Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP). (ACT for CP)
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|ClinicalTrials.gov Identifier: NCT01988584|
Recruitment Status : Completed
First Posted : November 20, 2013
Last Update Posted : November 9, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Cerebral Palsy||Biological: Umbilical Cord Blood Stem Cells Other: Saline Infusion (Placebo) Biological: Bone Marrow Stem Cells||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Triple (Participant, Care Provider, Outcomes Assessor)|
|Official Title:||Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)|
|Actual Study Start Date :||November 2013|
|Actual Primary Completion Date :||February 21, 2018|
|Actual Study Completion Date :||February 21, 2018|
Active Comparator: Umbilical Cord Blood (UCB) Arm
Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.
Biological: Umbilical Cord Blood Stem Cells
Autologous umbilical cord blood banked with the Cord Blood Registry.
Other Name: Autolgous Stem Cells
Active Comparator: Bone Marrow Stem Cells (BMMNC's)
Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.
Biological: Bone Marrow Stem Cells
Autologous stem cells from bone marrow harvest.
Other Name: Autologous Stem Cells
Placebo Comparator: Placebo (inactive substance) Group
Five children in each group will be randomly assigned to receive an inactive substance (placebo) at the baseline/treatment visit. Parents will be given the opportunity to cross-over to either the umbilical cord blood or bone marrow harvest group at the one year visit.
Other: 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.
- To determine if autologous cells using either BMMNCs or hUCBs are safe to administer in children with CP by assessing change at multiple time points. [ Time Frame: All study visits from baseline to the end of study visit at year 2. ]
- In-hospital infusion toxicity: pulmonary and hepatic function; new seizures, hemorrhagic lesions or ischemic lesions on imaging. (Composite Outcome Measure)
- Long-term safety: development of new mass lesions or other pathological structural changes; worsening neurological status. (Composite Outcome Measure)
- To determine if late functional outcomes are improved following the administration of autologous cells compared with patients in the control group. [ Time Frame: Follow-up visits at 6 and 12 months, and the end of study year 2 visit. ]
- Detailed analysis of MRI's done at baseline and follow-up visits. Specific white matter tract analysis will be identified at baseline MRI and correlated with motor function studies as the primary lesion of interest. Total volumes and specific tract lesions will be analyzed and correlated with functional outcomes.
The following functional outcomes studies will be performed at baseline, 6 months, 1 year after infusion, and 2 year after infusion.
- Gross Motor Function Measures
- Psychological Assessment Tests
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01988584
|United States, Texas|
|UTHealth, Medical School, Dept. of Pediatric Surgery|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Charles S Cox, MD||UTHealth, Medical School, Dept. of Pediatric Surgery|