection
Time Frame: Day 1
Day 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
13 weeks
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
Through study completion, up to 25 weeks
Proportion of patients with a response at Week 13
Time Frame: 13 weeks
13 weeks
Proportion of patients over time with a response
Time Frame: Through study completion, up to 25 weeks
Through study completion, up to 25 weeks
Time to response
Time Frame: Through study completion, up to 25 weeks
Through study completion, up to 25 weeks
Proportion of patients over time with loss of response
Time Frame: Through study completion, up to 25 weeks
Through study completion, up to 25 weeks
Proportion of patients over time with sustained response
Time Frame: Through study completion, up to 25 weeks
Through study completion, up to 25 weeks
Mean change from Baseline over time in Hct
Time Frame: Through study completion, up to 25 weeks
Through study completion, up to 25 weeks
Mean change from Baseline over time in Lactate Dehydrogenase (LDH)
Time Frame: Through study completion, up to 25 weeks
Through study completion, up to 25 weeks
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."
Baseline and every 4 weeks for 25 weeks
For AIHA: Proportion of patients with an Hgb >12 g/dL or 2 g/dL higher than Baseline at W13
Time Frame: 13 weeks
13 weeks
For AIHA: Number of blood transfusions and units of blood administered over time
Time Frame: Through study completion, up to 25 weeks
Through study completion, up to 25 weeks
For ITP: Number of platelet transfusions and units of platelets administered over time
Time Frame: Through study completion, up to 25 weeks
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)
Time Frame: Through study completion, up to 25 weeks
Through study completion, up to 25 weeks
Peak Plasma Concentration (Cmax) following KZR-616 injection
Time Frame: Day 1
Day 1
Peak Plasma Concentration (Cmax) following KZR-616 injection
Time Frame: Day 29
Day 29
Time to peak plasma concentration (Tmax) following KZR-616 injection
Time Frame: Day 29
Day 29
Area under the plasma concentration versus time curve (AUC) following KZR-616 injection
Time Frame: Day 29
Day 29
Half-life (T1/2) following KZR-616 injection
Time Frame: Day 1
Day 1
Half-life (T1/2) following KZR-616 injection
Time Frame: Day 29
Day 29

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Kezar Life Sciences, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ANTICIPATED)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2020

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 5, 2020

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 5, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 26, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 29, 2019

First Posted (ACTUAL)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 31, 2019

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 7, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 5, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • KZR-616-005

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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