Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 -
排除标准
- •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.
研究组 & 干预措施
umbilical cord blood (UCB) cells
Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.
干预措施: umbilical cord blood (hUCB) cells (Biological)
bone marrow-derived mononuclear cells (BMMNCs)
Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.
干预措施: bone marrow derived mononuclear cells (BMMNCs) (Biological)
saline infusion (placebo), then umbilical cord blood (UCB) cells
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.
干预措施: umbilical cord blood (hUCB) cells (Biological)
saline infusion (placebo), then umbilical cord blood (UCB) cells
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.
干预措施: Saline Infusion (Placebo) (Drug)
saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs)
干预措施: Saline Infusion (Placebo) (Drug)
saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs)
干预措施: bone marrow derived mononuclear cells (BMMNCs) (Biological)
结局指标
主要结局
Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
时间窗: 24 hours after infusion
In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
Long-term Safety
时间窗: from the time of infusion to 1 year after infusion
Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status
次要结局
- Number of Participants With an Improvement in White Matter Integrity.(from baseline to 1 year after infusion)
- Gross Motor Function Classification Score (GMFM-66)(1 year after infusion)
- Gross Motor Function Classification Score (GMFM-88)(1 year after infusion)
- Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication(1 year after infusion)
- Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living(1 year after infusion)
- Score on Vineland Adaptive Behavior Scales (VABS-2) - Social(1 year after infusion)
- Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor(1 year after infusion)
- Score on Pediatric Evaluation of Disability Inventory - Self-Care(1 year after infusion)
- Score on Pediatric Evaluation of Disability Inventory - Mobility(1 year after infusion)
- Score on Pediatric Evaluation of Disability Inventory - Social(1 year after infusion)
- Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall(1 year after infusion)
- Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued(1 year after infusion)
- Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)(1 year after infusion)
- Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family(1 year after infusion)
- Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social(1 year after infusion)
- Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings(1 year after infusion)
- Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation(1 year after infusion)
- Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional(1 year after infusion)
- Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access(1 year after infusion)
- Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain(1 year after infusion)
- Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)(1 year after infusion)
研究者
Charles Cox
The Children's Fund Distinguished Professor, Department of Pediatric Surgery
The University of Texas Health Science Center, Houston