Arterial Ischemic Stroke (AIS) in ChildrenPhase 1Pedi StrokeNCT01700166
Umbilical Cord Blood in the Treatment of Stroke in Children.
A Phase 1 study testing Autologous Human Cord Blood derived Stem Cell injection in people with Arterial Ischemic Stroke (AIS) in Children, run by Aryn Knight at 1 site in United States.
Trial summary
The specific aims of this study are: 1. To determine if Human Umbilical Cord Blood (hUCB) infusion is safe in children with perinatal arterial ischemic stroke (AIS). 2. To determine if late functional outcome, physiologic response, and anatomic findings are changed following hUCB infusion in children with perinatal… Read more about this study
- Who is it for?
- Ages 6 weeks to 6 years
- Conditions the study is for
- Arterial Ischemic Stroke (AIS) in Children
- What is being tested?
- Autologous Human Cord Blood derived Stem Cell injection
- 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
- Sep 2012to Dec 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 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Between 6 weeks and 6 years of age on the day of study cord blood infusion.
- MRI documented single arterial distribution infarction.
- Initial injury occurring in the pre-natal or perinatal period.
- Ability of caregivers to understand and speak English
- Ability of child and caregiver to travel to Houston, and stay for at least 4 days, and to return for all Follow-up visits (patient is responsible for cost of travel and lodging while in Houston)
Who cannot join
- Inability to obtain all pertinent medical records, including pertinent physician notes, laboratory findings, and radiographic images, related to the original injury, hospitalization and rehabilitation - must be sent to research team at least 14 days prior to scheduled study cord blood treatment.
- Recent radiographic evidence (imaging performed within past 2 weeks) of extensive stroke as evidenced by >100ml lesion.
- Multifocal infarctions on screening MRI.
- Evidence of hypoxic-ischemic encephalopathy on screening MRI.
- Uncorrected coagulopathy during the baseline period defined as INR > 1.4; PTT> 35 sec; PLT < 100,000.
- Known history of:
Show all 17Show fewer
- Recently diagnosed infection (within past 2 weeks) requiring treatment and/or medical intervention.
- Renal disease or altered renal function as defined by serum creatinine > 1.5 mg/dL at admission.
- Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and/or T. Bilirubin >1.3 mg/dL at enrollment.
- Malignancy.
- Immunosuppression as defined by WBC < 3 (10x3) at admission.
- HIV, Hepatitis B, Hepatitis C.
- Pneumonia, or chronic lung disease requiring oxygen.
- Cord blood sample contamination.
- Participation in a concurrent intervention study.
- Desire for organ-donation in the event of death.
- Unwillingness or inability to stay for at least four days following cord blood infusion (should any problems arise following the infusion) and to return for 6 month, 1 year, and 2 year follow-up visits.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Memorial Hermann Hospital; University of Texas Health Science Center - Houston
Houston, Texas, 77030
About this study
Official title: Safety of Autologous Human Umbilical Cord Blood in the Treatment of Stroke in Children.
The specific aims of this study are:
- To determine if Human Umbilical Cord Blood (hUCB) infusion is safe in children with perinatal arterial ischemic stroke (AIS).
- To determine if late functional outcome, physiologic response, and anatomic findings are changed following hUCB infusion in children with perinatal AIS.
Read the detailed description
This study will enroll ten pediatric patients who have their umbilical cord blood banked with Cord Blood Registry, Inc. (CBR), who have been diagnosed with an arterial ischemic stroke (AIS), and whose caregivers contacted CBR, Inc. to notify them that the child has experienced an AIS around the time of birth.
After receiving permission to release the patient's contact information, the CBR team will then relay the contact information to the Stem Cell Study Group at the University of Texas - Houston (UT) / Children's Memorial Hermann Hospital (CMHH) who will contact the family, explain the study, and send a consent form for their review.
Several weeks prior to the scheduled procedure date, the patient will go to their family doctor for a physical evaluation and have blood drawn to rule out problems with the child's health.
After successfully meeting initial pre-screening criteria, the patient's family will make their own arrangements to travel to Houston for the study procedures.
Once the patient is in Houston, he/she will undergo baseline assessments, including physical, neurological exams and tests, speech therapy testing, laboratory tests, and imaging (MRI) of the brain. If any of the blood tests are abnormal, or the patient has an active infection, the procedure may be postponed or the patient may possibly be excluded from the study.
If the patient continues to meet all criteria, the frozen cord blood will be shipped overnight to a special lab in Houston, TX for processing. Once release criteria have been met, the cells will be brought to the CMHH for infusion into the patient's vein. The patient will remain at CHMM for 24 hours of monitoring, after which they will be allowed to go home. Patients will be called daily by the research staff through Day 14, and once a month after that.
Patients will return to Houston, Texas for 6 month, 1 year and 2 year follow-up visits for repeat assessments, tests, and exams just like at the baseline visit. The MRI of the brain will only be repeated at the 6 month visit.
- Being tested
- Autologous Human Cord Blood derived Stem Cell injection
- Keywords
- Autologous, Cord Blood, Stem Cells, Pediatric stroke, Children with ischemic stroke, Cerebral Palsy, Perinatal AIS
- Medical subject headings
- Ischemic Stroke, Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Human Cord Blood derived Stem Cell injectionBiological
Also called Patient's own cord blood, Patient's own stem cells
One time intravenous (in the vein) injection with two year follow-up
| Study group | Role | What they receive |
|---|---|---|
| Biologic; Cord Blood Stem Cells; Intravenous injection | Experimental | Autologous Human Umbilical Cord Blood derived Stem Cell injection Receives: Biological: Autologous Human Cord Blood derived Stem Cell injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional outcome measure
Physiological (e.g., SSEP, EEG) and anatomic outcome (MRI) changes will be evaluated by repeating assessments, clinical tests and imaging exams at the pre-treatment and follow-up visits.
Measured: Two years
Secondary outcomes (1)
Speech Therapy Specific Neuropsychological outcome measures
Age appropriate Neurologic examinations and speech therapy specific standardized testing will be performed pre-treatment and at all follow-up visits.
Measured: Two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2012 (estimated)
- Study startSep 2012
- Primary completionAug 2013
- Study completionDec 2016 (estimated)
Why it stopped: Principal Investigator relocated; Study may resume in Fl Hosp. for Children, Orlando FL
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Arterial Ischemic Stroke (AIS) in ChildrenStudies 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
- The Institute for Rehabilitaion and Research Foundation, Cord Blood Registry, Inc., The University of Texas Health Science Center, Houston, M.D. Anderson Cancer Center - Houston, Baylor College of Medicine
- Responsible party
- Sponsor-investigator, Aryn Knight, Memorial Hermann Health System
- Investigators
- Timothy C. Foster, MD, Principal investigator, The University of Texas Health Science Center, HoustonLinda S. Baumgartner, MS, CCC-SLP, LSLS, Cert.AVT, Principal investigator, Florida Hospital for Children - OrlandoJames E. Baumgartner, MD, Study chair, Florida Hospital for Children - Orlando
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01700166
- Sponsor's protocol ID
- JB IND #14576
- Acronym
- Pedi Stroke
- Status last verified
- Mar 2024
- First submitted
- 2 Oct 2012
- 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 in the Treatment of Stroke in Children.
- ClinicalTrials.gov ID
- NCT01700166
- Acronym / sponsor ID
- Pedi Stroke / JB IND #14576
- Keywords
- Autologous, Cord Blood, Stem Cells, Pediatric stroke, Children with ischemic stroke, Cerebral Palsy, Perinatal AIS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Principal Investigator relocated; Study may resume in Fl Hosp. for Children, Orlando FL
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Aryn KnightUniversity, hospital or other
- Collaborators
- The Institute for Rehabilitaion and Research Foundation, Cord Blood Registry, Inc., The University of Texas Health Science Center, Houston, M.D. Anderson Cancer Center - Houston, Baylor College of Medicine
- Study start
- Sep 2012
- Primary completion
- Aug 2013 (actual)
- Study completion
- Dec 2016 (estimated)
- First posted
- 4 Oct 2012 (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 specific aims of this study are:
- To determine if Human Umbilical Cord Blood (hUCB) infusion is safe in children with perinatal arterial ischemic stroke (AIS).
- To determine if late functional outcome, physiologic response, and anatomic findings are changed following hUCB infusion in children with perinatal AIS.
Detailed description
This study will enroll ten pediatric patients who have their umbilical cord blood banked with Cord Blood Registry, Inc. (CBR), who have been diagnosed with an arterial ischemic stroke (AIS), and whose caregivers contacted CBR, Inc. to notify them that the child has experienced an AIS around the time of birth.
After receiving permission to release the patient's contact information, the CBR team will then relay the contact information to the Stem Cell Study Group at the University of Texas - Houston (UT) / Children's Memorial Hermann Hospital (CMHH) who will contact the family, explain the study, and send a consent form for their review.
Several weeks prior to the scheduled procedure date, the patient will go to their family doctor for a physical evaluation and have blood drawn to rule out problems with the child's health.
After successfully meeting initial pre-screening criteria, the patient's family will make their own arrangements to travel to Houston for the study procedures.
Once the patient is in Houston, he/she will undergo baseline assessments, including physical, neurological exams and tests, speech therapy testing, laboratory tests, and imaging (MRI) of the brain. If any of the blood tests are abnormal, or the patient has an active infection, the procedure may be postponed or the patient may possibly be excluded from the study.
If the patient continues to meet all criteria, the frozen cord blood will be shipped overnight to a special lab in Houston, TX for processing. Once release criteria have been met, the cells will be brought to the CMHH for infusion into the patient's vein. The patient will remain at CHMM for 24 hours of monitoring, after which they will be allowed to go home. Patients will be called daily by the research staff through Day 14, and once a month after that.
Patients will return to Houston, Texas for 6 month, 1 year and 2 year follow-up visits for repeat assessments, tests, and exams just like at the baseline visit. The MRI of the brain will only be repeated at the 6 month visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Biologic; Cord Blood Stem Cells; Intravenous injection | Experimental | Biological: Autologous Human Cord Blood derived Stem Cell injectionAutologous Human Umbilical Cord Blood derived Stem Cell injection |
Who may qualify
- Ages
- 6 weeks to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Between 6 weeks and 6 years of age on the day of study cord blood infusion.
- MRI documented single arterial distribution infarction.
- Initial injury occurring in the pre-natal or perinatal period.
- Ability of caregivers to understand and speak English
- Ability of child and caregiver to travel to Houston, and stay for at least 4 days, and to return for all Follow-up visits (patient is responsible for cost of travel and lodging while in Houston)
Key exclusion criteria (17 listed)
- Inability to obtain all pertinent medical records, including pertinent physician notes, laboratory findings, and radiographic images, related to the original injury, hospitalization and rehabilitation - must be sent to research team at least 14 days prior to scheduled study cord blood treatment.
- Recent radiographic evidence (imaging performed within past 2 weeks) of extensive stroke as evidenced by >100ml lesion.
- Multifocal infarctions on screening MRI.
- Evidence of hypoxic-ischemic encephalopathy on screening MRI.
- Uncorrected coagulopathy during the baseline period defined as INR > 1.4; PTT> 35 sec; PLT < 100,000.
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 Cord Blood derived Stem Cell injectionAlso: Patient's own cord blood, Patient's own stem cellsOne time intravenous (in the vein) injection with two year follow-up | Biological | Biologic; Cord Blood Stem Cells; Intravenous injection |
Primary outcome
Functional outcome measure
Time frame: Two years
Physiological (e.g., SSEP, EEG) and anatomic outcome (MRI) changes will be evaluated by repeating assessments, clinical tests and imaging exams at the pre-treatment and follow-up visits.
1 secondary outcome
Speech Therapy Specific Neuropsychological outcome measures
Time frame: Two years
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.
Related trials for Arterial Ischemic Stroke (AIS) in Children
Not yet recruitingObservational study
Prospective Registry of Acute Ischemic Stroke in Children Under 18 Years Old in China
Acute Ischemic Stroke (AIS), Pediatric Stroke
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.
Timothy C. Foster, MD principal investigator
The University of Texas Health Science Center, Houston
Linda S. Baumgartner, MS, CCC-SLP, LSLS, Cert.AVT principal investigator
Florida Hospital for Children - Orlando
James E. Baumgartner, MD study chair
Florida Hospital for Children - Orlando
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 Cord Blood derived Stem Cell injection in people with Arterial Ischemic Stroke (AIS) in Children, 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 5 inclusion and 17 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?
Functional outcome measure (time frame: Two years). 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.