Trial Outcomes & Findings for Intranasal Inhalations of Bioactive Factors Produced by M2 Macrophages in Patients With Organic Brain Syndrome (NCT NCT02957123)

Results Overview

Occurrence of severe adverse events and adverse reactions (allergic, toxic, inflammatory reactions; neurological deterioration, convulsive syndrome)

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

30 participants

Primary outcome timeframe

up to 6 months after treatment

Results posted on

2021-02-18

Participant Flow

Participant milestones

Participant milestones
Measure	Intranasal Auto-M2-BFs Intranasally-Administered Bioactive Factors (BF), Produced by Autologous M2 Macrophage (auto-M2-BFs). 30 patients with organic brain syndrome will receive auto-M2-BFs as intranasal inhalations with the aerosol inhaler device (nebulizer), 2.0 mL once a day up to 30 days.
Overall Study STARTED	30
Overall Study COMPLETED	30
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Intranasal Auto-M2-BFs n=30 Participants Intranasally-Administered Bioactive Factors, Produced by Autologous M2 Macrophage (auto-M2-BFs). 30 patients with organic brain syndrome will receive auto-M2-BFs as intranasal inhalations with the aerosol inhaler device (nebulizer), 2.0 mL once a day up to 30 days.
Age, Categorical <=18 years	0 Participants n=30 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=30 Participants
Age, Categorical >=65 years	14 Participants n=30 Participants
Sex: Female, Male Female	19 Participants n=30 Participants
Sex: Female, Male Male	11 Participants n=30 Participants
Region of Enrollment Russia	30 Participants n=30 Participants

PRIMARY outcome

Timeframe: up to 6 months after treatment

Population: The number of patients with severe adverse events and adverse reactions

Occurrence of severe adverse events and adverse reactions (allergic, toxic, inflammatory reactions; neurological deterioration, convulsive syndrome)

Outcome measures

Outcome measures
Measure	Intranasal Auto-M2-BFs n=30 Participants Intranasally-Administered Bioactive Factors, Produced by Autologous M2 Macrophage (auto-M2-BFs). M2 macrophages were generated in vitro from peripheral blood of patients during 7 days.Cell-free culture medium, containing auto-M2-BFs, was collected and aliquots of 2 mL/vial were cryopreserved. 30 patients with organic brain syndrome will receive their first doses (n=2-3) of auto-M2-BFs in clinic and wait 2 hrs to determine any short-time adverse effects of inhaled dose. The subsequent course of intranasal inhalations (once a day up to 30 days) performed as outpatient treatment. Intranasal auto-M2-BFs: Delivery is performed with the aerosol inhaler device (nebulizer), 2.0 mL once a day up to 30 days.
The Number of Patients With Severe Adverse Events and Adverse Reactions	0 Participants

SECONDARY outcome

Timeframe: Baseline and 6 months after treatment

Population: Patients with neurological improvements are assessed by SACS "total" score as \> 6 points' reduction from baseline.

Subjective Assessment of Clinical Symptoms (SACS) is a 5-point rating scale with standardized criteria (0 - no; 1 - mild; 2 - moderate; 3 - severe; 4 - intensive) subjective assessment of the severity of fifteen clinical symptoms most characteristic of neurological disorders (headache, dizziness, gait disturbance, speech, visual impairment, tremor et al). Minimum SACS "total" score is 0, and maximum SACS "total" score is 60. Neurological improvements are assessed by SACS "total" score as \> 6 points' reduction from baseline.

Outcome measures

Outcome measures
Measure	Intranasal Auto-M2-BFs n=30 Participants Intranasally-Administered Bioactive Factors, Produced by Autologous M2 Macrophage (auto-M2-BFs). M2 macrophages were generated in vitro from peripheral blood of patients during 7 days.Cell-free culture medium, containing auto-M2-BFs, was collected and aliquots of 2 mL/vial were cryopreserved. 30 patients with organic brain syndrome will receive their first doses (n=2-3) of auto-M2-BFs in clinic and wait 2 hrs to determine any short-time adverse effects of inhaled dose. The subsequent course of intranasal inhalations (once a day up to 30 days) performed as outpatient treatment. Intranasal auto-M2-BFs: Delivery is performed with the aerosol inhaler device (nebulizer), 2.0 mL once a day up to 30 days.
Change in Subjective Assessment of Clinical Symptoms (SACS)	12 Participants

SECONDARY outcome

Timeframe: Baseline and 6 months after treatment

Population: Improvements in patients with anxiety/depression symptoms are assessed by HADS "total" score as \> 4 points reduction from baseline.

Hospital Anxiety and Depression Scale (HADS) is used to diagnose anxiety/depression symptoms (absence - 0\~7 points; subclinical form - 8\~10 points; clinical form - 11 points or more). Minimum HADS "total" score (anxiety + depression subscale) is 0, and maximum HADS "total" score is 42. Improvements in patients with anxiety/depression symptoms are assessed by HADS "total" score as \> 4 points reduction from baseline.

Outcome measures

Outcome measures
Measure	Intranasal Auto-M2-BFs n=26 Participants Intranasally-Administered Bioactive Factors, Produced by Autologous M2 Macrophage (auto-M2-BFs). M2 macrophages were generated in vitro from peripheral blood of patients during 7 days.Cell-free culture medium, containing auto-M2-BFs, was collected and aliquots of 2 mL/vial were cryopreserved. 30 patients with organic brain syndrome will receive their first doses (n=2-3) of auto-M2-BFs in clinic and wait 2 hrs to determine any short-time adverse effects of inhaled dose. The subsequent course of intranasal inhalations (once a day up to 30 days) performed as outpatient treatment. Intranasal auto-M2-BFs: Delivery is performed with the aerosol inhaler device (nebulizer), 2.0 mL once a day up to 30 days.
Change in Hospital Anxiety and Depression Scale (HADS)	14 Participants

SECONDARY outcome

Timeframe: Baseline and 6 months after treatment

Population: Improved mobility is assessed as FMA "total" score enhancement \> 4 points from baseline.

Functional Mobility Assessment (FMA) iscale is designed to evaluate parameters characterizing stability (0\~24 points) and gait (0\~16 points). The maximum FMA "total" score on stability and gait subscales is 39-40 and corresponds to the norm, minimum FMA "total" score is 0 and corresponds to the gross impairment. The degree of impairment of "total" score is divided into significant (0\~20 points), moderate (21\~33 points), and light (34\~38 points), whereas 39\~40 points indicate no impairments. Improved mobility is assessed as FMA "total" score enhancement \> 4 points from baseline.

Outcome measures

Outcome measures
Measure	Intranasal Auto-M2-BFs n=28 Participants Intranasally-Administered Bioactive Factors, Produced by Autologous M2 Macrophage (auto-M2-BFs). M2 macrophages were generated in vitro from peripheral blood of patients during 7 days.Cell-free culture medium, containing auto-M2-BFs, was collected and aliquots of 2 mL/vial were cryopreserved. 30 patients with organic brain syndrome will receive their first doses (n=2-3) of auto-M2-BFs in clinic and wait 2 hrs to determine any short-time adverse effects of inhaled dose. The subsequent course of intranasal inhalations (once a day up to 30 days) performed as outpatient treatment. Intranasal auto-M2-BFs: Delivery is performed with the aerosol inhaler device (nebulizer), 2.0 mL once a day up to 30 days.
Change in Functional Mobility Assessment (FMA) Scale	16 Participants

SECONDARY outcome

Timeframe: Baseline and 6 months after treatment

Population: Improvements in patients with cognitive disorder are assessed as MoCA MoCA "total" score increase \> 3 points from baseline.

Montreal Cognitive Assessment (MoCa) is used to assess cognitive functions. The maximum MoCa "total" score is 26-30 points and corresponds to the norm, 19-25 points - mild cognitive disorder; 11-21 points - dementia. Improvements in patients with cognitive disorder are assessed as MoCA "total" score increase \> 3 points from baseline.

Outcome measures

Outcome measures
Measure	Intranasal Auto-M2-BFs n=19 Participants Intranasally-Administered Bioactive Factors, Produced by Autologous M2 Macrophage (auto-M2-BFs). M2 macrophages were generated in vitro from peripheral blood of patients during 7 days.Cell-free culture medium, containing auto-M2-BFs, was collected and aliquots of 2 mL/vial were cryopreserved. 30 patients with organic brain syndrome will receive their first doses (n=2-3) of auto-M2-BFs in clinic and wait 2 hrs to determine any short-time adverse effects of inhaled dose. The subsequent course of intranasal inhalations (once a day up to 30 days) performed as outpatient treatment. Intranasal auto-M2-BFs: Delivery is performed with the aerosol inhaler device (nebulizer), 2.0 mL once a day up to 30 days.
Change in Montreal Cognitive Assessment (МоСА)	10 Participants

Adverse Events

Intranasal Auto-M2-BFs

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Alexander A Ostanin, MD, PhD

Institute of Fundamental and Clinical Immunology

Phone: +7 (383) 236-03-29

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place