Autologous Bone Marrow Harvest and Transplant for Sensorineural Hearing Loss

Brief Title

Autologous Bone Marrow Harvest and Transplant for Sensorineural Hearing Loss

Official Title

Safety of Infusion of Autologous Human Bone Marrow Mononuclear Fraction in Children With Sensorineural Hearing Loss

Brief Summary

      Autologous human bone marrow mononuclear fraction (BMMF) will be harvested and given to
      children with bilateral moderate to severe sensorineural hearing loss. The aim is to
      determine if bone marrow mononuclear fraction (BMMF) infusion is safe, feasible, improves
      inner ear function, audition, and language development.
    

Detailed Description

      Autologous human bone marrow mononuclear fraction (BMMF) will be given to children with
      bilateral moderate to severe sensorineural hearing loss.

      Subjects will come to Orlando for pretesting to include an Magnetic Resonance Imaging (MRI),
      Auditory brainstem response (ABR), blood work: Complete metabolic panel (CMP), Complete blood
      count (CBC), Hepatic Function Panel, Prothrombin (PT), Partial thromboplastin time (PTT),
      International normalized ration (INR), Chest Xray, and a Speech and Language Evaluation.

      After pretesting, the subjects will undergo a bone marrow harvest and then receive their
      autologous bone marrow mononuclear fraction (BMMF) intravenously. The subjects will then be
      monitored for 24 hours post infusion. After 24 hours, the subject will undergo repeat blood
      work and a chest x ray. Subjects will then be discharged home. Subjects will follow up in
      Orlando at 1 month, 6 months and 1 year post infusion. Follow up testing will repeat the
      exams performed at pretesting.
    

Study Phase

Phase 1/Phase 2

Study Type

Interventional

Primary Outcome

physiological parameter: Blood Pressure

Secondary Outcome

 Auditory Brainstem Response

Condition

Sensorineural Hearing Loss

Intervention

Autologous Bone Marrow Infusion

Study Arms / Comparison Groups

 Autologous bone marrow infusion

Description:  One time administration of autologous bone marrow mononuclear cells intravenously, minimum dose of 6 million cells per kg Total nucleated cells.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Genetic

Estimated Enrollment

10

Start Date

October 2015

Completion Date

September 2018

Primary Completion Date

September 2018

Eligibility Criteria

        Inclusion Criteria:

          1. Evidence of sensorineural hearing loss that is,

               -  Bilaterally Moderate or Profound in degree

               -  Symmetrical or asymmetrical configuration

               -  Sudden or progressive in presentation

          2. Normally shaped cochlea, as determined by Magnetic Resonance Imaging or computed
             tomography (CT)

          3. The loss must be considered:

               -  Acquired

               -  Unknown with genetic testing negative. (Genetic testing is not required for
                  Cytomegalovirus (CMV) positive children due to Cytomegalovirus (CMV) known to be
                  number one cause of hearing loss)

          4. Fitted for hearing aids no later than six months post detection of loss unless not
             recommended by treating audiologist or physicians

          5. Enrollment in a parent/child intervention program

          6. Age 2 years - 6 years old at time of infusion with 2 to 4 years of time elapsed since
             diagnosis of hearing loss at the time of bone marrow mononuclear fraction (BMMF)
             infusion.

          7. 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.

        Exclusion Criteria:

          1. 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 visit.)

          2. Known history of:

               -  Recently treated (ear or any infections) 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 Alanine Transaminase
                  (SGPT) > 150 U/L, and or Total Bilirubin > 1.3 mg/dL

               -  Malignancy

               -  Immunosuppression as defined by White Blood Cell (WBC) < 3,000 at admission

               -  Human Immunodeficiency Virus (HIV)

               -  Hepatitis B

               -  Hepatitis C

               -  Pneumonia, or chronic lung disease requiring oxygen

          3. Any evidence of active maternal infection during the pregnancy

          4. Participation in a concurrent intervention study

          5. Mild hearing loss with no evidence of moderate of severe loss

          6. Unwillingness or inability to stay for 4 days following infusion (should problems
             arise following the infusion) and to return for the one month, six month and one year
             follow-up visits.

          7. Evidence of conductive hearing loss

          8. Documented recurrent middle ear infections which are frequent (>5 per year)

          9. Otitis media at the time of examination

         10. Before 2 years from identification of hearing loss at time of infusion

         11. After 4 years from identification of hearing loss at time of infusion

         12. Diagnosis of the following syndromic cause for hearing loss

               -  CHARGE

               -  Waardenburg

               -  Brachio-Oto-Renal

               -  Pendred

               -  Alport

               -  Treacher-Collins

               -  Usher

               -  Stickler Syndrome
      

Gender

All

Ages

2 Years - 6 Years

Accepts Healthy Volunteers

No

Contacts

James Baumgartner, MD, , 

Location Countries

United States

Location Countries

United States

NCT ID

NCT02616172

Organization ID

695389

Responsible Party

Sponsor-Investigator

Study Sponsor

James Baumgartner, MD

Study Sponsor

James Baumgartner, MD, Principal Investigator, AdventHealth

Verification Date

February 2018