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Autoimmune Hemolytic AnemiaPhase 2MARINANCT04039477

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

A Phase 2 study testing KZR-616 in people with Autoimmune Hemolytic Anemia and Immune Thrombocytopenia, run by Kezar Life Sciences, Inc. at 22 sites in 5 countries.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
KZR-616
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
22 sitesin 5 countries
Study dates
Jul 2020to 5 Aug 2020
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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:
  • 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

Who cannot join

  • 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:
Show all 18Show fewer
  • 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
  • History of any thrombotic or embolic event within 12 months prior to Screening
  • Clinical evidence of significant unstable or uncontrolled diseases
  • 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

Trial locations and contacts

22 sites in 5 countries.

22 sites in 5 countries

United States15 sites

  • KZR Research Site

    Boston, Massachusetts, 02114

  • KZR Research Site

    Cleveland, Ohio, 44195

  • KZR Research Site

    Columbus, Ohio, 43210

  • KZR Research Site

    Greenville, North Carolina, 27834

  • KZR Research Site

    Jacksonville, Florida, 32224

  • KZR Research Site

    Los Angeles, California, 90007

  • KZR Research Site

    Miami Lakes, Florida, 33014

  • KZR Research Site

    Minneapolis, Minnesota, 55454

  • KZR Research Site

    Morristown, New Jersey, 07960

  • KZR Research Site

    Peoria, Illinois, 61615

  • KZR-616 Research Site

    Rochester, Minnesota, 55455

  • KZR Research Site

    San Francisco, California, 94143

  • KZR Research Site

    Tampa, Florida, 33612

  • KZR Research Site

    The Bronx, New York, 10467

  • KZR Research Site

    Webster, Texas, 77598

Other trials in United States

Italy2 sites

  • KZR Research Site

    Bologna

  • KZR Research Site

    Genova

Other trials in Italy

Poland2 sites

  • KZR Research Site

    Krakow

  • KZR Research Site

    Poznan

Other trials in Poland

Russia2 sites

  • KZR Research Site

    Moscow

  • KZR Research Site

    Saint Petersburg

Other trials in Russia

Australia1 site

  • KZR Research Site

    Woolloongabba

Other trials in Australia

About this study

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)

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.

Being tested
KZR-616
Keywords
Autoimmune, Hemolytic, Anemia, Autoimmune Hemolytic Anemia, AIHA, Immune, Thrombocytopenia, Immune Thrombocytopenia, ITP, Blood disorders, Hematology
Medical subject headings
Anemia, Hemolytic, Autoimmune, Purpura, Thrombocytopenic, Idiopathic, Hemolysis, Anemia, Thrombocytopenia, Hematologic Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • KZR-616Drug

    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

Study groups in NCT04039477
Study groupRoleWhat they receive
Arm A - KZR-616 30mgExperimentalKZR-616 30mg Subcutaneous (SC) injection weekly for 13 weeks
Receives: Drug: KZR-616
Arm B - KZR-616 45mgExperimentalKZR-616 30 mg SC injection weekly for 1 dose then 45mg weekly for 12 weeks.
Receives: Drug: KZR-616

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: 13 weeks

Secondary outcomes (22)

  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

    Measured: 13 weeks

  2. Mean change from Baseline over time in hematologic parameters of interest (Hgb for AIHA; PLT for ITP)

    Measured: Through study completion, up to 25 weeks

  3. Proportion of patients with a response at Week 13

    Measured: 13 weeks

  4. Proportion of patients over time with a response

    Measured: Through study completion, up to 25 weeks

  5. Time to response

    Measured: Through study completion, up to 25 weeks

  6. Proportion of patients over time with loss of response

    Measured: Through study completion, up to 25 weeks

  7. Proportion of patients over time with sustained response

    Measured: Through study completion, up to 25 weeks

  8. Mean change from Baseline over time in Hct

    Measured: Through study completion, up to 25 weeks

  9. Mean change from Baseline over time in Lactate Dehydrogenase (LDH)

    Measured: Through study completion, up to 25 weeks

  10. 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."

    Measured: Baseline and every 4 weeks for 25 weeks

  11. For AIHA: Proportion of patients with an Hgb >12 g/dL or 2 g/dL higher than Baseline at W13

    Measured: 13 weeks

  12. For AIHA: Number of blood transfusions and units of blood administered over time

    Measured: Through study completion, up to 25 weeks

  13. For ITP: Number of platelet transfusions and units of platelets administered over time

    Measured: 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)

    Measured: Through study completion, up to 25 weeks

  15. Peak Plasma Concentration (Cmax) following KZR-616 injection

    Measured: Day 1

  16. Peak Plasma Concentration (Cmax) following KZR-616 injection

    Measured: Day 29

  17. Time to peak plasma concentration (Tmax) following KZR-616 injection

    Measured: Day 1

  18. Time to peak plasma concentration (Tmax) following KZR-616 injection

    Measured: Day 29

  19. Area under the plasma concentration versus time curve (AUC) following KZR-616 injection

    Measured: Day 1

  20. Area under the plasma concentration versus time curve (AUC) following KZR-616 injection

    Measured: Day 29

  21. Half-life (T1/2) following KZR-616 injection

    Measured: Day 1

  22. Half-life (T1/2) following KZR-616 injection

    Measured: Day 29

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Jul 2019
  2. Study startJul 2020 (estimated)
  3. Primary completion5 Aug 2020
  4. Study completion5 Aug 2020

Why it stopped: Clinical trial activity slowdown due to COVID-19 pandemic, high screen fail rate, and lack of enrollment led to the decision to withdraw the MARINA study.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04039477
Sponsor's protocol ID
KZR-616-005
Acronym
MARINA
Status last verified
Aug 2020
First submitted
26 Jul 2019
Last updated
7 Aug 2020
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing KZR-616 in people with Autoimmune Hemolytic Anemia and Immune Thrombocytopenia, run by Kezar Life Sciences, Inc. at 22 sites in 5 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 7 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in United States, Italy, Poland, Russia, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04039477.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2020.

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