Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).

Cerebral Palsy Clinical Trial

— ACT for CP  

Official title:

Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01988584
• Other study ID #: HSC-MS-12-0876
• Status: Completed
• Phase: Phase 2
• Start date: November 2013
• Est. completion date: February 21, 2018

Study information

• Verified date: October 2022
• Source: The University of Texas Health Science Center, Houston
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to compare the safety and effectiveness of two types of stem cells,(either banked cord blood or bone marrow), in children between the ages of 2 to 10 years with CP. 15 children with banked cord blood at CBR and 15 children without banked cord blood will be enrolled into the study. The study involves one baseline/treatment visit and 3 follow-up visits at 6 months, 12 months, and 2 years. Five children in each group will be randomized to a placebo control group at the baseline/treatment visit. Parents will not be told if their child received stem cells or a placebo until the 12 month follow-up visit. At that time parents may elect to have their child receive the stem cell treatment; either bone marrow harvest or umbilical cord blood if banked with CBR. All study visits will be conducted at the UTHealth Medical School and Children's Memorial Hermann Hospital in Houston, Texas. As of 1/21/2014 we have met our enrollment limit for children without banked cord blood undergoing bone marrow harvest for stem cells.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: February 21, 2018
• Est. primary completion date: February 21, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 10 Years

Eligibility

Inclusion Criteria:

1. Children with diagnosis of Cerebral Palsy (spastic CP due to periventricular white matter damage or neonatal brain injury from perinatal stroke or intra-ventricular hemorrhage)

2. Gross Motor Function Classification Score level II-V

3. Ages 24 months to 10 years

4. English speaking, if verbal

5. Ability to travel to Houston for treatment and follow-up -

Exclusion Criteria:

1. Known history of:

• Intractable seizures
• Traumatic brain injury
• Genetic disorder (as demonstrated by newborn screening or genetic diagnostic testing)
• Recently treated or current infection
• Renal insufficiency or altered renal function (as defined by serum creatinine > 1.5 mg/dl at screening)
• Hepatic disease or altered liver function (as defined by SGPT > 150 U/L [non-contusion related], and/or T. Bilirubin >1.3 mg/dL at screening)
• HIV+ (as demonstrated by positive blood test)
• Immunosuppression (as defined by WBC <3,000 cells/ml at screening)
• Infectious related neurological injury
• Sensitivity to Ethylene Oxide (EtO) [found in fumigants and disinfectants]

2. If Athetoid CP diagnosis, other etiologies such as degenerative, mitochondrial, and metabolic disorders must be excluded, and the outcome assessments must be able to be conducted to assess for potential treatment effects

3. Normal brain MRI

4. Evidence of acute illness at the time of infusion, such as, but not limited to, fever (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles

5. Progressing neurological disease (as defined by Batten Disease, Leukodystrophies, Metabolic disorders, Mitochondrial disorders, Neurotransmitter disorders)

6. Microcephaly, macrocephaly, cortical malformations, genetic disorders of dysgenesis brain malformations due to infection or metabolic disorders

7. Pulmonary disease requiring ventilator support

8. If hUCB candidate, banked cord cells totaling <10 million/kg

9. If hUCB candidate, any positive maternal infectious disease test (Hepatitis A, Hepatitis B, HIV 1, HIV 2, HTLV 1, HTLV 2, and Syphilis)

10. If hUCB candidate, cord blood sample contamination

11. Participation in a concurrent intervention study

12. Unwillingness to return for follow-up visits

13. Contraindications to MRI

14. Any patient that the investigators feel in their opinion the study intervention is unlikely to benefit the patient will be a screen failure.

15. Any patients who are currently or has previously been enrolled in a clinical stem cell study.

Related Conditions & MeSH terms

• Cerebral Palsy
• Paralysis

Intervention

Biological:
• umbilical cord blood (hUCB) cells
Autologous umbilical cord blood banked with the Cord Blood Registry.

Drug:
• Saline Infusion (Placebo)
A total of 10 children (5 from each cohort) will be randomized to a placebo infusion at the baseline visit and then have the opportunity to cross-over to stem cell treatment at the 1yr. visit.

Biological:
• bone marrow derived mononuclear cells (BMMNCs)
Autologous stem cells from bone marrow harvest.

Locations

Country	Name	City	State
United States	UTHealth, Medical School, Dept. of Pediatric Surgery	Houston	Texas

Sponsors (4)

Lead Sponsor	Collaborator
The University of Texas Health Science Center, Houston	Cord Blood Registry (CBR), Let's Cure CP Foundation, Mission Connect, a program of TIRR Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity	In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.	24 hours after infusion
Primary	Long-term Safety	Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status	from the time of infusion to 1 year after infusion
Secondary	Number of Participants With an Improvement in White Matter Integrity.	Number of participants with an improvement in white matter integrity as measured by diffusion tensor imaging (DTI) magnetic resonance imaging (MRI) reconstruction of corticospinal tract (CST) radial diffusivity (RD).; Improvement is defined as radial diffusivity (RD) decreased in at least one corticospinal tract (CST) reconstruction.	from baseline to 1 year after infusion
Secondary	Gross Motor Function Classification Score (GMFM-66)	Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.	baseline before infusion
Secondary	Gross Motor Function Classification Score (GMFM-66)	Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.	1 year after infusion
Secondary	Gross Motor Function Classification Score (GMFM-88)	Ordinal age adjusted scaled raw and percent scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.	baseline before infusion
Secondary	Gross Motor Function Classification Score (GMFM-88)	Ordinal age adjusted scaled raw and percent scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.	1 year after infusion
Secondary	Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication	Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication	Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living	Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living	Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Vineland Adaptive Behavior Scales (VABS-2) - Social	Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Vineland Adaptive Behavior Scales (VABS-2) - Social	Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor	Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor	Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Pediatric Evaluation of Disability Inventory - Self-Care	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Pediatric Evaluation of Disability Inventory - Self-Care	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Pediatric Evaluation of Disability Inventory - Mobility	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Pediatric Evaluation of Disability Inventory - Mobility	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Pediatric Evaluation of Disability Inventory - Social	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Pediatric Evaluation of Disability Inventory - Social	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall	Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.	baseline before infusion
Secondary	Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall	Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.	1 year after infusion
Secondary	Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued	Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.	baseline before infusion
Secondary	Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued	Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.	1 year after infusion
Secondary	Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)	Standard score ranging from 20 to 160. Higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)	Standard score ranging from 20 to 160. Higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	baseline before infusion
Secondary	Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain	Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.	1 year after infusion
Secondary	Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)	Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.	baseline before infusion
Secondary	Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)	Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.	1 year after infusion