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Safety of Autologous Human Umbilical Cord Blood Mononuclear Fraction to Treat Acquired Hearing Loss in Children

Primary Purpose

Hearing Loss

Status

Suspended

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Autologous Human Umbilical Cord Blood

About this trial

This is an interventional treatment trial for Hearing Loss focused on measuring Autologous, Stem Cells, Hearing Loss, Children

Eligibility Criteria

6 Weeks - 18 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Evidence of a moderate to profound sensorineural hearing loss.
Normally shaped cochlea, as determined by MRI.
The loss must be considered acquired, NOT syndromic.
The patient must be fitted for hearing aids of the detection of the loss.
Enrollment in a parent/child intervention program.
Between 6 weeks and 18 months of age at the time of cord blood infusion.
Ability of child and caregiver to travel to Houston for treatment and all follow-up appointments. (Patient's family is responsible for the cost of travel to and lodging in Houston).

Exclusion Criteria:

Inability to obtain pertinent medical records.
Known history or
- Recently treated ear or other infection.
- Renal disease.
- Hepatic disease.
- Malignancy.
- HIV.
- Immunosuppression (WBC < 3,000).
- Evidence of an extensive stroke (> 100ml).
- Pneumonia, or chronic lung disease.
hUCB sample contamination.
Participation in a concurrent intervention study.
Desire for organ donation in the event of death.
Unwillingness or inability to stay 4 days following hUCB infusion, and to return for the one month, six month and one year follow-up visits.
Presence of a cochlear implant device.
Evidence of a syndrome.
Positive test for genetic hearing loss.
Evidence of conductive hearing loss.
Documented evidence of recurrent middle ear infections (> 5/year).
Otitis media at the time of examination.
Mild sensorineural hearing loss.
Over 18 months at the time of infusion.

Sites / Locations

Children's Memorial Hermann Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Biologic; Autologous Cell Injection

Arm Description

Outcomes

Primary Outcome Measures

Physiologic Outcome

Age appropriate physiologic outcome measures will be recorded pre-treatment, and one year following hUCB treatment

Secondary Outcome Measures

Functional Outcome

Age appropriate Speech-Language assessments will be performed pre-treatment and one year post-treatment.

Full Information

NCT ID

NCT01343394

First Posted

April 25, 2011

Last Updated

January 21, 2014

Sponsor

James E.Baumgartner, MD

Collaborators

Cord Blood Registry, Inc., Speech Therapists for Children, The University of Texas Health Science Center, Houston, M.D. Anderson Cancer Center, Baylor College of Medicine, The Methodist Hospital Research Institute, Florida Hospital for Children

1. Study Identification

Unique Protocol Identification Number

NCT01343394

Brief Title

Safety of Autologous Human Umbilical Cord Blood Mononuclear Fraction to Treat Acquired Hearing Loss in Children

Official Title

Safety of Autologous Human Umbilical Cord Blood Mononuclear Fraction to Treat Acquired Hearing Loss in Children

Study Type

Interventional

2. Study Status

Record Verification Date

January 2014

Overall Recruitment Status

Suspended

Why Stopped

UT-IRB insists on protocol design change -Not rel to any pt. safety/non-compliance-Recruiting halted at UT/MHHS but may resume at FLHosp for Children-OrlandoFL

Study Start Date

April 2011 (undefined)

Primary Completion Date

January 2013 (Actual)

Study Completion Date

April 2016 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

James E.Baumgartner, MD

Collaborators

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The objectives of this study are: To see if autologous human umbilical cord blood treatment is safe for children with acquired hearing loss, and To determine if late functional outcome is improved following autologous human umbilical cord blood treatment for children with acquired hearing loss.

Detailed Description

Acquired sensorineural hearing loss is characterized by a loss of functioning hair cells in the Organ of Corti, with greater hair cell loss correlating with more severe hearing impairment. Children with sensorineural hearing loss experience difficulty developing normal language which usually leads to poor academic and social development. Currently, there are no reparative therapeutic options available, and treatments are designed to augment the diminished function of the injured Organ of Corti. Pre-clinical data suggest progenitor cell infusions may enhance intrinsic repair mechanisms in the Organ of Corti which may restore hair cells. This treatment could ultimately lead to hearing improvement. Human umbilical cord blood (hUCB) is an available, autologous, stored progenitor cell population available for potential therapeutic use. The primary objective of this study is to determine the safety of autologous hUCB infusion in children with acquired hearing loss. The secondary objective is to determine if functional, physiologic and anatomic outcomes are improved following hUCB treatment in this patient population.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hearing Loss

Keywords

Autologous, Stem Cells, Hearing Loss, Children

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Biologic; Autologous Cell Injection

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

Autologous Human Umbilical Cord Blood

Other Intervention Name(s)

Autologous Human Umbilical Cord Blood Mononuclear Fraction Cells, Patient's Own Stem Cells

Intervention Description

6 million cells/kg will be administered intravenously at one treatment time point.

Primary Outcome Measure Information:

Title

Physiologic Outcome

Description

Age appropriate physiologic outcome measures will be recorded pre-treatment, and one year following hUCB treatment

Time Frame

One year

Secondary Outcome Measure Information:

Title

Functional Outcome

Description

Age appropriate Speech-Language assessments will be performed pre-treatment and one year post-treatment.

Time Frame

one year

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Weeks

Maximum Age & Unit of Time

18 Months

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Evidence of a moderate to profound sensorineural hearing loss. Normally shaped cochlea, as determined by MRI. The loss must be considered acquired, NOT syndromic. The patient must be fitted for hearing aids of the detection of the loss. Enrollment in a parent/child intervention program. Between 6 weeks and 18 months of age at the time of cord blood infusion. Ability of child and caregiver to travel to Houston for treatment and all follow-up appointments. (Patient's family is responsible for the cost of travel to and lodging in Houston). Exclusion Criteria: Inability to obtain pertinent medical records. Known history or Recently treated ear or other infection. Renal disease. Hepatic disease. Malignancy. HIV. Immunosuppression (WBC < 3,000). Evidence of an extensive stroke (> 100ml). Pneumonia, or chronic lung disease. hUCB sample contamination. Participation in a concurrent intervention study. Desire for organ donation in the event of death. Unwillingness or inability to stay 4 days following hUCB infusion, and to return for the one month, six month and one year follow-up visits. Presence of a cochlear implant device. Evidence of a syndrome. Positive test for genetic hearing loss. Evidence of conductive hearing loss. Documented evidence of recurrent middle ear infections (> 5/year). Otitis media at the time of examination. Mild sensorineural hearing loss. Over 18 months at the time of infusion.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

James E. Baumgartner, MD

Organizational Affiliation

MHHS, Houston,TX & FL Hospital for Children, Orlando, FL

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Linda S. Baumgartner, CCC-SLP, LSLS CERT.AVT

Organizational Affiliation

Speech Therapists for Children

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Samir Fakhri, MD

Organizational Affiliation

The University of Texas Health Science Center, Houston

Official's Role

Principal Investigator

Facility Information:

Facility Name

Children's Memorial Hermann Hospital

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

12. IPD Sharing Statement

Learn more about this trial

Safety of Autologous Human Umbilical Cord Blood Mononuclear Fraction to Treat Acquired Hearing Loss in Children

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