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

This study has suspended participant recruitment.
(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)
Sponsor:
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 System
Florida Hospital for Children Orlando FL
Information provided by (Responsible Party):
James E.Baumgartner, MD, Memorial Hermann Healthcare System
ClinicalTrials.gov Identifier:
NCT01343394
First received: April 25, 2011
Last updated: January 21, 2014
Last verified: January 2014
  Purpose

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.

Condition Intervention Phase
Hearing Loss
Biological: Autologous Human Umbilical Cord Blood
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Safety of Autologous Human Umbilical Cord Blood Mononuclear Fraction to Treat Acquired Hearing Loss in Children

Resource links provided by NLM:


Further study details as provided by Memorial Hermann Healthcare System:

Primary Outcome Measures:
  • Physiologic Outcome [ Time Frame: One year ] [ Designated as safety issue: No ]
    Age appropriate physiologic outcome measures will be recorded pre-treatment, and one year following hUCB treatment


Secondary Outcome Measures:
  • Functional Outcome [ Time Frame: one year ] [ Designated as safety issue: No ]
    Age appropriate Speech-Language assessments will be performed pre-treatment and one year post-treatment.


Estimated Enrollment: 10
Study Start Date: April 2011
Estimated Study Completion Date: April 2016
Primary Completion Date: January 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   6 Weeks to 18 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01343394

Locations
United States, Texas
Children's Memorial Hermann Hospital
Houston, Texas, United States, 77030
Sponsors and Collaborators
James E.Baumgartner, MD
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 System
Florida Hospital for Children Orlando FL
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
  More Information

No publications provided

Responsible Party: James E.Baumgartner, MD, Board-certified Pediatric Neurosurgeon, Memorial Hermann Healthcare System
ClinicalTrials.gov Identifier: NCT01343394     History of Changes
Other Study ID Numbers: JB IND14312
Study First Received: April 25, 2011
Last Updated: January 21, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Memorial Hermann Healthcare System:
Autologous
Stem Cells
Hearing Loss
Children

Additional relevant MeSH terms:
Hearing Loss
Deafness
Hearing Disorders
Ear Diseases
Otorhinolaryngologic Diseases
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms

ClinicalTrials.gov processed this record on July 29, 2014