Umbilical Cord Blood in the Treatment of Stroke in Children. (Pedi Stroke)

Safety of Autologous Human Umbilical Cord Blood in the Treatment of Stroke in Children.

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 AIS.

Study Overview

• Status: Withdrawn
• Conditions: Arterial Ischemic Stroke (AIS) in Children
• Intervention / Treatment: Biological: Autologous Human Cord Blood derived Stem Cell injection

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 Type: Interventional
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • Houston, Texas, United States, 77030
      • Children's Memorial Hermann Hospital; University of Texas Health Science Center - Houston

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 6 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Between 6 weeks and 6 years of age on the day of study cord blood infusion.
2. MRI documented single arterial distribution infarction.
3. Initial injury occurring in the pre-natal or perinatal period.
4. Ability of caregivers to understand and speak English
5. 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)

Exclusion Criteria:

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.

1. Recent radiographic evidence (imaging performed within past 2 weeks) of extensive stroke as evidenced by >100ml lesion.
2. Multifocal infarctions on screening MRI.
3. Evidence of hypoxic-ischemic encephalopathy on screening MRI.
4. Uncorrected coagulopathy during the baseline period defined as INR > 1.4; PTT> 35 sec; PLT < 100,000.

5. Known history of:

   1. Recently diagnosed infection (within past 2 weeks) requiring treatment and/or medical intervention.
   2. Renal disease or altered renal function as defined by serum creatinine > 1.5 mg/dL at admission.
   3. Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and/or T. Bilirubin >1.3 mg/dL at enrollment.
   4. Malignancy.
   5. Immunosuppression as defined by WBC < 3 (10x3) at admission.
   6. HIV, Hepatitis B, Hepatitis C.
6. Pneumonia, or chronic lung disease requiring oxygen.
7. Cord blood sample contamination.
8. Participation in a concurrent intervention study.
9. Desire for organ-donation in the event of death.
10. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Biologic; Cord Blood Stem Cells; Intravenous injection; Autologous Human Umbilical Cord Blood derived Stem Cell injection	Biological: Autologous Human Cord Blood derived Stem Cell injection; One time intravenous (in the vein) injection with two year follow-up; Other Names: Patient's own cord blood; Patient's own stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Two years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Two years

Collaborators and Investigators

• Sponsor: Aryn Knight
• Collaborators: The University of Texas Health Science Center, Houston; Baylor College of Medicine; The Institute for Rehabilitaion and Research Foundation; Cord Blood Registry, Inc.; M.D. Anderson Cancer Center - Houston
• Investigators: Principal Investigator: Timothy C. Foster, MD, The University of Texas Health Science Center, Houston; Principal Investigator: Linda S. Baumgartner, MS, CCC-SLP, LSLS, Cert.AVT, Florida Hospital for Children - Orlando; Study Chair: James E. Baumgartner, MD, Florida Hospital for Children - Orlando

Study record dates

Study Major Dates

• Study Start: September 1, 2012
• Primary Completion (Actual): August 1, 2013
• Study Completion (Estimated): December 1, 2016

Study Registration Dates

• First Submitted: October 2, 2012
• First Submitted That Met QC Criteria: October 2, 2012
• First Posted (Estimated): October 4, 2012

Study Record Updates

• Last Update Posted (Actual): March 20, 2024
• Last Update Submitted That Met QC Criteria: March 18, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Cerebral Palsy; Stem Cells; Autologous; Cord Blood; Pediatric stroke; Children with ischemic stroke; Perinatal AIS
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Stroke; Ischemic Stroke
• Other Study ID Numbers: JB IND #14576