A Randomized, Blinded, Placebo-controlled, Crossover Study to Assess the Efficacy of Stem Cells From Autologous Umbilical Cord Blood to Improve Language and Behavior in Children With Autism
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Change in Language (Total Standard Score, Range 40 - 160)
研究概览
简要总结
Evaluate the efficacy of one infusion of stem cells from autologous umbilical cord blood in patients with autism over six months after infusion as measured by changes in expressive and receptive language.
Also demonstrate improved behavior, learning, and changes in Serum tumor necrosis factor alpha (TNF-α), tumor necrosis factor beta (TNF-β), interleukin 1-alpha (IL-1α), interleukin 1-beta (IL-1β), interleukin 6 (IL-6), interleukin 10 (IL-10), and interleukin 13 (IL-13).
详细描述
This is a single-center, randomized, placebo-controlled, crossover outpatient study with 15 subjects receiving one infusion of autologous umbilical cord blood (AUCB) containing a minimum of 10 million total nucleated cells per kilogram (TNC/kg) and 15 subjects receiving an infusion of placebo (saline). After the 24-week follow-up testing is conducted, the groups will crossover so that patients who initially received AUCB will receive placebo and patients who received placebo at baseline will receive the cord blood. Both groups will be tested again 24-weeks after infusion. The neuropsychologist, PI, staff from Cord Blood Registry (CBR), and parents will be blinded as to the infusion sequence.
The duration of participation for each study subject is approximately 55 weeks. This includes one screening visit over a period of approximately 6 weeks, one visit for baseline testing, one day for infusion of TNC (minimum 10 million/kg) or saline placebo followed by 24 weeks of follow-up. A second baseline visit is conducted at week-24 with the second infusion of TNC or saline placebo occurring 5-7 days after. Twenty-four additional weeks of follow-up occur after the second infusion.
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, Investigator)
入排标准
- 年龄范围
- 2 Years 至 7 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 2 to 7 years of age
- •Diagnosis of Autistic Disorder as diagnosed by the Diagnostic and Statistical Manual, 4th Edition, Text Revision (DSM-IV-TR) developmental delays, and ADOS
- •A sufficient quantity of autologous cord blood stored at Cord Blood Registry that was stored and processed using the Thermogenesis AutoXpress Platform
- •Stable on any current medications for at least 2 months prior to infusion of cord blood
- •Medical records indicating that patient does not have genetic conditions such as cerebral palsy, cystic fibrosis, muscular dystrophy, crohns disease, rheumatoid disease, fragile X, Retts Syndrome, Angelman Syndrome, tuberous sclerosis, epilepsy, or known genetic defects that overlap autism spectrum.
- •Results of an EEG within 12-months of baseline
- •English speaking
排除标准
- •CNS infection
- •Extreme prematurity (< 34 weeks gestation)
- •Severe Cognitive Disability IQ below 45 with autism
- •Clinical seizure activity within 6 months of baseline
- •Lennox Gastaut syndrome or infantile spasms
- •Dravet syndrome
- •HIV, renal or hepatic impairment
- •Prior hematological or malignant disease
- •Fever of 101 F within 2 weeks prior to infusion
- •Serious CNS infection or trauma
- •Unwilling to commit to follow-up
- •Mental illness including schizophrenia
- •Pervasive Developmental Disorder-Not Otherwise Specified
- •Asperger's Disorder
- •Cord blood unit is less than 85% viable, has a TNC of less than 10 million/kg, or sterility testing results are positive
- •Garlic allergy
- •Previous adverse reaction to Dimethyl Sulfoxide (DMSO)
- •Maternal medical records indicate communicable diseases including HIV, Hepatitis B or C, syphilis, cytomegalovirus (CMV)
- •Currently taking anti-inflammatory medications
- •History of asthma who may potentially require treatment with steroids
- •Inflammatory Disease
- •Renal/hepatic disease: serum Creatinine > 1.5 mg/dl and total Bilirubin > 1.5 mg/dl
- •Allergic to diphenhydramine (Benadryl)
- •Treatment with chelation therapy, hyperbaric oxygen therapy, pig worm therapy, or other alternative therapies the investigator deems clinically relevant
研究组 & 干预措施
Autologous Cord Blood Stem Cells
干预措施: Autologous Cord Blood Stem Cells (Biological)
Placebo
Saline
干预措施: Placebo (Biological)
结局指标
主要结局
Change in Language (Total Standard Score, Range 40 - 160)
时间窗: Baseline and 6 months
Change in language as measured by the Receptive One-Word Vocabulary Test (ROWVT-4) and Expressive One-Word Vocabulary Test (EOWVT-4) at baseline and six months following infusion of stem cells from AUCB or infusion of placebo. The ROWPVT-4 and EOWPVT-4 were administered by a neuropsychologist and are norm-referenced assessments. The ROWPVT-4 tests an individual's ability to match a spoken word with an image of an object, action, or concept. The EOWPVT-4 tests an individual's ability to name, with one word, objects, actions, and concepts when presented with color illustrations. The tests target the ability to understand the meaning of words spoken and name what is depicted on a test plate without context. Scores of 85-115 are considered to be within the average range of functioning.
次要结局
- Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)(Baseline and 6 months)
- Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)(Baseline and 6 months)
研究者
Michael Chez, MD
Principal Investigator
Sutter Health