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| Name | Class |
|---|---|
| CBR Systems, Inc. | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Autologous Stem Cells | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Autologous Stem Cells | Genetic | The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety of Autologous Stem Cell Infusion: Number of Participants With Adverse Events. | To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is safe. | 1 year |
| Feasibility of Autologous Stem Cell Infusion: Number of Participants Who Completed Cord Blood Infusion and 1 Month Follow-up. | To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is feasible. | 1 month |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Auditory Brainstem Response (ABR) Threshold in Decibels. | ABR Threshold is a measure of the sound necessary to activate transmission through the eighth cranial nerve. Each participant was evaluated before, 1,6 and 12 months after autologous stem cell treatments. For each ear, ABR threshold was measured at 500, 1000, 2000 and 4000 Hz and using "Click" ABR. | Pre-treatment (Baseline), 1,6 & 12 months post-treatment |
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Inclusion Criteria:
Evidence of a sensorineural hearing loss
Normally shaped cochlea, as determined by MRI
The loss must be considered:
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:
Known history of:
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
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| Name | Affiliation | Role |
|---|---|---|
| James Baumgartner, MD | AdventHealth | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Florida Hospital | Orlando | Florida | 32803 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Autologous Stem Cells | 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. Autologous Stem Cells: The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | Autologous Stem Cells | 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. Autologous Stem Cells: The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Safety of Autologous Stem Cell Infusion: Number of Participants With Adverse Events. | To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is safe. | Posted | Count of Participants | Participants | 1 year |
|
1 year
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Autologous Stem Cells | 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. Autologous Stem Cells: The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| James Baumgartner, Surgical Director Comprehensive Epilepsy Program | Florida Hospital | 4072360006 | james.baumgartner.md@flhosp.org |
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| ID | Term |
|---|---|
| D006319 | Hearing Loss, Sensorineural |
| ID | Term |
|---|---|
| D034381 | Hearing Loss |
| D006311 | Hearing Disorders |
| D004427 | Ear Diseases |
| D010038 | Otorhinolaryngologic Diseases |
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| ID | Term |
|---|---|
| D064987 | Cell- and Tissue-Based Therapy |
| ID | Term |
|---|---|
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
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Treatment consisted of autologous cord blood mononuclear fraction. Dose was the total nucleated cells delivered intravenously per kilogram of treatment subject weight
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| Change in Eight Cranial Nerve Wave 5 Latencies in Miliseconds. | .For each ear, latency was measured at 500, 1000, 2000 and 4000 Hz and using "Click" ABR. | Pre-treatment (Baseline), 1,6 & 12 months post-treatment |
| Change in Standard Language Scores. | Preschool Language Scale-5. This test measures language development in infants and children. Standard scores are derived, with a mean of 100 and a standard deviation of 15, to compare a child's performance to their peers. Higher values are associated with better language development. | Pre-treatment, 6 & 12 months post-treatment |
| Change Fractional Anisotropy in the White Sites Along the Audiotory Pathways | Fractional anisotropy are arbitrary values generated from the DTI sequences of an MRI scan. Higher values suggest increased integrity of white matter pathways. Responders and nonresponders were evaluated separately. (Define non-responders and responders).Although 11 subjects were enrolled and received an autologous cord blood infusion, three subjects did not complete the entire MRI study protocol and could not be analyzed at study completion. The reasons these subjects did not undergo the second MRI are as follows: Subject 7 dropped out of the study, and Subject 11 underwent bilateral cochlear implantation after the 1-month follow-up. Subject 10 refused testing at 12-month follow-up. | Pre-treatment & 12 months post-treatment |
| months |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Count of Participants | Participants |
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| Units | Counts |
|---|
| Participants |
|
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| Primary | Feasibility of Autologous Stem Cell Infusion: Number of Participants Who Completed Cord Blood Infusion and 1 Month Follow-up. | To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is feasible. | Posted | Count of Participants | Participants | 1 month |
|
|
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| Secondary | Change in Auditory Brainstem Response (ABR) Threshold in Decibels. | ABR Threshold is a measure of the sound necessary to activate transmission through the eighth cranial nerve. Each participant was evaluated before, 1,6 and 12 months after autologous stem cell treatments. For each ear, ABR threshold was measured at 500, 1000, 2000 and 4000 Hz and using "Click" ABR. | After the 1 month follow-up visit, two subjects dropped out. | Posted | Mean | Standard Deviation | decibels | Pre-treatment (Baseline), 1,6 & 12 months post-treatment |
|
|
|
| Secondary | Change in Eight Cranial Nerve Wave 5 Latencies in Miliseconds. | .For each ear, latency was measured at 500, 1000, 2000 and 4000 Hz and using "Click" ABR. | After the 1 month follow-up visit, two subjects dropped out. | Posted | Mean | Standard Deviation | milliseconds | Pre-treatment (Baseline), 1,6 & 12 months post-treatment |
|
|
|
| Secondary | Change in Standard Language Scores. | Preschool Language Scale-5. This test measures language development in infants and children. Standard scores are derived, with a mean of 100 and a standard deviation of 15, to compare a child's performance to their peers. Higher values are associated with better language development. | Posted | Mean | Standard Deviation | units on a scale | Pre-treatment, 6 & 12 months post-treatment |
|
|
|
| Secondary | Change Fractional Anisotropy in the White Sites Along the Audiotory Pathways | Fractional anisotropy are arbitrary values generated from the DTI sequences of an MRI scan. Higher values suggest increased integrity of white matter pathways. Responders and nonresponders were evaluated separately. (Define non-responders and responders).Although 11 subjects were enrolled and received an autologous cord blood infusion, three subjects did not complete the entire MRI study protocol and could not be analyzed at study completion. The reasons these subjects did not undergo the second MRI are as follows: Subject 7 dropped out of the study, and Subject 11 underwent bilateral cochlear implantation after the 1-month follow-up. Subject 10 refused testing at 12-month follow-up. | All participants who completed the trial and had appropriate MRI data before and after treatment. There were 4 "responders" and 4 "non-responders" analyzed. | Posted | Mean | Standard Deviation | unitless | Pre-treatment & 12 months post-treatment |
|
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| 0 |
| 11 |
| 0 |
| 11 |
| 0 |
| 11 |
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| D012678 |
| Sensation Disorders |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| f2 Right Ear Click |
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| f3 Left Ear 500 Hz |
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| f4 Right Ear 500 Hs |
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| f5 Left Ear 1000 Hz |
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| f6 Right Ear 1000 Hz |
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| f7 Left Ear 2000 Hz |
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| f8 Right Ear 2000 Hz |
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| f9 Left Ear 4000 Hz |
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| f10 Right Ear 4000Hz |
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| f2 Right Ear Click |
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| f3 Left Ear 500 Hz |
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| f4 Right Ear 500 Hs |
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| f5 Left Ear 1000 Hz |
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| f6 Right Ear 1000 Hz |
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| f7 Left Ear 2000 Hz |
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| f8 Right Ear 2000 Hz |
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| f9 Left Ear 4000 Hz |
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| f10 Right Ear 4000Hz |
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| Responder Baseline Right |
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| Responder Baseline Left |
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| Nonresponder 12 Month Right |
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| Nonresponder 12 Month Left |
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| Responder 12 Month Right |
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| Responder 12 Month Left |
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