Clinical Trial: Autologous Bone Marrow Harvest and Transplant for Sensorineural Hearing Loss

Study Status: Suspended
Recruit Status: Suspended
Study Type: Interventional

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 Summary:

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.

Current Primary Outcome:

• physiological parameter: Blood Pressure [ Time Frame: Change from baseline to 24 hours after stem cell infusion ]
  Assessing change from baseline systolic blood pressure to post stem cell infusion systolic blood pressure. The metric for summarizing measurements is millimeters of mercury.
• physiological parameter: Pulmonary Endothelial Damage [ Time Frame: Change from baseline to 24 hours post infusion ]
  Measured by the number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
• Change: Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v3.0 for Hepatic Injury [ Time Frame: Change from baseline to post infusion day 1 ]
  The reticuloendothelial system can sequester immature blood elements, theoretically resulting in hepatic injury. An acute elevation of the aspartate transaminase (AST) and Alanine Aminotransferase test (ALT) hepatic enzymes >5.0 - 20.0 x upper limit normal (ULN) in the first 24 hours post infusion will trigger the stopping rules. This level corresponds to the Common Toxicity Criteria for Adverse Effects (CTCAE) v4.0 Grade 3 adverse event. It is unlikely that "end vessel" microthrombosis would occur in the liver due to the dual blood supply of the liver and the lung is the first pass organ. This will be reported as the number of participants with abnormal laboratory values and adverse events related to treatment.
• Change: Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v3.0 for Neur
  
  

  Original Primary Outcome: Same as current
  
  

  Current Secondary Outcome: Auditory Brainstem Response [ Time Frame: Baseline, 1 month, 6 months, and 1 year ]

  Audiometry, to-acoustic emissions and Auditory Brainstem Response will be used to assess the physiologic integrity of the neural structures which are critical to normal audition and speech. Changes in these areas will be evaluated by repeating the measures all follow-up visits.
  
  
  

  Original Secondary Outcome: Same as current
  
  Information By: Florida Hospital
  

  Dates:
  Date Received: August 19, 2015
  Date Started: October 2015
  Date Completion: September 2018
  Last Updated: August 10, 2016
  Last Verified: August 2016