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.
調査の概要
詳細な説明
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.
研究の種類
介入
入学 (予想される)
10
段階
- フェーズ2
- フェーズ 1
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究場所
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Florida
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Orlando、Florida、アメリカ、32803
- Florida Hospital for Children
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参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
2年～6年 (子)
健康ボランティアの受け入れ
いいえ
受講資格のある性別
全て
説明
Inclusion Criteria:
Evidence of sensorineural hearing loss that is,
- Bilaterally Moderate or Profound in degree
- Symmetrical or asymmetrical configuration
- Sudden or progressive in presentation
- Normally shaped cochlea, as determined by Magnetic Resonance Imaging or computed tomography (CT)
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)
- Fitted for hearing aids no later than six months post detection of loss unless not recommended by treating audiologist or physicians
- Enrollment in a parent/child intervention program
- 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.
- 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:
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.)
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
- Any evidence of active maternal infection during the pregnancy
- Participation in a concurrent intervention study
- Mild hearing loss with no evidence of moderate of severe loss
- 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.
- Evidence of conductive hearing loss
- Documented recurrent middle ear infections which are frequent (>5 per year)
- Otitis media at the time of examination
- Before 2 years from identification of hearing loss at time of infusion
- After 4 years from identification of hearing loss at time of infusion
Diagnosis of the following syndromic cause for hearing loss
- CHARGE
- Waardenburg
- Brachio-Oto-Renal
- Pendred
- Alport
- Treacher-Collins
- Usher
- Stickler Syndrome
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的：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.
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The subjects autologous bone marrow cells harvested at Florida Hospital will be infused intravenously by gravity
他の名前：
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この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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physiological parameter: Blood Pressure
時間枠：Change from baseline to 24 hours after stem cell infusion
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Assessing change from baseline systolic blood pressure to post stem cell infusion systolic blood pressure.
The metric for summarizing measurements is millimeters of mercury.
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Change from baseline to 24 hours after stem cell infusion
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physiological parameter: Pulmonary Endothelial Damage
時間枠：Change from baseline to 24 hours post infusion
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Measured by the number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
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Change from baseline to 24 hours post infusion
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Change: Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v3.0 for Hepatic Injury
時間枠：Change from baseline to post infusion day 1
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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.
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Change from baseline to post infusion day 1
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Change: Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v3.0 for Neurological status
時間枠：Change in baseline to 1 day post infusion
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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.
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Change in baseline to 1 day post infusion
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Incidence of Treatment-Emergent Adverse Events for Pulmonary Status
時間枠：Baseline to 24 hours after infusion
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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
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Baseline to 24 hours after infusion
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Auditory Brainstem Response
時間枠：Baseline, 1 month, 6 months, and 1 year
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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.
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Baseline, 1 month, 6 months, and 1 year
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協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
スポンサー
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始
2015年10月1日
一次修了 (予想される)
2022年9月1日
研究の完了 (予想される)
2022年9月1日
試験登録日
最初に提出
2015年8月19日
QC基準を満たした最初の提出物
2015年11月25日
最初の投稿 (見積もり)
2015年11月26日
学習記録の更新
投稿された最後の更新 (実際)
2022年3月18日
QC基準を満たした最後の更新が送信されました
2022年3月16日
最終確認日
2022年3月1日
詳しくは
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。