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Autologous Bone Marrow Harvest and Transplant for Sensorineural Hearing Loss

16 de marzo de 2022 actualizado por: James Baumgartner, MD

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

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.

Descripción general del estudio

Estado

Suspendido

Descripción detallada

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.

Tipo de estudio

Intervencionista

Inscripción (Anticipado)

10

Fase

  • Fase 2
  • Fase 1

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

    • Florida
      • Orlando, Florida, Estados Unidos, 32803
        • Florida Hospital for Children

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

2 años a 6 años (Niño)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

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

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: N / A
  • Modelo Intervencionista: Asignación de un solo grupo
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Autologous bone marrow infusion
One time administration of autologous bone marrow mononuclear cells intravenously, minimum dose of 6 million cells per kg Total nucleated cells.
The subjects autologous bone marrow cells harvested at Florida Hospital will be infused intravenously by gravity
Otros nombres:
  • Terapia basada en células

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
physiological parameter: Blood Pressure
Periodo de tiempo: 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.
Change from baseline to 24 hours after stem cell infusion
physiological parameter: Pulmonary Endothelial Damage
Periodo de tiempo: 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 from baseline to 24 hours post infusion
Change: Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v3.0 for Hepatic Injury
Periodo de tiempo: 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 from baseline to post infusion day 1
Change: Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v3.0 for Neurological status
Periodo de tiempo: Change in baseline to 1 day post infusion
Change in the subject's acute neurologic status will be monitored hourly for 4 hours after infusion. Data recorded include Glasgow Coma Scale (GCS) from infusion to discharge. Grade 3 Central Nervous System (CNS) event as defined in the National Cancer Institute (NCI) Common Toxicity Criteria for Adverse Effects (CTCAE) v4.0 occurring within 12 hours of cellular product infusion will trigger the stopping rules. Other changes temporally related to infusion (those events occurring within 12 hours of infusion) will be considered associated with the protocol and recorded as an adverse event. This will be reported as the number of participants with adverse events related to treatment.
Change in baseline to 1 day post infusion
Incidence of Treatment-Emergent Adverse Events for Pulmonary Status
Periodo de tiempo: Baseline to 24 hours after infusion
Blood-oxygen saturation will be monitored by finger oximeter. Moderate respiratory dysfunction within the first 24 hours post infusion will be considered an adverse event but will not warrant stopping the trial unless recommended by the Data Safety Monitoring Board. In the event of pulmonary dysfunction, standard supportive therapy will be given. Pulmonary symptoms/events corresponding to the Common Toxicity Criteria for Adverse Effects (CTCAE) v3.0 Grade 3 will trigger the stopping rules
Baseline to 24 hours after infusion

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Auditory Brainstem Response
Periodo de tiempo: 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.
Baseline, 1 month, 6 months, and 1 year

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio

1 de octubre de 2015

Finalización primaria (Anticipado)

1 de septiembre de 2022

Finalización del estudio (Anticipado)

1 de septiembre de 2022

Fechas de registro del estudio

Enviado por primera vez

19 de agosto de 2015

Primero enviado que cumplió con los criterios de control de calidad

25 de noviembre de 2015

Publicado por primera vez (Estimar)

26 de noviembre de 2015

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

18 de marzo de 2022

Última actualización enviada que cumplió con los criterios de control de calidad

16 de marzo de 2022

Última verificación

1 de marzo de 2022

Más información

Términos relacionados con este estudio

Plan de datos de participantes individuales (IPD)

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INDECISO

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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