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First in Human Study: LIS1, an Induction Treatment in Kidney Transplanted Patients

Phase 1
Completed
Conditions
Kidney Transplant
Interventions
Biological: LIS1
Registration Number
NCT04431219
Lead Sponsor
Xenothera SAS
Brief Summary

This first in human study aims at evaluating LIS1, a stabilized solution of purified anti-T lymphocytes polyclonal glyco-humanized swine IgG with immunosuppressive activity, in regards of safety, T cell depletion, and pharmacokinetics / pharmacodynamics in 10 kidney transplant recipients.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
10
Inclusion Criteria
  • Participants must be listed for kidney transplantation,
  • AD cohort participants: First transplantation, Panel Reactive Antibody (PRA) < 20%, negative Donor Specific Antibody (DSA), no anti-HLA antibodies, Epstein-Barr Virus positive (EBV+) serology,
  • TD cohort participants: First transplantation, 0-50 % PRA, negative DSA, negative flow cytometry crossmatch (FCXM) for any patients with anti-HLA antibodies on screening is mandatory, Epstein-Barr Virus positive (EBV+) serology
  • Participants must weigh at least 50 kg and have a Body Mass Index (BMI) 18.0 ≤ BMI < 35.0 kg/m2,
  • White Blood Cells > 3000/mm3, platelets > 75000/mm3,
  • Female participants (WOCBP) must have a negative pregnancy test at screening and use a highly effective birth control until 90 days after the last administration of study drug,
  • Non-vasectomized male subjects having a female partner of childbearing potential must agree to the use of a highly effective method of contraception until 90 days after the last administration of study drug,
  • Participants must be capable of giving signed informed consent.
Exclusion Criteria
  • Patients with an active cancer or a history of kidney cancer,
  • Patients who have previously been exposed to other anti-lymphocyte globulins,
  • Patients with previous organ transplantation,
  • Patients with a history of specific viral infection that would contraindicate depleting antibody therapy (Hepatitis B and C, HIV),
  • Patients with a positive HIV and/or Hepatitis B and C tests
  • Patients who have uncontrolled concomitant bacterial or viral infections (unresolved during screening), mycosis and/or parasitosis,
  • Patients with a significant liver function impairment: enzyme (AST and/or ALT) values must not exceed 1.5 times upper limit of normal,
  • Patients with positive testing for tuberculosis (using QuantiFERON-TB test), Patients with CMV D+/R- constellation at transplant,
  • Patients with seronegative EBV prior to transplantation,
  • Patients who have previously been exposed to antibodies of swine origin,
  • Expanded Criteria Donor (ECD) defined as donor older than 60 years,
  • Participants who have participated in another research study involving an investigational product in the previous 3 months,
  • Patients with cardiovascular or severe respiratory comorbidities (severe chronic respiratory failure, severe pulmonary fibrosis, obesity-ventilation syndrome, severe idiopathic pulmonary arterial hypertension) not allowing general anesthesia,
  • Patients with type 1 diabetes,
  • Participants who are pregnant, breast feeding or planning pregnancy during the study,
  • Participants who have any form of substance abuse (drug, alcohol...), any other health abnormalities (psychiatric disorders) or condition that according to the investigator's opinion might endanger patient during his/her participation in the study.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Ascending Dose CohortLIS1The AD cohort will be first recruited and will include 5 patients: 1 patient per dose, sequentially recruited, the recruitment of the next dose level patient will be assessed by Data Safety Monitoring Board : * Patient 1: 0.6 mg/Kg/day * Patient 2: 1 mg/Kg/day * Patient 3: 3 mg/Kg/day * Patient 4: 6 mg/Kg/day * Patient 5: 8 mg/Kg/day Once the 5 AD patients complete LIS1 treatment, the sponsor and the DSMB will rule on the LIS1 dose to obtain an optimal CD3+ cells depletion, with a good safety profile and will determine the therapeutic dose.
Therapeutic Dose CohortLIS1The TD cohort will be recruited once the therapeutic dose is defined. This cohort will be divided in 2 subgroups of respectively 2 and 3 patients sequentially recruited. DSMB will review the safety and efficacy profile of the first 2 patients (Subgroup1) and decide: * To continue at the same dose and recruit the next 3 patients of Subgroup 2 * To recruit the next 3 patients with a lower dose, estimated from AD as bringing efficient depletion * To recruit the next patient with a higher dose (+2 mg/kg), if the depletion is not considered satisfactory and if the safety profile is considered acceptable * To end the trial if LIS1 toxicity is too important vs its efficacy in CD3+ depletion. If the decision to increase the dose after the first two TD patients is made, an additional DSMB review will be planned after patient 8. The DSMB will decide to maintain the dose for the last 2 patients or to get back to the previous dose
Primary Outcome Measures
NameTimeMethod
Safety of the treatment with LIS1: aPTTup to 3 months after the transplant

Laboratory parameters (3): activated Partial Thromboplastin Time (aPTT, seconds).

Safety of the treatment with LIS1: Infectionup to 3 months after the transplant

Clinical safety parameters (5): Viral infections (namely Cytomegalovirus \[CMV\], BK virus) (yes/no).

Safety of the treatment with LIS1: Hospitalizationup to 3 months after the transplant

Clinical safety parameters (7): Prolonged stay in hospital for \>4 weeks (days).

Safety of the treatment with LIS1: CRPup to 3 months after the transplant

Laboratory parameters (1): C-Reactive Protein (CRP, mg/L).

Pharmacodynamics (depletion of T lymphocytes) of LIS1up to 3 months after the transplant

Absolute T lymphocyte counts (10\^9/L).

Safety of the treatment with LIS1: Graft rejectionup to 3 months after the transplant

Clinical safety parameters (4): Graft rejection (yes/no).

Safety of the treatment with LIS1: Re-admissionup to 3 months after the transplant

Clinical safety parameters (6): Re-admission after patient discharge (yes/no).

Safety of the treatment with LIS1: Body temperatureup to 3 months after the transplant

Clinical safety parameters (3): Body temperature (Celsius degrees).

Safety of the treatment with LIS1: Complete Blood Count (CBC)up to 3 months after the transplant

Laboratory parameters (4): Platelets (10\^9/L), white blood cells (10\^9/L), absolute neutrophil count (10\^9/L), absolute lymphocyte count (10\^9/L), absolute monocyte count (10\^9/L), absolute eosinophil count (10\^9/L), absolute basophil count (10\^9/L).

Safety of the treatment with LIS1: Blood pressureup to 3 months after the transplant

Clinical safety parameters (1): Systolic and diastolic blood pressure (mm Hg).

Safety of the treatment with LIS1: Pulse rateup to 3 months after the transplant

Clinical safety parameters (2): Pulse rate (beats per minute \[bpm\]) .

Safety of the treatment with LIS1: LDHup to 3 months after the transplant

Laboratory parameters (2): Lactate Dehydrogenase (LDH, µkat/L).

Secondary Outcome Measures
NameTimeMethod
Biology of LIS1 (1): electrolytes plasma concentrationup to 3 months after the transplant

Plasma biochemistry: electrolytes (Na+, K+, Cl-, Ca++, Mg++, bicarbonates plasma concentrations mmol/L)

Pharmacodynamics of LIS1 (2): cytokinesup to 3 months after the transplant

Cytokine concentration (IL6, TNFα) (ng/mL).

Biology of LIS1 (3): total plasma proteinsup to 3 months after the transplant

Plasma biochemistry: Total protein plasma concentration (g/L)

Immunogenicity of LIS1up to 3 months after the transplant

Detection of antidrug antibodies in serum

Pharmacokinetics of LIS1 (1): swine IgGup to 3 months after the transplant

Serum concentration of swine IgG

Biology of LIS1 (2): urea and creatinineup to 3 months after the transplant

Plasma biochemistry: urea and creatinine plasma concentration (mmol/L)

Biology of LIS1 (4): plasmatic proteinsup to 3 months after the transplant

Plasma biochemistry: electrophoresis of plasmatic proteins (percentages of albumin, alpha-1-globulin, alpha-2-globulin, beta-globulin, gamma-globulin)

Trial Locations

Locations (1)

Institut klinické a experimentální medicíny

🇨🇿

Praha 4, Czechia

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