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

NCT02038972 | Completed Phase 1 DMC

• 1 other identifier: 434269
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

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.

Conditions

Sensorineural Hearing Loss

Outcome Measures

Primary Outcomes (1)

• Safety of Autologous Stem Cell Infusion

  To determine if autologous human umbilical cord blood (hUBC) infusion in children with hearing loss is safe and feasible. Infusion related toxicity as measured by: i. hemodynamic instability: An adverse event will be defined as a sustained (\> 10 minutes) \>20% decrease in MAP. ii. acute lung injury: Chest X-ray will be done at baseline and at 1 day post infusion to assess for polymorphonuclear infiltrates iii. hepatic injury/toxicity: Hepatic panel will be performed at baseline and 1 day post infusion. Injury is defined as acute elevation of the AST/ALT hepatic enzymes \> 900 U/dl in the first 24 hours post infusion iv. renal injury/insufficiency: CMP will be performed at baseline and 1 day after infusion v. exacerbation of neurological status: defined as a change in Glasgow Coma Scale, pupillary size/reactivity, motor/sensory evaluation of extremities, and seizure activity from infusion to discharge.

  1 year

Secondary Outcomes (1)

• Inner Ear Function, Audition, and Language Development

  1 year

Study Arms (1)

Autologous Stem Cells

EXPERIMENTAL

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.

Genetic: Autologous Stem Cells

Interventions

Autologous Stem Cells GENETIC

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

Also known as: Cell based therapy

Autologous Stem Cells

Eligibility Criteria

• Age: 6 Weeks - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• Normally shaped cochlea, as determined by MRI
• The loss must be considered:
• Acquired
• Unknown with a negative genetic test.
• Fitted for hearing aids no later than six months post detection of loss.
• Enrollment in a parent/child intervention program
• 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.
• 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.

You may not qualify if:

• 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.)
• 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
• hUBC sample contamination

Sponsors & Collaborators

• James Baumgartner, MD: lead
• CBR Systems, Inc.: collaborator

Study Sites (1)

Florida Hospital

Orlando, Florida, 32803, United States

Location

MeSH Terms

Conditions

Hearing Loss, Sensorineural

Interventions

Cell- and Tissue-Based Therapy

Condition Hierarchy (Ancestors)

Hearing Loss; Hearing Disorders; Ear Diseases; Otorhinolaryngologic Diseases; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• James Baumgartner, MD

  AdventHealth

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Primary Investigator

Study Record Dates

• First Submitted: January 8, 2014
• First Posted: January 17, 2014
• Study Start: January 1, 2013
• Primary Completion: December 11, 2015
• Study Completion: January 10, 2017
• Last Updated: March 5, 2018

Record last verified: 2017-08

Locations

US (1)