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GNR-084 Safety and Pharmacological Characteristics in Refractory or Relapse B-cell Precursor ALL

Phase 1
Recruiting
Conditions
ALL
GNR-084
B-precursor Acute Lymphoblastic Leukemia
Interventions
Biological: Cohort 1, GNR-084
Biological: Cohort 2, GNR-084
Biological: Cohort 5, GNR-084
Biological: Cohort 6, GNR-084
Biological: Cohort 3, GNR-084
Biological: Cohort 4, GNR-084
Registration Number
NCT04601584
Lead Sponsor
AO GENERIUM
Brief Summary

It is an open-label dose-escalating study in sequential cohorts to assess safety and pharmacokinetics of GNR-084.

Detailed Description

Acute lymphoblastic leukemias (ALL) are a heterogeneous group of malignant clonal diseases of the blood system originating from precursor cells of hematopoiesis, predominantly of lymphoid differentiation.

More than 7,200 new cases of ALL are diagnosed annually in the European Union (EU), with approximately 40% (approximately 3,000 cases) occurring in adults The main reason for the failure of treatment of acute B-cell lymphoblastic leukemias (B-ALL) is the primary refractoriness to chemical exposure and relapses of the disease, which actually occur in 40-50% of adult patients with ALL. The prognosis in these cases is regarded as extremely unfavorable. Escalation of the chemotherapeutic approach is associated with the development of severe toxic infectious and hemorrhagic complications.

The active substance of the preparation GNR-084 is a bispecific antibody to CD19 / CD3 in the BiMS format (bispecific IgG-like molecules).

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
36
Inclusion Criteria
  1. Voluntarily signed informed consent form to participate in the study;
  2. Men and women between aged 18 to 45 inclusive;
  3. Patients with incurable morphologically / immunophenotypically confirmed refractory/ relapse of B-cell precursors CD19-positive acute lymphoblastic leukemia from (Ph "-" or Ph "+").
  4. Two or more previous lines of anti-leucosis therapy.
  5. 5-50% of bone marrow blast cells at screening;
  6. Functional status on the scale of the Eastern Cooperative Oncology Group (ECOG) 0-2 points at the screening;
  7. Life expectancy ≥ 60 days;
Exclusion Criteria
  1. Hematopoietic stem cells transplantation within 12 weeks prior to study inclusion;

  2. Active and widespread chronic graft versus host (GVHD) reaction (grade II-IV), including taking immunosuppressants for the prevention and treatment of GVHD within 2 weeks prior the GNR-084 infusion;

  3. Investigator and / or sponsor has doubts that patient will complete the study due to rapid disease progression;

  4. Chemotherapeutic agent using within 14 days prior the first GNR-084 infusion;

    Exceptions:

    • Emergency leukapheresis;
    • Emergency hydroxyurea using due to hyperleukocytosis for ≤ 7 days;
    • Other supportive care, including antibiotics, at Investigator's discretion
  5. Biochemical blood test:

    • The level of total bilirubin> 1.5 upper limit of norm;
    • Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT)> 3 upper limit of norm;
    • Glomerular filtration rate (GFR) level ≤30 (СKD-EPI)
  6. Medical history of blinatumomab and other bispecific antibodies using;

  7. Persistent toxicity event of 3rd and 4th severity degrees (CTCAE ver 5.0) due to previous treatment;

  8. HIV-positive status and / or detection of any hepatitis B and / or hepatitis C blood markers;

  9. Severe cardiovascular diseases: uncontrolled arterial hypertension, New York Heart Association (NYHA) functional class III or IV chronic heart failure, unstable angina pectoris, stroke, myocardial infarction, transient ischemic attack, coronary artery bypass grafting and coronary revascularization within last 12 months, or signs of pericardial effusion;

  10. Individual sensitivity to:

    • GNR-084 components / excipients;
    • human or humanized investigational drug antibodies;
  11. Major surgical interventions, accompanied by hospitalization and anesthesia application within 30 days before the patient is included in the study (biopsy is not a significant surgical intervention);

  12. Any other malignant neoplasm presence at the present time or within 5 years prior to inclusion in the study;

  13. Known suspected Central Nervous System (CNS) lesion by any genesis now or in medical history, including, but not limited to: neuroleukemia, epilepsy, ischemic or hemorrhagic stroke, severe traumatic brain injury, dementia, Parkinson's disease, organic brain damage, cerebellar disorders, psychosis;

  14. Extramedullary lesion of any localization;

  15. Other clinical trials participation within 30 days before screening;

  16. Mental, physical and other reasons hindering patient to adequately assess their behavior and correctly comply with the conditions of the research protocol;

  17. Pregnancy and / or lactation;

  18. Male and female patients refusal to use adequate methods of contraception throughout the study;

  19. Drug addiction;

  20. Alcohol addiction.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
GNR-084, dose level 1Cohort 1, GNR-084Anti-CD19/CD3 antibody
GNR-084, dose level 2Cohort 2, GNR-084Anti-CD19/CD3 antibody
GNR-084, dose level 5Cohort 5, GNR-084Anti-CD19/CD3 antibody
GNR-084, dose level 6Cohort 6, GNR-084Anti-CD19/CD3 antibody
GNR-084, dose level 3Cohort 3, GNR-084Anti-CD19/CD3 antibody
GNR-084, dose level 4Cohort 4, GNR-084Anti-CD19/CD3 antibody
Primary Outcome Measures
NameTimeMethod
GNR-084 safety and tolerability.Week 10

The GNR-084 safety and tolerability will be assessed based on an analysis of the frequency of adverse events (AEs) over the period of treatment and observation of patients

Secondary Outcome Measures
NameTimeMethod
Complete clinical and hematological remission rate (CR)After 2 and 5 GNR-084 cycles (each cycle is 28 days)
Frequency of complete remission with incomplete restoration of blood cellularity (CRi)After 2 and 5 GNR-084 cycles (each cycle is 28 days)
Event-free survival (EFS)Week 104
Overall survival (OS)Week 104
GNR-084 Peak Plasma Concentration (Cmax)First infusion: 5 minutes before administration, immediately after infusion, 30 minutes, 1, 3, 6, 12, 18, 24, 48, 72, 96 hours after infusion.
GNR-084 area under the plasma concentration versus time curve (AUC)First infusion: 5 minutes before administration, immediately after infusion, 30 minutes, 1, 3, 6, 12, 18, 24, 48, 72, 96 hours after infusion.
GNR-084 elimination rate constant (Kel)First infusion: 5 minutes before administration, immediately after infusion, 30 minutes, 1, 3, 6, 12, 18, 24, 48, 72, 96 hours after infusion.
GNR-084 overall clearance (Cl)First infusion: 5 minutes before administration, immediately after infusion, 30 minutes, 1, 3, 6, 12, 18, 24, 48, 72, 96 hours after infusion.
Peripheral B-cells/T-cells ratioFirst infusion: 5 minutes before administration, 30 minutes, 1, 24, 48, 96 and 144 hours after infusion. Other infusions: 5 minutes before administration and 1 hour after infusion
ImmunogenicityWeek 33
GNR-084 mean retention time (MRT)First infusion: 5 minutes before administration, immediately after infusion, 30 minutes, 1, 3, 6, 12, 18, 24, 48, 72, 96 hours after infusion.
CD45+ peripheral cell countFirst infusion: 5 minutes before administration, 30 minutes, 1, 24, 48, 96 and 144 hours after infusion. Other infusions: 5 minutes before administration and 1 hour after infusion
Peripheral T-lymphocytes count (CD3, CD4, CD8)First infusion: 5 minutes before administration, 30 minutes, 1, 24, 48, 96 and 144 hours after infusion. Other infusions: 5 minutes before administration and 1 hour after infusion
Peripheral T-memory cells (CD45RA+, CD28+, CCR7+) countFirst infusion: 5 minutes before administration, 30 minutes, 1, 24, 48, 96 and 144 hours after infusion. Other infusions: 5 minutes before administration and 1 hour after infusion
Cytokine dynamicsFirst infusion: 5 minutes before administration, 30 minutes, 1, 24, 48, 96 and 144 hours after infusion. Other infusions: 5 minutes before administration and 1 hour after infusion
The frequency of specific toxicity eventsWeek 104
GNR-84 half-life (T1/2)First infusion: 5 minutes before administration, immediately after infusion, 30 minutes, 1, 3, 6, 12, 18, 24, 48, 72, 96 hours after infusion.
GNR-084 kinetic volume of distribution (Vz)First infusion: 5 minutes before administration, immediately after infusion, 30 minutes, 1, 3, 6, 12, 18, 24, 48, 72, 96 hours after infusion.
Peripheral blood B-lymphocyte depletion (CD19, CD20).First infusion: 5 minutes before administration, 30 minutes, 1, 24, 48, 96 and 144 hours after infusion. Other infusions: 5 minutes before administration and 1 hour after infusion
Objective response rate (ORR)After 2 and 5 GNR-084 cycles (each cycle is 28 days)
Duration of an objective response (DoR)Week 104
Relapse-free survival (RFS)Week 104
Minimal residual disease (MRD) (-) rate in CR-patientAfter 5 GNR-084 cycles (each cycle is 28 days)

Trial Locations

Locations (3)

Federal State Budget Funded Institution National Medical Research Center of Hematology, Ministry of Health of the Russian Federation (MoH of Russia)

🇷🇺

Moscow, Russian Federation

Almazov National Medical Research Centre

🇷🇺

Saint Petersburg, Russian Federation

Pavlov First Saint Petersburg State Medical University

🇷🇺

Saint Petersburg, Russian Federation

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