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

NCT ID: NCT02038972

Last Updated: 2018-03-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-01-31
• Study Completion Date: 2017-01-10

Brief 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.

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Autologous Stem Cells

Intervention Type: GENETIC

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

Interventions

Autologous Stem Cells

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

Intervention Type: GENETIC

Other Intervention Names

Cell based therapy

Eligibility Criteria

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

• Minimum Eligible Age: 6 Weeks
• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CBR Systems, Inc.

OTHER

Sponsor Role: collaborator

James Baumgartner, MD

OTHER

Sponsor Role: lead

Responsible Party

James Baumgartner, MD

Primary Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

James Baumgartner, MD

Role: PRINCIPAL_INVESTIGATOR

AdventHealth

Locations

Florida Hospital

Orlando, Florida, United States

Countries

United States

Other Identifiers

434269

Identifier Type: -

Identifier Source: org_study_id