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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04039477
Registration number
NCT04039477
Ethics application status
Date submitted
26/07/2019
Date registered
31/07/2019
Date last updated
7/08/2020
Titles & IDs
Public title
A Phase 2 Study to Evaluate the Safety and Efficacy of KZR-616 in Patients With AIHA and ITP
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Scientific 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)
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Secondary ID [1]
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KZR-616-005
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Universal Trial Number (UTN)
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Trial acronym
MARINA
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Autoimmune Hemolytic Anemia
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Immune Thrombocytopenia
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Condition category
Condition code
Blood
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Anaemia
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Blood
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Haematological diseases
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Blood
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Other blood disorders
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Inflammatory and Immune System
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Autoimmune diseases
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Blood
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Anaemia
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Blood
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Haematological diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - KZR-616
Experimental: Arm A - KZR-616 30mg - KZR-616 30mg Subcutaneous (SC) injection weekly for 13 weeks
Experimental: Arm B - KZR-616 45mg - KZR-616 30 mg SC injection weekly for 1 dose then 45mg weekly for 12 weeks.
Treatment: Drugs: 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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Mean change from Baseline to Week 13 in hematologic parameters of interest in evaluable patients (Hgb for AIHA; Platelets [PLT] for ITP)
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Assessment method [1]
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Timepoint [1]
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13 weeks
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Secondary outcome [1]
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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
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Assessment method [1]
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Timepoint [1]
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13 weeks
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Secondary outcome [2]
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Mean change from Baseline over time in hematologic parameters of interest (Hgb for AIHA; PLT for ITP)
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Assessment method [2]
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Timepoint [2]
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Through study completion, up to 25 weeks
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Secondary outcome [3]
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Proportion of patients with a response at Week 13
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Assessment method [3]
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Timepoint [3]
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13 weeks
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Secondary outcome [4]
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Proportion of patients over time with a response
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Assessment method [4]
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Timepoint [4]
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Through study completion, up to 25 weeks
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Secondary outcome [5]
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Time to response
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Assessment method [5]
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Timepoint [5]
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Through study completion, up to 25 weeks
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Secondary outcome [6]
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Proportion of patients over time with loss of response
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Assessment method [6]
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Timepoint [6]
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Through study completion, up to 25 weeks
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Secondary outcome [7]
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Proportion of patients over time with sustained response
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Assessment method [7]
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Timepoint [7]
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Through study completion, up to 25 weeks
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Secondary outcome [8]
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Mean change from Baseline over time in Hct
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Assessment method [8]
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Timepoint [8]
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Through study completion, up to 25 weeks
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Secondary outcome [9]
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Mean change from Baseline over time in Lactate Dehydrogenase (LDH)
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Assessment method [9]
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Timepoint [9]
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Through study completion, up to 25 weeks
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Secondary outcome [10]
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Change from Baseline over time in Patient Global Assessment scores
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Assessment method [10]
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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."
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Timepoint [10]
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Baseline and every 4 weeks for 25 weeks
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Secondary outcome [11]
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For AIHA: Proportion of patients with an Hgb >12 g/dL or 2 g/dL higher than Baseline at W13
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Assessment method [11]
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Timepoint [11]
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13 weeks
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Secondary outcome [12]
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For AIHA: Number of blood transfusions and units of blood administered over time
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Assessment method [12]
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Timepoint [12]
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Through study completion, up to 25 weeks
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Secondary outcome [13]
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For ITP: Number of platelet transfusions and units of platelets administered over time
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Assessment method [13]
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Timepoint [13]
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Through study completion, up to 25 weeks
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Secondary outcome [14]
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Safety and tolerability of KZR-616 in patients with AIHA or ITP as assessed by monitoring incidence and severity of adverse events (AEs)
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Assessment method [14]
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Timepoint [14]
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Through study completion, up to 25 weeks
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Secondary outcome [15]
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Peak Plasma Concentration (Cmax) following KZR-616 injection
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Assessment method [15]
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Timepoint [15]
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Day 1
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Secondary outcome [16]
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Peak Plasma Concentration (Cmax) following KZR-616 injection
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Assessment method [16]
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Timepoint [16]
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Day 29
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Secondary outcome [17]
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Time to peak plasma concentration (Tmax) following KZR-616 injection
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Assessment method [17]
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Timepoint [17]
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Day 1
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Secondary outcome [18]
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Time to peak plasma concentration (Tmax) following KZR-616 injection
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Assessment method [18]
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Timepoint [18]
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Day 29
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Secondary outcome [19]
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Area under the plasma concentration versus time curve (AUC) following KZR-616 injection
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Assessment method [19]
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Timepoint [19]
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Day 1
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Secondary outcome [20]
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Area under the plasma concentration versus time curve (AUC) following KZR-616 injection
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Assessment method [20]
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Timepoint [20]
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Day 29
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Secondary outcome [21]
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Half-life (T1/2) following KZR-616 injection
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Assessment method [21]
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Timepoint [21]
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Day 1
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Secondary outcome [22]
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Half-life (T1/2) following KZR-616 injection
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Assessment method [22]
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Timepoint [22]
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Day 29
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Eligibility
Key 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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Withdrawn
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
1/07/2020
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Actual
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
5/08/2020
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Sample size
Target
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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KZR Research Site - Woolloongabba
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Recruitment postcode(s) [1]
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- Woolloongabba
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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State/province [2]
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Florida
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United States of America
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State/province [3]
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Illinois
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Country [4]
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United States of America
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State/province [4]
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Massachusetts
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Country [5]
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United States of America
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State/province [5]
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Minnesota
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Country [6]
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United States of America
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State/province [6]
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New Jersey
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Country [7]
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United States of America
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State/province [7]
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New York
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Country [8]
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United States of America
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State/province [8]
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North Carolina
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Country [9]
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United States of America
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State/province [9]
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Ohio
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Country [10]
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United States of America
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State/province [10]
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Texas
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Country [11]
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Italy
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State/province [11]
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Bologna
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Country [12]
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Italy
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State/province [12]
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Genova
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Country [13]
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Poland
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State/province [13]
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Kraków
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Country [14]
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Poland
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State/province [14]
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Poznan
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Country [15]
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Russian Federation
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State/province [15]
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Moscow
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Country [16]
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Russian Federation
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State/province [16]
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Saint Petersburg
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Kezar Life Sciences, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04039477
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Kezar
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Address
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Kezar Life Sciences, Inc.
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04039477
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