Sensorineural Hearing LossPhase 1NCT02038972
Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss
A Phase 1 study testing Autologous Stem Cells in people with Sensorineural Hearing Loss, run by James Baumgartner, MD at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 weeks to 6 years
- Conditions the study is for
- Sensorineural Hearing Loss
- What is being tested?
- Autologous Stem Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2013to 10 Jan 2017
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 weeks to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Sensorineural Hearing Loss
- Being tested
- Autologous Stem Cells
- Medical subject headings
- Hearing Loss, Sensorineural
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Stem CellsGenetic
Also called Cell based therapy
The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Genetic: Autologous Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 1 month
Secondary outcomes (4)
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.
Measured: Pre-treatment (Baseline), 1,6 & 12 months post-treatment
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.
Measured: 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.
Measured: 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.
Measured: Pre-treatment & 12 months post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2014 (estimated)
- Study startJan 2013
- Primary completion11 Dec 2015
- Study completion10 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sensorineural Hearing LossStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Treatment consisted of autologous cord blood mononuclear fraction. Dose was the total nucleated cells delivered intravenously per kilogram of treatment subject weight
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- James Baumgartner, MD
- Sponsor type
- University, hospital or other
- Collaborators
- CBR Systems, Inc.
- Responsible party
- Sponsor-investigator, James Baumgartner, MD, AdventHealth
- Investigators
- James Baumgartner, MD, Principal investigator, AdventHealth
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02038972
- Sponsor's protocol ID
- 434269
- Status last verified
- Oct 2023
- First submitted
- 8 Jan 2014
- Last updated
- 9 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss
- ClinicalTrials.gov ID
- NCT02038972
- Acronym / sponsor ID
- 434269
- Conditions
- Sensorineural Hearing Loss
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- James Baumgartner, MDUniversity, hospital or other
- Collaborators
- CBR Systems, Inc.
- Study start
- Jan 2013
- Primary completion
- 11 Dec 2015 (actual)
- Study completion
- 10 Jan 2017 (actual)
- First posted
- 17 Jan 2014 (estimated)
- Results
- Posted 9 Jul 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Treatment consisted of autologous cord blood mononuclear fraction. Dose was the total nucleated cells delivered intravenously per kilogram of treatment subject weight
| Arm | Type | Receives |
|---|---|---|
| Autologous Stem Cells | Experimental | Genetic: Autologous Stem CellsA 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. |
Who may qualify
- Ages
- 6 weeks to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (13 listed)
- 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
Key exclusion criteria (26 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Stem CellsAlso: Cell based therapyThe subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity. | Genetic | Autologous Stem Cells |
Primary outcome
Safety of Autologous Stem Cell Infusion: Number of Participants With Adverse Events.
Time frame: 1 year
To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is safe.
Feasibility of Autologous Stem Cell Infusion: Number of Participants Who Completed Cord Blood Infusion and 1 Month Follow-up.
Time frame: 1 month
To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is feasible.
4 secondary outcomes
Change in Auditory Brainstem Response (ABR) Threshold in Decibels.
Time frame: Pre-treatment (Baseline), 1,6 & 12 months post-treatment
Change in Eight Cranial Nerve Wave 5 Latencies in Miliseconds.
Time frame: Pre-treatment (Baseline), 1,6 & 12 months post-treatment
Change in Standard Language Scores.
Time frame: Pre-treatment, 6 & 12 months post-treatment
Change Fractional Anisotropy in the White Sites Along the Audiotory Pathways
Time frame: Pre-treatment & 12 months post-treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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Post-Market Registry Study for Vibrant Hearing Solutions
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James Baumgartner, MD principal investigator
AdventHealth
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Stem Cells in people with Sensorineural Hearing Loss, run by James Baumgartner, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Jul 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of Autologous Stem Cell Infusion: Number of Participants With Adverse Events. (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jul 2026.