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The Use of Exosomes In Craniofacial Neuralgia

Primary Purpose

Neuralgia

Status
Suspended
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Exosomes
Sponsored by
Neurological Associates of West Los Angeles
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neuralgia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Male or Female at least 18 years of age
  • Clinical diagnosis of craniofacial neuralgia

Exclusion Criteria:

  • Subjects unable to give informed consent
  • Subjects who would not be able to lay down without excessive movement in a calm environment sufficiently long enough to be able to achieve sleep
  • Recent surgery or dental work within 3 months of the scheduled procedure.
  • Pregnancy, women who may become pregnant or are breastfeeding
  • Advanced terminal illness
  • Any active cancer or chemotherapy
  • Bone marrow disorder
  • Myeloproliferative disorder
  • Sickle cell disease
  • Primary pulmonary hypertension
  • Immunocompromising conditions and/or immunosuppressive therapies
  • Macular degeneration
  • Subjects with scalp rash or open wounds on the scalp (for example from treatment of squamous cell cancer)
  • Advanced kidney, pulmonary, cardiac or liver failure
  • Subjects with vascular causes of dementia
  • Bleeding disorder, untreated

Sites / Locations

  • Neurological Associates of West LA

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment of Craniofacial Neuralgia

Arm Description

All patients will receive the same amount (5mL concentrated) of exosomes delivered via ultrasound-guided, regional epineural injection and the same amount (5mL unconcentrated) delivered via IV. Patients will be given 3 mL of the exosome product intravenously, which contains about 45mg of the exosome product containing 15-21 million neonatal stem cell products, and 3 mL of the exosome hyperconcentrate product delivered epineurally using ultrasound guidance, which contains about 15mg of the exosome product carrying 5-7 million neonatal stem cell products.

Outcomes

Primary Outcome Measures

Brief Pain Inventory (BPI)
The BPI is a 9 item self-report questionnaire used to evaluate the severity of a patient's pain and the impact of this pain on the patient's daily functioning. Self-report measure containing a composite pain score and functional interference score. The pain subscale contains 4 questions, each with answers ranging from 0 'no pain' to 10 'pain as bad as you can imagine.' Total possible score for the pain subscale is 40 points. The functional/interference subscale contains 7 questions, with each answer ranging from 0 'does not interfere' to 10 'completely interferes.' The maximum possible score for the interference subscale is 70 points. The total overall composite BPI score is out of 100 maximum points. A clinical improvement is considered a decrease in BPI overall composite score by at least 30% from baseline.
Patient Health Questionnaire (PHQ-9)
The Patient Health Questionnaire (PHQ) is a self-administered version of the PRIME-MD diagnostic instrument for common mental disorders. The PHQ-9 is the depression module, which scores each of the 9 DSM-IV criteria as "0" (not at all) to "3" (nearly every day). Total possible score is 27 points. A clinical improvement is considered a decrease in BPI overall composite score by at least 30% from baseline.
Adverse Event Reporting
Adverse events (AEs) and any other untoward signs or symptoms were collected at each study timepoint starting at the treatment injection. Serious adverse events (SAEs) determined by the investigator to be related to the study treatment were formally recorded.

Secondary Outcome Measures

Global Rating of Change (GRC)
The GRC is a self-administered one-item Likert scale questionnaire on the patient's overall satisfaction with the treatment; with scores from "-5" (very much worse) to "+5" (very much better). A GRC of at least is considered to be clinically significant improvement.
Brief Pain Inventory (BPI)
The BPI is a 9 item self-report questionnaire used to evaluate the severity of a patient's pain and the impact of this pain on the patient's daily functioning. Self-report measure containing a composite pain score and functional interference score. The pain subscale contains 4 questions, each with answers ranging from 0 'no pain' to 10 'pain as bad as you can imagine.' Total possible score for the pain subscale is 40 points. The functional/interference subscale contains 7 questions, with each answer ranging from 0 'does not interfere' to 10 'completely interferes.' The maximum possible score for the interference subscale is 70 points. The total overall composite BPI score is out of 100 maximum points. A clinical improvement is considered a decrease in BPI overall composite score by at least 30% from baseline.
Patient Health Questionnaire (PHQ-9)
The Patient Health Questionnaire (PHQ) is a self-administered version of the PRIME-MD diagnostic instrument for common mental disorders. The PHQ-9 is the depression module, which scores each of the 9 DSM-IV criteria as "0" (not at all) to "3" (nearly every day). Total possible score is 27 points. A clinical improvement is considered a decrease in BPI overall composite score by at least 30% from baseline.

Full Information

First Posted
December 16, 2019
Last Updated
September 26, 2022
Sponsor
Neurological Associates of West Los Angeles
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1. Study Identification

Unique Protocol Identification Number
NCT04202783
Brief Title
The Use of Exosomes In Craniofacial Neuralgia
Official Title
The Use of Exosomes In Craniofacial Neuralgia
Study Type
Interventional

2. Study Status

Record Verification Date
September 2022
Overall Recruitment Status
Suspended
Why Stopped
Pending COVID-19 pandemic; pending product development
Study Start Date
December 1, 2023 (Anticipated)
Primary Completion Date
December 1, 2024 (Anticipated)
Study Completion Date
December 1, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurological Associates of West Los Angeles

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This study is designed to evaluate the safety and efficacy of exosome deployment in patients with Craniofacial Neuralgia. Secondarily, this study is designed to rigorously evaluate for any adverse events that may be related to the administration and reception of exosomes.
Detailed Description
The present study is designed to amplify the delivery of growth factors and anti-inflammatory agents to localized targets (determined by specific condition) by using focused transcranial ultrasound prior to intravenous infusion of exosomes. Exosomes delivered intravenously can be demonstrated to cross the blood brain barrier naturally. Exosomes are thought to play a normal physiological role in intercellular signaling, and demonstrate anti-inflammatory and pro-growth effects in preclinical models and clinical cases reports. Clinical trials have deployed exosomes intravenously and with intracerebral and intrathecal injection, and have claimed safety and clinical efficacy. Focused ultrasound has been shown to enhance local blood flow and has been presented as a non-invasive means of targeting delivery of therapeutic agents.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuralgia

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
The present study is being undertaken as an open label study to evaluate the safety and feasibility of exosomes as an intervention for patients with craniofacial neuralgia. Baseline and outcome measures in this study utilize validated tests that are appropriate for repeated measures. The present study can be easily implemented because the neuropsychological testing instruments have been in routine clinical deployment providing for a high degree of availability and reliability. Quality assurance is tightly controlled. The study population is sufficiently broad and the conditions of interest are sufficiently prevalent so that recruitment of subjects is not a limiting factor.
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Treatment of Craniofacial Neuralgia
Arm Type
Experimental
Arm Description
All patients will receive the same amount (5mL concentrated) of exosomes delivered via ultrasound-guided, regional epineural injection and the same amount (5mL unconcentrated) delivered via IV. Patients will be given 3 mL of the exosome product intravenously, which contains about 45mg of the exosome product containing 15-21 million neonatal stem cell products, and 3 mL of the exosome hyperconcentrate product delivered epineurally using ultrasound guidance, which contains about 15mg of the exosome product carrying 5-7 million neonatal stem cell products.
Intervention Type
Other
Intervention Name(s)
Exosomes
Intervention Description
Focused ultrasound delivery of intravenously-infused exosomes
Primary Outcome Measure Information:
Title
Brief Pain Inventory (BPI)
Description
The BPI is a 9 item self-report questionnaire used to evaluate the severity of a patient's pain and the impact of this pain on the patient's daily functioning. Self-report measure containing a composite pain score and functional interference score. The pain subscale contains 4 questions, each with answers ranging from 0 'no pain' to 10 'pain as bad as you can imagine.' Total possible score for the pain subscale is 40 points. The functional/interference subscale contains 7 questions, with each answer ranging from 0 'does not interfere' to 10 'completely interferes.' The maximum possible score for the interference subscale is 70 points. The total overall composite BPI score is out of 100 maximum points. A clinical improvement is considered a decrease in BPI overall composite score by at least 30% from baseline.
Time Frame
8 weeks from baseline
Title
Patient Health Questionnaire (PHQ-9)
Description
The Patient Health Questionnaire (PHQ) is a self-administered version of the PRIME-MD diagnostic instrument for common mental disorders. The PHQ-9 is the depression module, which scores each of the 9 DSM-IV criteria as "0" (not at all) to "3" (nearly every day). Total possible score is 27 points. A clinical improvement is considered a decrease in BPI overall composite score by at least 30% from baseline.
Time Frame
8 weeks from baseline
Title
Adverse Event Reporting
Description
Adverse events (AEs) and any other untoward signs or symptoms were collected at each study timepoint starting at the treatment injection. Serious adverse events (SAEs) determined by the investigator to be related to the study treatment were formally recorded.
Time Frame
Baseline to 24 weeks
Secondary Outcome Measure Information:
Title
Global Rating of Change (GRC)
Description
The GRC is a self-administered one-item Likert scale questionnaire on the patient's overall satisfaction with the treatment; with scores from "-5" (very much worse) to "+5" (very much better). A GRC of at least is considered to be clinically significant improvement.
Time Frame
8 weeks from baseline
Title
Brief Pain Inventory (BPI)
Description
The BPI is a 9 item self-report questionnaire used to evaluate the severity of a patient's pain and the impact of this pain on the patient's daily functioning. Self-report measure containing a composite pain score and functional interference score. The pain subscale contains 4 questions, each with answers ranging from 0 'no pain' to 10 'pain as bad as you can imagine.' Total possible score for the pain subscale is 40 points. The functional/interference subscale contains 7 questions, with each answer ranging from 0 'does not interfere' to 10 'completely interferes.' The maximum possible score for the interference subscale is 70 points. The total overall composite BPI score is out of 100 maximum points. A clinical improvement is considered a decrease in BPI overall composite score by at least 30% from baseline.
Time Frame
24 weeks from baseline
Title
Patient Health Questionnaire (PHQ-9)
Description
The Patient Health Questionnaire (PHQ) is a self-administered version of the PRIME-MD diagnostic instrument for common mental disorders. The PHQ-9 is the depression module, which scores each of the 9 DSM-IV criteria as "0" (not at all) to "3" (nearly every day). Total possible score is 27 points. A clinical improvement is considered a decrease in BPI overall composite score by at least 30% from baseline.
Time Frame
24 weeks from baseline

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or Female at least 18 years of age Clinical diagnosis of craniofacial neuralgia Exclusion Criteria: Subjects unable to give informed consent Subjects who would not be able to lay down without excessive movement in a calm environment sufficiently long enough to be able to achieve sleep Recent surgery or dental work within 3 months of the scheduled procedure. Pregnancy, women who may become pregnant or are breastfeeding Advanced terminal illness Any active cancer or chemotherapy Bone marrow disorder Myeloproliferative disorder Sickle cell disease Primary pulmonary hypertension Immunocompromising conditions and/or immunosuppressive therapies Macular degeneration Subjects with scalp rash or open wounds on the scalp (for example from treatment of squamous cell cancer) Advanced kidney, pulmonary, cardiac or liver failure Subjects with vascular causes of dementia Bleeding disorder, untreated
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sheldon Jordan, MD
Organizational Affiliation
Neurological Associates of West Los Angeles
Official's Role
Principal Investigator
Facility Information:
Facility Name
Neurological Associates of West LA
City
Santa Monica
State/Province
California
ZIP/Postal Code
90403
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Data from this study will not be made publicly available due to ethical and privacy concerns. Anonymized data will be available upon reasonable request from any qualified investigator.
Citations:
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18710080
Citation
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The Use of Exosomes In Craniofacial Neuralgia

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