Hearing LossPhase 1NCT01343394
Safety of Autologous Human Umbilical Cord Blood Mononuclear Fraction to Treat Acquired Hearing Loss in Children
A Phase 1 study testing Autologous Human Umbilical Cord Blood in people with Hearing Loss, run by Aryn Knight at 1 site in United States.
Trial summary
The objectives of this study are: 1. To see if autologous human umbilical cord blood treatment is safe for children with acquired hearing loss, and 2. To determine if late functional outcome is improved following autologous human umbilical cord blood treatment for children with acquired hearing loss. Read more about this study
- Who is it for?
- Ages 6 weeks to 18 months
- Conditions the study is for
- Hearing Loss
- What is being tested?
- Autologous Human Umbilical Cord Blood
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2011to Apr 2016
- Phase
- Phase 1
- 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 6 weeks to 18 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Evidence of a moderate to profound sensorineural hearing loss.
- Normally shaped cochlea, as determined by MRI.
- The loss must be considered acquired, NOT syndromic.
- The patient must be fitted for hearing aids of the detection of the loss.
- Enrollment in a parent/child intervention program.
- Between 6 weeks and 18 months of age at the time of cord blood infusion.
- Ability of child and caregiver to travel to Houston for treatment and all follow-up appointments. (Patient's family is responsible for the cost of travel to and lodging in Houston).
Who cannot join
- Inability to obtain pertinent medical records.
- Known history or
- Recently treated ear or other infection.
- Renal disease.
- Hepatic disease.
- Malignancy.
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- HIV.
- Immunosuppression (WBC < 3,000).
- Evidence of an extensive stroke (> 100ml).
- Pneumonia, or chronic lung disease.
- hUCB sample contamination.
- Participation in a concurrent intervention study.
- Desire for organ donation in the event of death.
- Unwillingness or inability to stay 4 days following hUCB infusion, and to return for the one month, six month and one year follow-up visits.
- Presence of a cochlear implant device.
- Evidence of a syndrome.
- Positive test for genetic hearing loss.
- Evidence of conductive hearing loss.
- Documented evidence of recurrent middle ear infections (> 5/year).
- Otitis media at the time of examination.
- Mild sensorineural hearing loss.
- Over 18 months at the time of infusion.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Memorial Hermann Hospital
Houston, Texas, 77030
About this study
The objectives of this study are:
- To see if autologous human umbilical cord blood treatment is safe for children with acquired hearing loss, and
- To determine if late functional outcome is improved following autologous human umbilical cord blood treatment for children with acquired hearing loss.
Read the detailed description
Acquired sensorineural hearing loss is characterized by a loss of functioning hair cells in the Organ of Corti, with greater hair cell loss correlating with more severe hearing impairment. Children with sensorineural hearing loss experience difficulty developing normal language which usually leads to poor academic and social development. Currently, there are no reparative therapeutic options available, and treatments are designed to augment the diminished function of the injured Organ of Corti.
Pre-clinical data suggest progenitor cell infusions may enhance intrinsic repair mechanisms in the Organ of Corti which may restore hair cells. This treatment could ultimately lead to hearing improvement. Human umbilical cord blood (hUCB) is an available, autologous, stored progenitor cell population available for potential therapeutic use. The primary objective of this study is to determine the safety of autologous hUCB infusion in children with acquired hearing loss. The secondary objective is to determine if functional, physiologic and anatomic outcomes are improved following hUCB treatment in this patient population.
- Conditions
- Hearing Loss
- Being tested
- Autologous Human Umbilical Cord Blood
- Keywords
- Autologous, Stem Cells, Hearing Loss, Children
- Medical subject headings
- Hearing Loss
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Human Umbilical Cord BloodBiological
Also called Autologous Human Umbilical Cord Blood Mononuclear Fraction Cells, Patient's Own Stem Cells
6 million cells/kg will be administered intravenously at one treatment time point.
| Study group | Role | What they receive |
|---|---|---|
| Biologic; Autologous Cell Injection | Experimental | Not described Receives: Biological: Autologous Human Umbilical Cord Blood |
What is being measured
The primary outcome is the main question the study is designed to answer.
Physiologic Outcome
Age appropriate physiologic outcome measures will be recorded pre-treatment, and one year following hUCB treatment
Measured: One year
Secondary outcomes (1)
Functional Outcome
Age appropriate Speech-Language assessments will be performed pre-treatment and one year post-treatment.
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2011 (estimated)
- Study startApr 2011
- Primary completionJan 2013
- Study completionApr 2016 (estimated)
Why it stopped: UT-IRB insists on protocol design change -Not rel to any pt. safety/non-compliance-Recruiting halted at UT/MHHS but may resume at FLHosp for Children-OrlandoFL
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aryn Knight
- Sponsor type
- University, hospital or other
- Collaborators
- Cord Blood Registry, Inc., The University of Texas Health Science Center, Houston, M.D. Anderson Cancer Center, Baylor College of Medicine, The Methodist Hospital Research Institute, Florida Hospital for Children
- Responsible party
- Sponsor-investigator, Aryn Knight, Memorial Hermann Health System
- Investigators
- James E. Baumgartner, MD, Principal investigator, MHHS, Houston,TX & FL Hospital for Children, Orlando, FLLinda S. Baumgartner, CCC-SLP, LSLS CERT.AVT, Principal investigator, Speech Therapists for ChildrenSamir Fakhri, MD, Principal investigator, The University of Texas Health Science Center, Houston
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01343394
- Sponsor's protocol ID
- JB IND14312
- Status last verified
- Mar 2024
- First submitted
- 25 Apr 2011
- Last updated
- 20 Mar 2024
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety of Autologous Human Umbilical Cord Blood Mononuclear Fraction to Treat Acquired Hearing Loss in Children
- ClinicalTrials.gov ID
- NCT01343394
- Acronym / sponsor ID
- JB IND14312
- Conditions
- Hearing Loss
- Keywords
- Autologous, Stem Cells, Hearing Loss, Children
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: UT-IRB insists on protocol design change -Not rel to any pt. safety/non-compliance-Recruiting halted at UT/MHHS but may resume at FLHosp for Children-OrlandoFL
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Aryn KnightUniversity, hospital or other
- Collaborators
- Cord Blood Registry, Inc., The University of Texas Health Science Center, Houston, M.D. Anderson Cancer Center, Baylor College of Medicine, The Methodist Hospital Research Institute, Florida Hospital for Children
- Study start
- Apr 2011
- Primary completion
- Jan 2013 (actual)
- Study completion
- Apr 2016 (estimated)
- First posted
- 28 Apr 2011 (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.
The objectives of this study are:
- To see if autologous human umbilical cord blood treatment is safe for children with acquired hearing loss, and
- To determine if late functional outcome is improved following autologous human umbilical cord blood treatment for children with acquired hearing loss.
Detailed description
Acquired sensorineural hearing loss is characterized by a loss of functioning hair cells in the Organ of Corti, with greater hair cell loss correlating with more severe hearing impairment. Children with sensorineural hearing loss experience difficulty developing normal language which usually leads to poor academic and social development. Currently, there are no reparative therapeutic options available, and treatments are designed to augment the diminished function of the injured Organ of Corti.
Pre-clinical data suggest progenitor cell infusions may enhance intrinsic repair mechanisms in the Organ of Corti which may restore hair cells. This treatment could ultimately lead to hearing improvement. Human umbilical cord blood (hUCB) is an available, autologous, stored progenitor cell population available for potential therapeutic use. The primary objective of this study is to determine the safety of autologous hUCB infusion in children with acquired hearing loss. The secondary objective is to determine if functional, physiologic and anatomic outcomes are improved following hUCB treatment in this patient population.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Biologic; Autologous Cell Injection | Experimental | Biological: Autologous Human Umbilical Cord Blood |
Who may qualify
- Ages
- 6 weeks to 18 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Evidence of a moderate to profound sensorineural hearing loss.
- Normally shaped cochlea, as determined by MRI.
- The loss must be considered acquired, NOT syndromic.
- The patient must be fitted for hearing aids of the detection of the loss.
- Enrollment in a parent/child intervention program.
Key exclusion criteria (22 listed)
- Inability to obtain pertinent medical records.
- Known history or
- hUCB sample contamination.
- Participation in a concurrent intervention study.
- Desire for organ donation in the event of death.
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 Human Umbilical Cord BloodAlso: Autologous Human Umbilical Cord Blood Mononuclear Fraction Cells, Patient's Own Stem Cells6 million cells/kg will be administered intravenously at one treatment time point. | Biological | Biologic; Autologous Cell Injection |
Primary outcome
Physiologic Outcome
Time frame: One year
Age appropriate physiologic outcome measures will be recorded pre-treatment, and one year following hUCB treatment
1 secondary outcome
Functional Outcome
Time frame: one year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2024.
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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 E. Baumgartner, MD principal investigator
MHHS, Houston,TX & FL Hospital for Children, Orlando, FL
Linda S. Baumgartner, CCC-SLP, LSLS CERT.AVT principal investigator
Speech Therapists for Children
Samir Fakhri, MD principal investigator
The University of Texas Health Science Center, Houston
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Human Umbilical Cord Blood in people with Hearing Loss, run by Aryn Knight 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 7 inclusion and 22 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: withdrawn, as of the sponsor's last update on 20 Mar 2024. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Physiologic Outcome (time frame: One year). 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 20 Mar 2024.