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 Information
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
Administrative Informations
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