Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss
Primary Purpose
Sensorineural Hearing Loss
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Autologous Stem Cells
Sponsored by
About this trial
This is an interventional treatment trial for Sensorineural Hearing Loss
Eligibility Criteria
Inclusion Criteria:
Evidence of a sensorineural hearing loss
- Unilateral or bilateral in configuration
- Symmetrical or asymmetrical configuration
- Sudden or progressive in presentation
- Moderate to profound in degree (40-90 Decibels (dB) in at least one ear
- Normally shaped cochlea, as determined by MRI
The loss must be considered:
- Acquired
- Unknown with a negative genetic test.
- Fitted for hearing aids no later than six months post detection of loss.
- Enrollment in a parent/child intervention program
- Age 6 weeks - 6 years old at time of infusion with less than 18 months of hearing loss at the time of cord blood infusion.
- Ability of the child and caregiver to travel to Orlando, and stay for at least 4 days, and to return for all follow-up visits.
Exclusion Criteria
Inability to obtain all pertinent medical records:
- (pertinent physician notes, speech language pathology notes, laboratory findings, test results and imaging studies-must be sent to the research team at least prior to the subject arriving at the study location for preliminary screening and eligibility assessment, preferably14 days before the scheduled hUBC treatment.)
Known history of:
- Recently treated infection less than 2 weeks before infusion.
- Renal disease of altered renal function as defined by serum creatinine > 1.5 mg/dl at admission.
- Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and or T. Bilirubin > 1.3 mg/dL
- Malignancy
- Immunosuppression as defined by WBC < 3,000 at admission
- Human Immunodeficiency Virus (HIV)
- Hepatitis B
- Hepatitis C
- Evidence of an extensive stroke (> 100ml lesion)
- Pneumonia, or chronic lung disease requiring oxygen
- Genetic syndromic sensorineural hearing loss
- hUBC sample contamination
- Banked cord cells totaling less than 6x106 mononuclear cells/kilogram body weight.
- Evidence of the following maternal infections during the pregnancy (Hepatitis A, Hepatitis B, Hepatitis C, HIV 1, HIV 2, Human T-lymphotropic Virus (HTLV) 1, HTLV 2 (CMV and Syphilis can be included in the study)
- participation in a concurrent intervention study
- Unwillingness or inability to stay for 4 days following hUBC infusion (should problems arise following the infusion) and to return for the one month, six month and one year follow-up visits.
- Presence of a cochlear implantation device
- Evidence of a genetic syndrome
- Evidence of conductive hearing loss
- Documented recurrent middle ear infections which are frequent (>5 per year)
- Otitis media at the time of examination
- Sensorineural loss is mild
- Over 18 months from identification of hearing loss at time of infusion
Sites / Locations
- Florida Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Autologous Stem Cells
Arm Description
A single dose of intravenously administered autologous hUCB will be done. The minimum acceptable dose will be 6x10 6th mononuclear cells/kilogram body weight. The hUCB reanimation, cell processing and product infusion will occur at Florida Hospital for Children and the Florida Hospital Center for Cellular Therapy.
Outcomes
Primary Outcome Measures
Safety of Autologous Stem Cell Infusion
To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is safe and feasible. Infusion related toxicity as measured by:
i. hemodynamic instability: An adverse event will be defined as a sustained (> 10 minutes) >20% decrease in MAP.
ii. acute lung injury: Chest X-ray will be done at baseline and at 1 day post infusion to assess for polymorphonuclear infiltrates iii. hepatic injury/toxicity: Hepatic panel will be performed at baseline and 1 day post infusion. Injury is defined as acute elevation of the AST/ALT hepatic enzymes > 900 U/dl in the first 24 hours post infusion iv. renal injury/insufficiency: CMP will be performed at baseline and 1 day after infusion v. exacerbation of neurological status: defined as a change in Glasgow Coma Scale, pupillary size/reactivity, motor/sensory evaluation of extremities, and seizure activity from infusion to discharge.
Secondary Outcome Measures
Inner Ear Function, Audition, and Language Development
To determine if autologous hUBC transplantation in children with hearing loss improves inner ear function, audition and language development.
Full Information
NCT ID
NCT02038972
First Posted
January 8, 2014
Last Updated
March 1, 2018
Sponsor
James Baumgartner, MD
Collaborators
CBR Systems, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT02038972
Brief Title
Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss
Official Title
Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss
Study Type
Interventional
2. Study Status
Record Verification Date
August 2017
Overall Recruitment Status
Completed
Study Start Date
January 2013 (undefined)
Primary Completion Date
December 11, 2015 (Actual)
Study Completion Date
January 10, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
James Baumgartner, MD
Collaborators
CBR Systems, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
To determine if autologous human umbilical cord blood infusion in children with acquired hearing loss is safe, feasible, improves inner ear function, audition and language development.
Detailed Description
There is currently no treatment available to repair/reverse acquired sensorineural hearing loss. Recent experiments using human umbilical cord blood treatment of a mouse and guinea pig models have demonstrated hair cell re-growth following acquired sensorineural loss as well as partial restoration of ABR. Autologous human umbilical cord blood therapy, which has been used for over twenty years, has an excellent safety record. This study will determine if autologous human umbilical cord blood infusion in children with hearing loss is safe and feasible, improves inner ear function, audition, and language development. The patients umbilical cord stem cells collected at birth and stored at Cord Blood Registry will be used for infusion.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sensorineural Hearing Loss
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Autologous Stem Cells
Arm Type
Experimental
Arm Description
A single dose of intravenously administered autologous hUCB will be done. The minimum acceptable dose will be 6x10 6th mononuclear cells/kilogram body weight. The hUCB reanimation, cell processing and product infusion will occur at Florida Hospital for Children and the Florida Hospital Center for Cellular Therapy.
Intervention Type
Genetic
Intervention Name(s)
Autologous Stem Cells
Other Intervention Name(s)
Cell based therapy
Intervention Description
The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity.
Primary Outcome Measure Information:
Title
Safety of Autologous Stem Cell Infusion
Description
To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is safe and feasible. Infusion related toxicity as measured by:
i. hemodynamic instability: An adverse event will be defined as a sustained (> 10 minutes) >20% decrease in MAP.
ii. acute lung injury: Chest X-ray will be done at baseline and at 1 day post infusion to assess for polymorphonuclear infiltrates iii. hepatic injury/toxicity: Hepatic panel will be performed at baseline and 1 day post infusion. Injury is defined as acute elevation of the AST/ALT hepatic enzymes > 900 U/dl in the first 24 hours post infusion iv. renal injury/insufficiency: CMP will be performed at baseline and 1 day after infusion v. exacerbation of neurological status: defined as a change in Glasgow Coma Scale, pupillary size/reactivity, motor/sensory evaluation of extremities, and seizure activity from infusion to discharge.
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Inner Ear Function, Audition, and Language Development
Description
To determine if autologous hUBC transplantation in children with hearing loss improves inner ear function, audition and language development.
Time Frame
1 year
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Weeks
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Evidence of a sensorineural hearing loss
Unilateral or bilateral in configuration
Symmetrical or asymmetrical configuration
Sudden or progressive in presentation
Moderate to profound in degree (40-90 Decibels (dB) in at least one ear
Normally shaped cochlea, as determined by MRI
The loss must be considered:
Acquired
Unknown with a negative genetic test.
Fitted for hearing aids no later than six months post detection of loss.
Enrollment in a parent/child intervention program
Age 6 weeks - 6 years old at time of infusion with less than 18 months of hearing loss at the time of cord blood infusion.
Ability of the child and caregiver to travel to Orlando, and stay for at least 4 days, and to return for all follow-up visits.
Exclusion Criteria
Inability to obtain all pertinent medical records:
(pertinent physician notes, speech language pathology notes, laboratory findings, test results and imaging studies-must be sent to the research team at least prior to the subject arriving at the study location for preliminary screening and eligibility assessment, preferably14 days before the scheduled hUBC treatment.)
Known history of:
Recently treated infection less than 2 weeks before infusion.
Renal disease of altered renal function as defined by serum creatinine > 1.5 mg/dl at admission.
Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and or T. Bilirubin > 1.3 mg/dL
Malignancy
Immunosuppression as defined by WBC < 3,000 at admission
Human Immunodeficiency Virus (HIV)
Hepatitis B
Hepatitis C
Evidence of an extensive stroke (> 100ml lesion)
Pneumonia, or chronic lung disease requiring oxygen
Genetic syndromic sensorineural hearing loss
hUBC sample contamination
Banked cord cells totaling less than 6x106 mononuclear cells/kilogram body weight.
Evidence of the following maternal infections during the pregnancy (Hepatitis A, Hepatitis B, Hepatitis C, HIV 1, HIV 2, Human T-lymphotropic Virus (HTLV) 1, HTLV 2 (CMV and Syphilis can be included in the study)
participation in a concurrent intervention study
Unwillingness or inability to stay for 4 days following hUBC infusion (should problems arise following the infusion) and to return for the one month, six month and one year follow-up visits.
Presence of a cochlear implantation device
Evidence of a genetic syndrome
Evidence of conductive hearing loss
Documented recurrent middle ear infections which are frequent (>5 per year)
Otitis media at the time of examination
Sensorineural loss is mild
Over 18 months from identification of hearing loss at time of infusion
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James Baumgartner, MD
Organizational Affiliation
AdventHealth
Official's Role
Principal Investigator
Facility Information:
Facility Name
Florida Hospital
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
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Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss
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