Sensorineural Hearing LossPhase 1/2NCT02616172
Autologous Bone Marrow Harvest and Transplant for Sensorineural Hearing Loss
A Phase 1/2 study testing Autologous Bone Marrow Infusion in people with Sensorineural Hearing Loss, run by James Baumgartner, MD at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 6 years
- Conditions the study is for
- Sensorineural Hearing Loss
- What is being tested?
- Autologous Bone Marrow Infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2015to Sep 2022
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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:
Show all 12Show fewer
- 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.
Who cannot join
- 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
Show all 30Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Florida Hospital for Children
Orlando, Florida, 32803
About this study
Official title: 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.
Read the detailed description
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.
- Conditions
- Sensorineural Hearing Loss
- Being tested
- Autologous Bone Marrow Infusion
- Medical subject headings
- Hearing Loss, Sensorineural
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Bone Marrow InfusionGenetic
Also called Cell based therapy
The subjects autologous bone marrow cells harvested at Florida Hospital will be infused intravenously by gravity
| Study group | Role | What they receive |
|---|---|---|
| Autologous bone marrow infusion | Experimental | One time administration of autologous bone marrow mononuclear cells intravenously, minimum dose of 6 million cells per kg Total nucleated cells. Receives: Genetic: Autologous Bone Marrow Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
physiological parameter: Blood Pressure
Assessing change from baseline systolic blood pressure to post stem cell infusion systolic blood pressure. The metric for summarizing measurements is millimeters of mercury.
Measured: Change from baseline to 24 hours after stem cell infusion
physiological parameter: Pulmonary Endothelial Damage
Measured by the number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
Measured: 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
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.
Measured: 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
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.
Measured: Change in baseline to 1 day post infusion
Incidence of Treatment-Emergent Adverse Events for Pulmonary Status
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
Measured: Baseline to 24 hours after infusion
Secondary outcomes (1)
Auditory Brainstem Response
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.
Measured: Baseline, 1 month, 6 months, and 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2015 (estimated)
- Study startOct 2015
- Primary completionSep 2022 (estimated)
- Study completionSep 2022 (estimated)
Why it stopped: Restructuring Cell Lab
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sensorineural Hearing LossStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- James Baumgartner, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, James Baumgartner, MD, AdventHealth
- Investigators
- James Baumgartner, MD, Principal investigator, AdventHealth
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02616172
- Sponsor's protocol ID
- 695389
- Status last verified
- Mar 2022
- First submitted
- 19 Aug 2015
- Last updated
- 18 Mar 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety of Infusion of Autologous Human Bone Marrow Mononuclear Fraction in Children With Sensorineural Hearing Loss
- ClinicalTrials.gov ID
- NCT02616172
- Acronym / sponsor ID
- 695389
- Conditions
- Sensorineural Hearing Loss
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Restructuring Cell Lab
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- James Baumgartner, MDUniversity, hospital or other
- Study start
- Oct 2015
- Primary completion
- Sep 2022 (estimated)
- Study completion
- Sep 2022 (estimated)
- First posted
- 26 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous bone marrow infusion | Experimental | Genetic: Autologous Bone Marrow InfusionOne time administration of autologous bone marrow mononuclear cells intravenously, minimum dose of 6 million cells per kg Total nucleated cells. |
Who may qualify
- Ages
- 2 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- Evidence of sensorineural hearing loss that is,
- Normally shaped cochlea, as determined by Magnetic Resonance Imaging or computed tomography (CT)
- The loss must be considered:
- 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
Key exclusion criteria (30 listed)
- Inability to obtain all pertinent medical records:
- Known history of:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Bone Marrow InfusionAlso: Cell based therapyThe subjects autologous bone marrow cells harvested at Florida Hospital will be infused intravenously by gravity | Genetic | Autologous bone marrow infusion |
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 Neurological status
Time frame: 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.
Incidence of Treatment-Emergent Adverse Events for Pulmonary Status
Time frame: 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
1 secondary outcome
Auditory Brainstem Response
Time frame: Baseline, 1 month, 6 months, and 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2022.
- Individual participant dataSharing planned: Undecided
Related trials for Sensorineural Hearing Loss
RecruitingObservational study
Retrospective Analysis of Speech Performance Using the HiRes Ultra and HiRes Ultra 3D Cochlear Implant
Cochlear Hearing Loss
RecruitingObservational study
Retrospectively Investigating Longitudinal Outcomes in MED-EL SONATA 2 Cochlear Implant Recipients.
Cochlear Implantation, Sensorineural Hearing Loss
Enrolling by invitationObservational study
Post-Market Registry Study for Vibrant Hearing Solutions
Conductive Hearing Loss, Mixed Hearing Loss
Enrolling by invitationObservational study
Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
Otoferlin Gene-mediated Hearing Loss
Active, not recruiting
Performance of a Remote Microphone Compared to Hearing Aids Alone
Hearing Loss, Bilateral Sensorineural
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James Baumgartner, MD principal investigator
AdventHealth
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Autologous Bone Marrow Infusion in people with Sensorineural Hearing Loss, run by James Baumgartner, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 30 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 18 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
physiological parameter: Blood Pressure (time frame: Change from baseline to 24 hours after stem cell infusion). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Mar 2022.