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

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.

Study Overview

• Status: Completed
• Conditions: Sensorineural Hearing Loss
• Intervention / Treatment: Genetic: Autologous Stem Cells

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 Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Orlando, Florida, United States, 32803
      • Florida Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 2 years (Child)
• Accepts Healthy Volunteers: No

Description

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

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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; The subjects autologous stem cells banked at Cord Blood Registry will be infused intravenously by gravity.; Other Names: Cell based therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	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
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

Collaborators and Investigators

• Sponsor: James Baumgartner, MD
• Collaborators: CBR Systems, Inc.
• Investigators: Principal Investigator: James Baumgartner, MD, AdventHealth

Study record dates

Study Major Dates

• Study Start: January 1, 2013
• Primary Completion (Actual): December 11, 2015
• Study Completion (Actual): January 10, 2017

Study Registration Dates

• First Submitted: January 8, 2014
• First Submitted That Met QC Criteria: January 16, 2014
• First Posted (Estimated): January 17, 2014

Study Record Updates

• Last Update Posted (Actual): July 9, 2026
• Last Update Submitted That Met QC Criteria: June 12, 2026
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Otorhinolaryngologic Diseases; Sensation Disorders; Ear Diseases; Hearing Loss; Hearing Disorders; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hearing Loss, Sensorineural; Therapeutics; Biological Therapy; Cell- and Tissue-Based Therapy
• Other Study ID Numbers: 434269

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED