Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02038972 |
Recruitment Status :
Completed
First Posted : January 17, 2014
Last Update Posted : March 5, 2018
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Condition or disease | Intervention/treatment | Phase |
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Sensorineural Hearing Loss | Genetic: Autologous Stem Cells | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 11 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss |
Study Start Date : | January 2013 |
Actual Primary Completion Date : | December 11, 2015 |
Actual Study Completion Date : | January 10, 2017 |

Arm | Intervention/treatment |
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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.
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Genetic: Autologous Stem Cells
The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity.
Other Name: Cell based therapy |
- Safety of Autologous Stem Cell Infusion [ Time Frame: 1 year ]
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.
- Inner Ear Function, Audition, and Language Development [ Time Frame: 1 year ]To determine if autologous hUBC transplantation in children with hearing loss improves inner ear function, audition and language development.

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Ages Eligible for Study: | 6 Weeks to 6 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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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.
Exclusion Criteria
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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.)
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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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02038972
United States, Florida | |
Florida Hospital | |
Orlando, Florida, United States, 32803 |
Principal Investigator: | James Baumgartner, MD | AdventHealth |
Responsible Party: | James Baumgartner, MD, Primary Investigator, AdventHealth |
ClinicalTrials.gov Identifier: | NCT02038972 |
Other Study ID Numbers: |
434269 |
First Posted: | January 17, 2014 Key Record Dates |
Last Update Posted: | March 5, 2018 |
Last Verified: | August 2017 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Hearing Loss Deafness Hearing Loss, Sensorineural Hearing Disorders Ear Diseases |
Otorhinolaryngologic Diseases Sensation Disorders Neurologic Manifestations Nervous System Diseases |