Clinical trial NCT02460484: Safety of Autologous Human Umbilical Cord Blood Treatment for Perinatal Arterial Ischemic Stroke
Official title: Safety of Autologous Human Umbilical Cord Blood Treatment for Perinatal
Study type: INTERVENTIONAL
Overall status: SUSPENDED
Phase: PHASE1
PHASE2
Conditions: Perinatal Arterial Ischemic Stroke
Lead sponsor: AdventHealth
Sponsor class: OTHER
Collaborators: name: Cord Blood Registry, Inc.
First posted: 2015-06-02
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Autologous human umbilical cord blood (hUCB) stored at Cord Blood Registry will be given to children who have suffered from a Perinatal Arterial Ischemic Stroke. The aim is to determine if hUCB infusion is safe, if late functional outcome is improved, if hUCB treatment improves physiologic response in the child's SSEP \& EEG, and the effect of hUCB infusion in altering anatomic findings on MRI.

Detailed description: Autologous human umbilical cord blood (hUCB) stored at Cord Blood Registry will be given to children who have suffered from a Perinatal Arterial Ischemic Stroke.

Subjects will come to Orlando for pretesting to include an MRI, SSEP, Urodynamics, blood work: CBC, CMP, Hepatic Function Panel, PT/PTT/INR, Chest Xray, EEG, Gross Motor Function Classification, Manual Ability Classification System, and a Speech and Language Evaluation.

After pretesting, the subjects will receive their autologous cord blood infusion intravenously. The subjects will then be monitored for 24 hours post infusion. After 24 hours, the subject will undergo repeat blood work and a chest x ray. Subjects will then be discharged home.

Subjects will follow up in Orlando at 6 months and 1 year post infusion. Follow up testing will repeat the exams performed at pretesting.

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 prenatal or perinatal period
4. Ability of child and caregiver to travel to Orlando, 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 Orlando)

Exclusion Criteria:

1. 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 FHFC research team 14 days prior to scheduled study cord blood treatment. Need brain MRI with flair sequence \<2 weeks old.
2. Recent radiographic evidence (imaging performed within past 2 weeks) of extensive stroke as evidenced by \>100ml lesion
3. Multifocal infarctions on screening MRI.
4. Evidence of hypoxic-ischemic encephalopathy on screening MRI.
5. Uncorrected coagulopathy during the baseline period defined as INR \> 1.4; PTT\> 35 sec; PLT \< 100,000
6. 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
7. Any evidence of active maternal infection during the pregnancy (Hepatitis A, Hepatitis B, Hepatitis C, HIV 1, HIV 2, Human T-lymphotropic Virus (HTLV) 1, HTLV 2
8. Pneumonia, or chronic lung disease requiring oxygen
9. Cord blood sample contamination
10. Participation in a concurrent intervention study
11. Desire for organ-donation in the event of death
12. 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, and 1 year follow-up visits

Primary outcomes:
measure: Composite Outcome: Hemodynamic Safety
timeFrame: 1 year
measure: Composite Outcome: Pulmonary Safety
timeFrame: 1 year
measure: Composite Outcome: Renal Safety
timeFrame: 1 year
measure: Composite Outcome: Neurological Safety
timeFrame: 1 year
Secondary outcomes:
measure: EEG
timeFrame: 1 year
measure: Fine and Gross Motor
timeFrame: 1 year
measure: Language
timeFrame: 1 year
measure: Speech
timeFrame: 1 year
measure: Bladder: Urodynamics
timeFrame: 1 year
Other outcomes:
Locations:
city: Orlando
country: United States
facility: Florida Hospital for Children
state: Florida
Study officials:
affiliation: AdventHealth
name: James Baumgartner, MD
role: PRINCIPAL_INVESTIGATOR
References: