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

The objectives of this study are:

1. To see if autologous human umbilical cord blood treatment is safe for children with acquired hearing loss, and
2. To determine if late functional outcome is improved following autologous human umbilical cord blood treatment for children with acquired hearing loss.

Study Overview

• Status: Withdrawn
• Conditions: Hearing Loss
• Intervention / Treatment: Biological: Autologous Human Umbilical Cord Blood

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.

• Study Type: Interventional
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • Houston, Texas, United States, 77030
      • Children's Memorial Hermann Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 1 year (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Evidence of a moderate to profound sensorineural hearing loss.
2. Normally shaped cochlea, as determined by MRI.
3. The loss must be considered acquired, NOT syndromic.
4. The patient must be fitted for hearing aids of the detection of the loss.
5. Enrollment in a parent/child intervention program.
6. Between 6 weeks and 18 months of age at the time of cord blood infusion.
7. 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:

1. Inability to obtain pertinent medical records.

2. 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.
3. hUCB sample contamination.
4. Participation in a concurrent intervention study.
5. Desire for organ donation in the event of death.
6. 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.
7. Presence of a cochlear implant device.
8. Evidence of a syndrome.
9. Positive test for genetic hearing loss.
10. Evidence of conductive hearing loss.
11. Documented evidence of recurrent middle ear infections (> 5/year).
12. Otitis media at the time of examination.
13. Mild sensorineural hearing loss.
14. Over 18 months at the 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: Biologic; Autologous Cell Injection	Biological: Autologous Human Umbilical Cord Blood; 6 million cells/kg will be administered intravenously at one treatment time point.; Other Names: Autologous Human Umbilical Cord Blood Mononuclear Fraction Cells; Patient's Own Stem Cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Physiologic Outcome	Age appropriate physiologic outcome measures will be recorded pre-treatment, and one year following hUCB treatment	One year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Functional Outcome	Age appropriate Speech-Language assessments will be performed pre-treatment and one year post-treatment.	one year

Collaborators and Investigators

• Sponsor: Aryn Knight
• Collaborators: M.D. Anderson Cancer Center; The University of Texas Health Science Center, Houston; Baylor College of Medicine; The Methodist Hospital Research Institute; Florida Hospital for Children; Cord Blood Registry, Inc.
• Investigators: Principal Investigator: James E. Baumgartner, MD, MHHS, Houston,TX & FL Hospital for Children, Orlando, FL; Principal Investigator: Linda S. Baumgartner, CCC-SLP, LSLS CERT.AVT, Speech Therapists for Children; Principal Investigator: Samir Fakhri, MD, The University of Texas Health Science Center, Houston

Study record dates

Study Major Dates

• Study Start: April 1, 2011
• Primary Completion (Actual): January 1, 2013
• Study Completion (Estimated): April 1, 2016

Study Registration Dates

• First Submitted: April 25, 2011
• First Submitted That Met QC Criteria: April 27, 2011
• First Posted (Estimated): April 28, 2011

Study Record Updates

• Last Update Posted (Actual): March 20, 2024
• Last Update Submitted That Met QC Criteria: March 18, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Children; Stem Cells; Autologous; Hearing Loss
• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Otorhinolaryngologic Diseases; Ear Diseases; Sensation Disorders; Hearing Disorders; Hearing Loss; Deafness
• Other Study ID Numbers: JB IND14312