Traumatic Brain InjuryPhase 1NCT00254722
Safety of Autologous Stem Cell Treatment for Traumatic Brain Injury in Children
A Phase 1 study testing Autologous bone marrow precursor cell harvest and transplant in people with Traumatic Brain Injury, run by The University of Texas Health Science Center, Houston.
Trial summary
The purpose of this study is to determine if bone marrow progenitor cell (BMPC) autologous transplantation in children after isolated traumatic brain injury is safe and will improve functional outcome. Read more about this study
- Who is it for?
- Ages 5 to 14 years
- Conditions the study is for
- Traumatic Brain Injury
- What is being tested?
- Autologous bone marrow precursor cell harvest and transplant
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- No sites listed yet
- Study dates
- Apr 2006to Oct 2009
- 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 5 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Between 5 and 14 years of age on the day of injury
- Hospital admission Glasgow coma score between 5 and 8
- Initial injury occurring less than 24 hours prior to consent
Who cannot join
- Known history of:
- Previous brain injury
- Developmental delay
- Neurologic impairment and/or deficit
- Seizure disorder requiring anti-convulsant therapy
- Renal disease or altered renal function as defined by serum creatinine > 1.5 mg/dL at admission
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- Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and/or T. bilirubin > 1.3 mg/dL at admission
- Cancer
- Immunosuppression as defined by WBC < 3 (10x3) at admission
- HIV
- Obliteration of perimesencephalic cistern on initial head CT/MRI suggesting prolonged hypoxic ischemic insult
- Initial hospital ICP > 40
- Hemodynamic instability at the time of consent defined as ongoing fluid resuscitation and/or requirement for inotropic support to maintain MAP at or above normals for age - does not include CPP based inotropic support
- Uncorrected coagulopathy at the time of consent defined as INR > 1.4; PTT > 35 sec; PLT < 100,000; fibrinogen < 100 g/dL
- Unstable pelvic fractures defined as requiring operative fixation to manage
- Pulmonary contusions defined as a chest x-ray with non-anatomic opacification and/or PaO2:FIO2 ratio < 250 associated with the mechanism or injury
- Solid or hollow visceral injury of the abdomen and/or pelvis as diagnosed by CT or other imaging
- Spinal cord injury as diagnosed by CT or MR imaging or by clinical findings.
- Persistent hypoxia defined as SaO2 < 94% for > 30 minutes occurring at any time from hospital admission to time of consent
- Positive urine pregnancy test
- Participation in an intervention study
- Unwillingness to return for follow-up visits
About this study
The purpose of this study is to determine if bone marrow progenitor cell (BMPC) autologous transplantation in children after isolated traumatic brain injury is safe and will improve functional outcome.
Read the detailed description
Traumatic brain injury (TBI) contributes to 50% of all trauma deaths. The mortality rate for children following severe TBI (Glasgow Coma Scale < 9) ranges from 14-24%. There is currently no therapy to reverse the primary injury associated with TBI. Bone marrow precursor cells (BMPC) or bone marrow mononuclear cellular fractions of bone marrow contain mesenchymal stem cells (MSC) and hematopoetic stem cells (HSC). These cells are a component of bone marrow that preferentially migrate to the site of brain injury and differentiate into neurons and cell supporting elements, improving functional outcome in animals. The primary objective of this study is to determine if BMPC harvest and autologous transplantation is safe in children after TBI. The secondary objective is to determine if late functional outcome is improved with BMPC autologous transplantation compared to age and severity matched concomitant controls. Safety will be determined by monitoring cerebral and systemic hemodynamics during harvest and transplantation, neurologic events (seizure, change in GCS, stroke), local site inflammation/injury, and secondary organ injury. Late outcomes will be determined using age-corrected Glasgow Outcome Scores, and a battery of functional outcome measures. In vitro, an aliquot of cells harvested from patients will be studied for labeling with magnetodendrimers as a feasibility study, and these cells will not be reinfused into the patients. The primary endpoint is to assess the safety of autologous BMPC harvest/transplantation in the acute injury phase (hospital stay) and the secondary endpoint is to assess efficacy through 1 and 6 month post-injury follow-up. The rationale for the use of autologous BMPC transplantation is based on a large volume of in vitro and in vivo animal data (see background and significance section). The rationale for using children as the primary population is that children have a greater neurologic plasticity with a unique injury pattern when compared to adults. Children are more likely to have isolated TBI that is more diffuse and less likely to be secondary to extra-axial fluid collections. Patients aged 5-14 years old with GCS of 5-8 will be considered for enrollment into the study. Within 24-36 hours of injury, enrolled patients will undergo bone marrow harvest/BMPC separation and re-infusion. Daily monitoring of the safety outcomes measures and long term neurologic outcomes will be performed. This study should determine if bone marrow harvest, BMPC separation, and reinfusion is safe in children after severe TBI.
- Conditions
- Traumatic Brain Injury
- Being tested
- Autologous bone marrow precursor cell harvest and transplant
- Keywords
- Traumatic Brain Injury, Stem Cell
- Medical subject headings
- Brain Injuries, Traumatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous bone marrow precursor cell harvest and transplantDrug
Bone marrow harvest (3 ml/kg of body weight) performed between 12 and 30 hours post injury, followed by single intravenous infusion of bone marrow progenitor cells - target dose is 6x10^6 mononuclear cells/kg body weight, administered within 36 hours of injury
| Study group | Role | What they receive |
|---|---|---|
| I | Experimental | single arm study Receives: Drug: Autologous bone marrow precursor cell harvest and transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
neurologic events [seizures, change in Glasgow coma scale (GCS), cerebral vascular accident (CVA)]
Measured: 12 hours post cellular product infusion, up to 21 days post infusion
infectious morbidity
Measured: up to 21 days post cellular product infusion
secondary organ injury
Measured: up to 21 days post cellular product infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2005 (estimated)
- Study startApr 2006
- Primary completionNov 2008
- Study completionOct 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Traumatic Brain InjuryStudies of the same condition, open and under way.
- More trials run by The University of Texas Health Science Center, HoustonThe sponsor's other registered studies.
- 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
- 10 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Charles Cox, The University of Texas Health Science Center, Houston
- Investigators
- Charles S. Cox, Jr., M.D., Principal investigator, The University of Texas Health Science Center, Houston
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00254722
- Sponsor's protocol ID
- HSC-MS-05-0004
- Other IDs
- 1R21HD042659-01A1
- Status last verified
- May 2020
- First submitted
- 14 Nov 2005
- Last updated
- 13 May 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety of Autologous Stem Cell Treatment for Traumatic Brain Injury in Children
- ClinicalTrials.gov ID
- NCT00254722
- Acronym / sponsor ID
- HSC-MS-05-0004
- Conditions
- Traumatic Brain Injury
- Keywords
- Traumatic Brain Injury, Stem Cell
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- The University of Texas Health Science Center, HoustonUniversity, hospital or other
- Study start
- Apr 2006
- Primary completion
- Nov 2008 (actual)
- Study completion
- Oct 2009 (actual)
- First posted
- 16 Nov 2005 (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 purpose of this study is to determine if bone marrow progenitor cell (BMPC) autologous transplantation in children after isolated traumatic brain injury is safe and will improve functional outcome.
Detailed description
Traumatic brain injury (TBI) contributes to 50% of all trauma deaths. The mortality rate for children following severe TBI (Glasgow Coma Scale < 9) ranges from 14-24%. There is currently no therapy to reverse the primary injury associated with TBI. Bone marrow precursor cells (BMPC) or bone marrow mononuclear cellular fractions of bone marrow contain mesenchymal stem cells (MSC) and hematopoetic stem cells (HSC). These cells are a component of bone marrow that preferentially migrate to the site of brain injury and differentiate into neurons and cell supporting elements, improving functional outcome in animals. The primary objective of this study is to determine if BMPC harvest and autologous transplantation is safe in children after TBI. The secondary objective is to determine if late functional outcome is improved with BMPC autologous transplantation compared to age and severity matched concomitant controls. Safety will be determined by monitoring cerebral and systemic hemodynamics during harvest and transplantation, neurologic events (seizure, change in GCS, stroke), local site inflammation/injury, and secondary organ injury. Late outcomes will be determined using age-corrected Glasgow Outcome Scores, and a battery of functional outcome measures. In vitro, an aliquot of cells harvested from patients will be studied for labeling with magnetodendrimers as a feasibility study, and these cells will not be reinfused into the patients. The primary endpoint is to assess the safety of autologous BMPC harvest/transplantation in the acute injury phase (hospital stay) and the secondary endpoint is to assess efficacy through 1 and 6 month post-injury follow-up. The rationale for the use of autologous BMPC transplantation is based on a large volume of in vitro and in vivo animal data (see background and significance section). The rationale for using children as the primary population is that children have a greater neurologic plasticity with a unique injury pattern when compared to adults. Children are more likely to have isolated TBI that is more diffuse and less likely to be secondary to extra-axial fluid collections. Patients aged 5-14 years old with GCS of 5-8 will be considered for enrollment into the study. Within 24-36 hours of injury, enrolled patients will undergo bone marrow harvest/BMPC separation and re-infusion. Daily monitoring of the safety outcomes measures and long term neurologic outcomes will be performed. This study should determine if bone marrow harvest, BMPC separation, and reinfusion is safe in children after severe TBI.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| I | Experimental | Drug: Autologous bone marrow precursor cell harvest and transplantsingle arm study |
Who may qualify
- Ages
- 5 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Between 5 and 14 years of age on the day of injury
- Hospital admission Glasgow coma score between 5 and 8
- Initial injury occurring less than 24 hours prior to consent
Key exclusion criteria (22 listed)
- Known history of:
- Obliteration of perimesencephalic cistern on initial head CT/MRI suggesting prolonged hypoxic ischemic insult
- Initial hospital ICP > 40
- Hemodynamic instability at the time of consent defined as ongoing fluid resuscitation and/or requirement for inotropic support to maintain MAP at or above normals for age - does not include CPP based inotropic support
- Uncorrected coagulopathy at the time of consent defined as INR > 1.4; PTT > 35 sec; PLT < 100,000; fibrinogen < 100 g/dL
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 precursor cell harvest and transplantBone marrow harvest (3 ml/kg of body weight) performed between 12 and 30 hours post injury, followed by single intravenous infusion of bone marrow progenitor cells - target dose is 6x10^6 mononuclear cells/kg body weight, administered within 36 hours of injury | Drug | I |
Primary outcome
neurologic events [seizures, change in Glasgow coma scale (GCS), cerebral vascular accident (CVA)]
Time frame: 12 hours post cellular product infusion, up to 21 days post infusion
infectious morbidity
Time frame: up to 21 days post cellular product infusion
secondary organ injury
Time frame: up to 21 days post cellular product infusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2020.
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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.
Charles S. Cox, Jr., M.D. principal investigator
The University of Texas Health Science Center, Houston
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous bone marrow precursor cell harvest and transplant in people with Traumatic Brain Injury, run by The University of Texas Health Science Center, Houston.
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 3 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: finished, as of the sponsor's last update on 13 May 2020. Status can change between updates, so confirm with the study contact before making plans.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
neurologic events [seizures, change in Glasgow coma scale (GCS), cerebral vascular accident (CVA)] (time frame: 12 hours post cellular product infusion, up to 21 days post infusion). The study lists 3 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 13 May 2020.