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Autologous Bone Marrow Harvest and Transplant for Sensorineural Hearing Loss

16 maart 2022 bijgewerkt door: 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.

Studie Overzicht

Toestand

Geschorst

Gedetailleerde beschrijving

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.

Studietype

Ingrijpend

Inschrijving (Verwacht)

10

Fase

  • Fase 2
  • Fase 1

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

    • Florida
      • Orlando, Florida, Verenigde Staten, 32803
        • Florida Hospital for Children

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

2 jaar tot 6 jaar (Kind)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: NVT
  • Interventioneel model: Opdracht voor een enkele groep
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: 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
Andere namen:
  • Celgebaseerde therapie

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
physiological parameter: Blood Pressure
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Auditory Brainstem Response
Tijdsspanne: 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

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start

1 oktober 2015

Primaire voltooiing (Verwacht)

1 september 2022

Studie voltooiing (Verwacht)

1 september 2022

Studieregistratiedata

Eerst ingediend

19 augustus 2015

Eerst ingediend dat voldeed aan de QC-criteria

25 november 2015

Eerst geplaatst (Schatting)

26 november 2015

Updates van studierecords

Laatste update geplaatst (Werkelijk)

18 maart 2022

Laatste update ingediend die voldeed aan QC-criteria

16 maart 2022

Laatst geverifieerd

1 maart 2022

Meer informatie

Termen gerelateerd aan deze studie

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

ONBESLIST

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