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

• ClinicalTrials.gov Identifier: NCT01700166
• Recruitment Status: Withdrawn
• First Posted: October 4, 2012
• Last Update Posted: January 23, 2014
• Sponsor: James E.Baumgartner, MD
• 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
• Information provided by (Responsible Party): James E.Baumgartner, MD, Memorial Hermann Health System

Tracking Information

• First Submitted Date: October 2, 2012
• First Posted Date: October 4, 2012
• Last Update Posted Date: January 23, 2014
• Study Start Date: September 2012
• Actual Primary Completion Date: August 2013 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 2, 2012)

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.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: October 2, 2012)

Speech Therapy Specific Neuropsychological outcome measures [ Time Frame: Two years ]

Age appropriate Neurologic examinations and speech therapy specific standardized testing will be performed pre-treatment and at all follow-up visits.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Umbilical Cord Blood in the Treatment of Stroke in Children.
• Official Title: Safety of Autologous Human Umbilical Cord Blood in the Treatment of Stroke in Children.

Brief 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 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 Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Arterial Ischemic Stroke (AIS) in Children

Intervention

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

Study Arms

Experimental: Biologic; Cord Blood Stem Cells; Intravenous injection

Autologous Human Umbilical Cord Blood derived Stem Cell injection

Intervention: Biological: Autologous Human Cord Blood derived Stem Cell injection

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Withdrawn
• Actual Enrollment (submitted: January 21, 2014): 0
• Original Estimated Enrollment (submitted: October 2, 2012): 10
• Estimated Study Completion Date: December 2016
• Actual Primary Completion Date: August 2013 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Weeks to 6 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01700166
• Other Study ID Numbers: JB IND #14576
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: James E.Baumgartner, MD, Memorial Hermann Health System
• Study Sponsor: James E.Baumgartner, MD

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

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

• PRS Account: Memorial Hermann Health System
• Verification Date: January 2014