Clinical trial NCT04331405: Allogeneic Cord Blood Cells for Adults With Severe Acute Contusion Spinal Cord Injury
Official title: Allogeneic Mononuclear Umbilical Cord Blood Systemic Infusions for Adult Patients With Severe Acute Contusion Spinal Cord Injury: Phase I Safety Study and Phase IIa Primary Efficiency Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Spinal Cord Contusion
Keywords: spinal cord injury
ASIA A/B
cell therapy
cord blood stem cells
regenerative medicine
Lead sponsor: Sklifosovsky Institute of Emergency Care
Sponsor class: OTHER_GOV
Collaborators: name: National Medical Research Center for Cardiology, Ministry of Health of Russian Federation
First posted: 2020-04-02
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 20 patients having acute contusion spinal cord injury (3 days post-trauma max) and ASIA A/B neurological deficit were divided into 2 groups: pilot group (n = 10) receiving hUCBMC treatment in addition to standard therapy and control group (n = 10) receiving standard treatment.
Primary purpose: TREATMENT

Brief summary: Study evaluates the safety and primary efficiency of systemic (i.v.) allogeneic human umbilical cord blood mononuclear cell infusions in patients with severe acute contusion spinal cord injury (ASIA A/B). 20 patients were included. Half of patients received cell therapy in addition to standard therapy, while the other half received standard therapy only.

Detailed description: Present study was prospective randomized open-label clinical study, Phases I/IIa. Randomization was performed using envelope method (50 envelopes including Cell Therapy and Standard Therapy, 25 of each).

20 patients with severe scute contusion spinal cord injury (cervical, thoracic and lumbar cone segments) were included into the study. Patients were divided into 2 groups: pilot group (n = 10) obtaining standard conservative therapy and 4 i.v. infusions of Human Allogeneic Umbilical Cord Blood Mononuclear Cells (hUCBMCs) (\~ 300 M in 100 mL) and control group (n = 10) obtaining standard conservative therapy only. Cell Therapy was initiated in patients within 3 days after SCI episode after primary surgical decompression and/or stabilization was performed. Patients inclusion decision depended on meeting the inclusion/exclusion criteria and informed consent signed.

hUCBMC samples were prepared in the specialized laboratory and transported to the hospital immediately within 2 hours. Cells infusions were performed following 3 tests for biological compatibility and potential intolerance for each patient.

All four infusion were performed during in-hospital treatment. Observational period reached 1 year after SCI. All included patients were examined daily during in-hospital treatment following 4 times within observational period (1 year).

All adverse events and potential complications were analyzed within 1 year after SCI using CTCAE v5.0 classification.

Outcome parameters were analyzed at the end of observation and examined using standard statistical instruments.

Eligibility criteria: Inclusion Criteria:

* acute spinal cord injury (first 3 days after trauma)
* contusion type of SCI (MRI-confirmed)
* Russian citizenship
* Informed consent understood and signed by the patient

Exclusion Criteria:

* systolic arterial pressure (APsyst) \> 185 mmHg OR diastolic AP (APdia) \> 105 mmHg OR indications for aggressive AP lowering using i.v. therapy
* myocardial infarction within 3 months prior to SCI
* glucose level \< 3.5 mM/L or \> 21 mM/L
* organic CNS pathology
* acute internal organs diseases requiring surgical treatment
* autoimmune diseases
* serious surgical treatment or severe trauma within 3 months prior to SCI
* pregnancy or breast feeding
* acute infections including tuberculosis, syphilis, HIV, hepatitis B and C etc.
* severe acute and chronic hematological diseases
* rare patient's blood group parameters preventing adequate hUCBMCs sample selection (e.g. Kell+, A2B group, rare phenotype etc.)
* any benign or oncological tumors (if not fully treated prior to SCI)
* inability to participate in key examination following clinic discharge
* any psychiatric diseases preventing patient from informed consent OR treatment plan understanding
* confirmed hypersensitivity and/or allergy to any component of the studied biological compound
* inability to accomplish present study protocol

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events [Safety]
timeFrame: 1 year
measure: Neurological Deficit Change [ASIA scale]
timeFrame: Change from Baseline at 1 year after SCI
measure: Quality of Life [FIM scale]
timeFrame: 1 year after SCI
Secondary outcomes:
measure: Motor Deficit Change [Muscle Strength]
timeFrame: Change from Baseline at 1 year after SCI
measure: Independent Verticalization Ability
timeFrame: 1 year after SCI
measure: Lower Extremities Spasticity Level [Modified Asworth scale]
timeFrame: 1 year after SCI
measure: Feeling of Bladder Filling
timeFrame: 1 year after SCI
measure: Urinary Inconsistence
timeFrame: 1 year after SCI
measure: Spinal Cord Conductivity [Electrophysiological assessment]
timeFrame: 1 year after SCI
Other outcomes:
Locations:
city: Moscow
country: Russian Federation
facility: N.V. Sklifosovsky Emergency Care Institute
Study officials:
References: