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

Tracking Information
First Submitted Date ICMJE	November 4, 2013
First Posted Date ICMJE	November 20, 2013
Last Update Posted Date	February 22, 2018
Actual Study Start Date ICMJE	November 2013
Actual Primary Completion Date	February 21, 2018 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: November 19, 2013)	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)
Original Primary Outcome Measures ICMJE	Same as current
Change History	Complete list of historical versions of study NCT01988584 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: November 19, 2013)	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
Original Secondary Outcome Measures ICMJE	Same as current
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Safety and Effectiveness of Banked Cord Blood or Bone Morrow Stem Cells in Children With Cerebral Palsy (CP).
Official Title ICMJE	Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)
Brief 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 morrow 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.
Detailed Description	Not Provided
Study Type ICMJE	Interventional
Study Phase	Phase 2
Study Design ICMJE	Allocation: Randomized; Intervention Model: Crossover Assignment; Masking: Triple (Participant, Care Provider, Outcomes Assessor); Primary Purpose: Treatment
Condition ICMJE	Cerebral Palsy
Intervention ICMJE	Biological: Autologous Stem Cells Other Name: Autolgous stem cells; from either banked umbilical cord blood or bone morrow harvest.; Other: Saline Infusion (Placebo)
Study Arms	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. Intervention: Biological: Autologous 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. Intervention: Biological: 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. Intervention: Other: Saline Infusion (Placebo)
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Completed
Actual Enrollment ICMJE; (submitted: November 10, 2016)	20
Original Estimated Enrollment ICMJE; (submitted: November 19, 2013)	30
Actual Study Completion Date	February 21, 2018
Actual Primary Completion Date	February 21, 2018 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	Inclusion Criteria: 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); Gross Motor Function Classification Score level II-V; Ages 24 months to 10 years; English speaking, if verbal; Ability to travel to Houston for treatment and follow-up - Exclusion Criteria: 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]; 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; Normal brain MRI; 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; Progressing neurological disease (as defined by Batten Disease, Leukodystrophies, Metabolic disorders, Mitochondrial disorders, Neurotransmitter disorders); Microcephaly, macrocephaly, cortical malformations, genetic disorders of dysgenesis brain malformations due to infection or metabolic disorders; Pulmonary disease requiring ventilator support; If hUCB candidate, banked cord cells totaling <10 million/kg; If hUCB candidate, any positive maternal infectious disease test (Hepatitis A, Hepatitis B, HIV 1, HIV 2, HTLV 1, HTLV 2, and Syphilis); If hUCB candidate, cord blood sample contamination; Participation in a concurrent intervention study; Unwillingness to return for follow-up visits; Contraindications to MRI; Any patient that the investigators feel in their opinion the study intervention is unlikely to benefit the patient will be a screen failure.; Any patients who are currently or has previously been enrolled in a clinical stem cell study.
Sex/Gender	Sexes Eligible for Study: All
Ages	2 Years to 10 Years (Child)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	United States
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT01988584
Other Study ID Numbers ICMJE	HSC-MS-12-0876
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Charles Cox, The University of Texas Health Science Center, Houston
Study Sponsor ICMJE	The University of Texas Health Science Center, Houston
Collaborators ICMJE	Cord Blood Registry, Inc.; Let's Cure CP Foundation; Mission Connect, a program of TIRR Foundation
Investigators ICMJE	Principal Investigator: Charles S Cox, MD UTHealth, Medical School, Dept. of Pediatric Surgery
PRS Account	The University of Texas Health Science Center, Houston
Verification Date	February 2018
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP