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CompletedNCT02038972Updated Jul 9, 2026Results posted

Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss

A Phase 1 interventional study of Autologous Stem Cells in Sensorineural Hearing Loss, sponsored by James Baumgartner, MD. Completed at 1 site in United States. Open to participants aged 6 Weeks to 6 Years. Per ClinicalTrials.gov, last updated 2026-07-09.

Sponsored by James Baumgartner, MD · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
11
Allocation
Not applicable
Ages
6 Weeks to 6 Years
Sex
All
01

Study 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 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.

02

Conditions studied

  • Sensorineural Hearing Loss
03

In context

Hearing Loss, Sensorineural

371 studies on the registry are indexed under Hearing Loss, Sensorineural; 112 are open to participants now.

This study's enrollment of 11 is below the median of 32 across 264 interventional studies indexed under Hearing Loss, Sensorineural.

Browse Hearing Loss, Sensorineural studies →

Lead sponsor

James Baumgartner, MD is the lead sponsor of 3 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Weeks to 6 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. 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
  2. Normally shaped cochlea, as determined by MRI

  3. The loss must be considered:

    • Acquired
    • Unknown with a negative genetic test.
  4. Fitted for hearing aids no later than six months post detection of loss.
  5. Enrollment in a parent/child intervention program
  6. 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.
  7. 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

Exclusion Criteria

  1. 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.)

  2. 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
  3. hUBC sample contamination
  4. Banked cord cells totaling less than 6x106 mononuclear cells/kilogram body weight.
  5. 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)
  6. participation in a concurrent intervention study
  7. 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.
  8. Presence of a cochlear implantation device
  9. Evidence of a genetic syndrome
  10. Evidence of conductive hearing loss
  11. Documented recurrent middle ear infections which are frequent (>5 per year)
  12. Otitis media at the time of examination
  13. Sensorineural loss is mild
  14. Over 18 months from identification of hearing loss at time of infusion
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
11 participants (actual)

Study arms

  • Experimental
    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.

    Genetic: Autologous Stem Cells

Interventions

  • GeneticAutologous Stem Cells

    The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity.

    Also known as: Cell based therapy

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: 1 year

  2. 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.

    Time frame: 1 month

Secondary outcomes

  1. 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.

    Time frame: Pre-treatment (Baseline), 1,6 & 12 months post-treatment

  2. 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.

    Time frame: Pre-treatment (Baseline), 1,6 & 12 months post-treatment

  3. 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.

    Time frame: Pre-treatment, 6 & 12 months post-treatment

  4. 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.

    Time frame: Pre-treatment & 12 months post-treatment

07

Results

Posted Jul 9, 2026

Participant flow

Participant flow — Overall Study
MilestoneAutologous Stem Cells
Started11
Completed9
Not completed2
Withdrew: Withdrawal by subject2

Outcome measures

PrimarySafety 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.

Time frame:
1 year
Reported as:
Count of participants · Participants
Safety of Autologous Stem Cell Infusion: Number of Participants With Adverse Events.
ParticipantsAutologous Stem Cells
Safety of Autologous Stem Cell Infusion: Number of Participants With Adverse Events.0
PrimaryFeasibility 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.

Time frame:
1 month
Reported as:
Count of participants · Participants
Feasibility of Autologous Stem Cell Infusion: Number of Participants Who Completed Cord Blood Infusion and 1 Month Follow-up.
ParticipantsAutologous Stem Cells
Feasibility of Autologous Stem Cell Infusion: Number of Participants Who Completed Cord Blood Infusion and 1 Month Follow-up.11
SecondaryChange 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.

Time frame:
Pre-treatment (Baseline), 1,6 & 12 months post-treatment
Reported as:
Mean · decibels
Change in Auditory Brainstem Response (ABR) Threshold in Decibels.
decibelsAutologous Stem Cells-BaselineAutologous Stem Cells-1 Month Post-treatmentAutologous Stem Cell-6 Months Post-treatmentAutologous Stem Cell-12 Months Post-treatment
f1 Left Ear Click59.09 ± 27.0047.27 ± 23.6050.91 ± 23.8647.78 ± 23.06
f2 Right Ear Click58.00 ± 22.3953.64 ± 15.9859.09 ± 23.2248.13 ± 11.00
f3 Left Ear 500 Hz60.45 ± 23.7160.38 ± 25.6657.00 ± 22.3956.67 ± 25.00
f4 Right Ear 500 Hs60.00 ± 27.1156.36 ± 25.2156.00 ± 24.8156.67 ± 24.87
f5 Left Ear 1000 Hz52.78 ± 21.2351.50 ± 23.4649.44 ± 23.5151.88 ± 24.04
f6 Right Ear 1000 Hz63.75 ± 29.2457.08 ± 25.6261.67 ± 24.8953.13 ± 20.34
f7 Left Ear 2000 Hz60.50 ± 21.2755.83 ± 27.2165.00 ± 23.0958.13 ± 17.92
f8 Right Ear 2000 Hz63.64 ± 24.3061.67 ± 24.1562.00 ± 23.2455.63 ± 17.00
f9 Left Ear 4000 Hz60.00 ± 25.6964.23 ± 27.9858.33 ± 22.2258.13 ± 21.70
f10 Right Ear 4000Hz62.50 ± 25.4160.83 ± 27.7061.11 ± 25.8358.13 ± 25.20
SecondaryChange 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.

Time frame:
Pre-treatment (Baseline), 1,6 & 12 months post-treatment
Reported as:
Mean · milliseconds
Change in Eight Cranial Nerve Wave 5 Latencies in Miliseconds.
millisecondsAutologous Stem Cells-BaselineAutologous Stem Cells-1 Month Post-treatmentAutologous Stem Cell-6 Months Post-treatmentAutologous Stem Cell-12 Months Post-treatment
f1 Left Ear Click1.33 ± 0.091.38 ± 0.081.53 ± 0.421.26 ± 0.17
f2 Right Ear Click3.78 ± 0.343.93 ± 0.453.98 ± 0.563.60 ± 0.36
f3 Left Ear 500 Hz6.51 ± 1.266.40 ± 1.076.05 ± 1.235.55 ± 0.65
f4 Right Ear 500 Hs1.96 ± 1.072.02 ± 1.672.38 ± 1.062.01 ± 0.92
f5 Left Ear 1000 Hz3.77 ± 2.663.59 ± 2.591.40 ± 3.552.47 ± 1.84
f6 Right Ear 1000 Hz5.55 ± 1.685.61 ± 1.524.29 ± 2.134.48 ± 1.00
f7 Left Ear 2000 Hz1.36 ± 0.191.65 ± 0.451.47 ± 0.221.46 ± 0.51
f8 Right Ear 2000 Hz3.95 ± 0.484.01 ± 0.333.72 ± 0.273.78 ± 0.56
f9 Left Ear 4000 Hz6.75 ± 1.406.52 ± 1.096.27 ± 1.506.30 ± 1.46
f10 Right Ear 4000Hz1.73 ± 1.311.13 ± 1.641.84 ± 0.941.99 ± 0.91
SecondaryChange 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.

Time frame:
Pre-treatment, 6 & 12 months post-treatment
Reported as:
Mean · units on a scale
Change in Standard Language Scores.
units on a scaleAutologous Stem Cells
Month 6-Pre-treatment2.4 ± 9.8
Month 12-Pre-treatment12.5 ± 14.7
SecondaryChange 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.

Time frame:
Pre-treatment & 12 months post-treatment
Reported as:
Mean · unitless
Change Fractional Anisotropy in the White Sites Along the Audiotory Pathways
unitlessAutologous Stem Cells
Nonresponder Baseline Right0.2 ± 0.05
Nonresponder Baseline Left0.21 ± 0.06
Responder Baseline Right0.22 ± 0.05
Responder Baseline Left0.19 ± 0.06
Nonresponder 12 Month Right0.26 ± 0.06
Nonresponder 12 Month Left0.18 ± 0.05
Responder 12 Month Right0.26 ± 0.06
Responder 12 Month Left0.24 ± 0.06

Adverse events

Collected over 1 year. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Autologous Stem Cells0/11 (0%)0/11 (0%)0/11 (0%)

Baseline characteristics

Age, Continuous
Age, Continuous(months)Autologous Stem Cells
Mean30.27 ± 25.3
Sex: Female, Male
Sex: Female, Male(Participants)Autologous Stem Cells
Female5
Male6
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Autologous Stem Cells
American Indian or Alaska Native0
Asian4
Native Hawaiian or Other Pacific Islander0
Black or African American1
White6
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(Participants)Autologous Stem Cells
United States11
08

Study locations

1 site
  • Florida Hospital
    Orlando, Florida 32803, United States
09

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 9, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02038972
Lead sponsor
James Baumgartner, MD
Collaborators
CBR Systems, Inc.
Responsible party
James Baumgartner, MD (Primary Investigator, AdventHealth) — Sponsor-investigator
First posted
Jan 17, 2014
Start date
Jan 2013
Primary completion
Dec 11, 2015
Completion
Jan 10, 2017
Results posted
Jul 9, 2026
Last update
Jul 9, 2026

Study contacts

James Baumgartner, MD
principal investigator · AdventHealth

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Oct 2023. You cannot join it, but the record below documents what was studied.

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