NCT04651855Unknown1 期
The Evaluation of the Effect of Wharton's Jelly Mesenchymal Stem Cells (WJMSCs) on the Immune System of Patients With Amyotrophic Lateral Sclerosis (ALS)
Polski Bank Komorek Macierzystych JSC (PBKM)1 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2020年12月2日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- The number of (S)AESI [(Serious) Adverse Event of Special Interest]
研究概览
简要总结
The objective of this study is to evaluate the safety of intrathecal administration of Wharton's Jelly Mesenchymal Stem Cells (WJMSC) and the impact on the immune system of patients with Amyotrophic Lateral Sclerosis.
详细描述
Clinical Phase: I/II
Population: Patients with Amyotrophic Lateral Sclerosis.
Project Design: One arm, non-blinded, open label study
Planned Sample Size: 20 patients
Investigational Medicinal Product: active IMP - mesenchymal stem cells isolated from Wharton's jelly
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult patients (at least 18 years old)
- •The minimum patient's weight is not less than 40 kg
- •Diagnosis of sporadic ALS, definite or probable, as defined by El Escorial World Federation of Neurology criteria
- •History of ALS symptoms less than 2 years duration from the first symptoms of the disease
- •More than 6 months from diagnosis of the disease
- •Disease progression at 6 past months at least 3 points during this period of time assessed in ALSFRS-R scale
- •ALSFRS-R scale of at least 30 at screening appointment
- •Forced vital capacity >70% of predicted value for age, gender and height
- •Treatment with stable dose of riluzole(2x 50mg per 24h) before baseline visit (for at least 1 month)
- •Capable of providing written informed consent
- •Able to comply with study requirements and willing to follow all study procedures and follow-up visits
- •Women of child-bearing age and men with partners of child-bearing potential must agree to use two forms of contraceptive therapy throughout the course of the trial
- •Women of child-bearing age must undergo pregnancy test
- •Polish-language native speakers or patients who are proficient in the Polish language
排除标准
- •Pregnancy or breastfeeding
- •Tracheostomy
- •Ventilator dependence
- •Renal disease with creatinine >2mg/dl
- •Liver disease with ALT, AST or GGTP 2-fold higher than upper normal limit
- •Positive test for HBV, HCV, HIV with NAT method
- •Positive tests for syphilis
- •Any other clinically significant abnormalities on laboratory evaluation
- •Any condition that would compromise ability of undergoing lumbar puncture
- •Active systemic disease
- •Autoimmune disease (Hashimoto disease under control is allowed)
- •Uncontrolled diabetes (HbA1c > 8%)
- •Pulmonary disease that could affect interpretation of spirometry
- •Neurological concomitant disease
- •Unstable psychiatric concomitant disease
- •High risk of suicide
- •History of substance abuse within past year
- •History of malignancy, within the previous 5 years, including melanoma with exception of localized skin cancers
- •Any other clinically significant medical condition that can compromise patient's safety in the opinion of the investigator
- •Treatment with immunomodulatory drugs (for example immunoglobulins, corticosteroids or other immunosuppressant) in last 6 months
- •Participation in another clinical trial in last 6 months
- •Previous cellular therapy of any kind
- •Hypersensitivity to any component used in the cell culture
- •Nuchal rigidity and other signs of meningitis
- •Patients on chronic anticoagulation treatment (heparin/ warfarin/acenocoumarol/(N)OAC)
研究组 & 干预措施
Treatment arm
Experimental
It is planned that IMP administration will be performed three times for each enrolled patient. IMP administration could be performed only if the patients does not have any contraindications for lumbar puncture.
干预措施: Mesenchymal stem cells isolated from Wharton's jelly (Drug)
结局指标
主要结局
The number of (S)AESI [(Serious) Adverse Event of Special Interest]
时间窗: 3 month FU (follow-up)
(S)AESI are defined as: 1. Meningitis and encephalitis. 2. Toxic encephalopathy. 3. High fever \>39⁰C. 4. Epileptic seizures that are not connected to conditions above (meningitis, encephalitis, toxic encephalopathy, high fever).
次要结局
- Muscle strength decline(screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 6, 9 and 12 month FU)
- Disease progression(screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 and 18 month FU)
- Pulmonary function decline(screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 6, 9 and 12 month FU.)
- Upper motor neuron function(screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 6, 9 and 12 month FU)
- Quality of life changes(screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 and 18 month FU)
- Muscle function changes(baseline and at 1, 2, 6 and 12 month FU)
- Survival period to disease progression(18 month FU)
- Cognitive function(screening and 12 month FU)
- The change of defined cytokines, chemokines, growth factors and pNFH (phosphorylated neurofilament heavy chain) level in CSF (Cerebrospinal fluid)(run-in visit (-60 day), at baseline and at 1, 2 and 6 month FU (12 month FU optional))
- The change of defined cytokines, chemokines level in blood(screening visit, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 6, 9 and 12 month FU.)
- The change of creatinine and p75ECD level in urine(screening visit, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 6, 9 and 12 month FU.)
- SAE (Serious Adverse Event)/AE (Adverse Event) and (S)AESI(18 month FU)
- The change of the brain visualization(run-in visit (-60 day), 6 and 12 month FU)
- Mortality rate(18 month FU)
研究者
研究点 (1)
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