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Autologous Bone Marrow Harvest and Transplant for Sensorineural Hearing Loss

2022年3月16日 更新者: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.

研究概览

详细说明

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.

研究类型

介入性

注册 (预期的)

10

阶段

  • 阶段2
  • 阶段1

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习地点

    • Florida
      • Orlando、Florida、美国、32803
        • Florida Hospital for Children

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

2年 至 6年 (孩子)

接受健康志愿者

有资格学习的性别

全部

描述

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

学习计划

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

研究是如何设计的?

设计细节

  • 主要用途:治疗
  • 分配:不适用
  • 介入模型:单组作业
  • 屏蔽:无(打开标签)

武器和干预

参与者组/臂
干预/治疗
实验性的: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
其他名称:
  • 细胞疗法

研究衡量的是什么?

主要结果指标

结果测量
措施说明
大体时间
physiological parameter: Blood Pressure
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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

次要结果测量

结果测量
措施说明
大体时间
Auditory Brainstem Response
大体时间: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

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始

2015年10月1日

初级完成 (预期的)

2022年9月1日

研究完成 (预期的)

2022年9月1日

研究注册日期

首次提交

2015年8月19日

首先提交符合 QC 标准的

2015年11月25日

首次发布 (估计)

2015年11月26日

研究记录更新

最后更新发布 (实际的)

2022年3月18日

上次提交的符合 QC 标准的更新

2022年3月16日

最后验证

2022年3月1日

更多信息

与本研究相关的术语

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)?

未定

此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.

Autologous Bone Marrow Infusion的临床试验

3
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