Clinical trial NCT01343394: 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
Overall status: WITHDRAWN
Phase: PHASE1
Conditions: Hearing Loss
Keywords: Autologous
Stem Cells
Hearing Loss
Children
Lead sponsor: Memorial Hermann Health System
Sponsor class: OTHER
Collaborators: name: Cord Blood Registry, Inc.
name: The University of Texas Health Science Center, Houston
name: M.D. Anderson Cancer Center
name: Baylor College of Medicine
name: The Methodist Hospital Research Institute
name: Florida Hospital for Children
First posted: 2011-04-28
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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 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.

Primary outcomes:
measure: Physiologic Outcome
timeFrame: One year
Secondary outcomes:
measure: Functional Outcome
timeFrame: one year
Other outcomes:
Locations:
city: Houston
country: United States
facility: Children's Memorial Hermann Hospital
state: Texas
Study officials:
affiliation: MHHS, Houston,TX & FL Hospital for Children, Orlando, FL
name: James E. Baumgartner, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Speech Therapists for Children
name: Linda S. Baumgartner, CCC-SLP, LSLS CERT.AVT
role: PRINCIPAL_INVESTIGATOR
affiliation: The University of Texas Health Science Center, Houston
name: Samir Fakhri, MD
role: PRINCIPAL_INVESTIGATOR
References: