Evaluation of Safety and Exploratory Efficacy of CARTISTEM®, a Cell Therapy Product for Articular Cartilage Defects: A Phase I/IIa Clinical Trial in Patients With Focal, Full-thickness Grade 3-4 Articular Cartilage Defects of the Knee
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- IKDC Score
研究概览
简要总结
The purpose of this study is to determine whether CARTISTEM, a cell therapeutic product, is safe and effective in the treatment of articular cartilage defects of the knee as a result of ageing, trauma, or degenerative diseases.
详细描述
Conventional treatment modalities have not been able to provide complete and sustained resolution of symptoms following damage to the articular cartilage. Despite the numerous techniques available today, complete healing of damaged or defective cartilage or consistent reproduction of normal hyaline cartilage does not occur, and continuous drug administration or secondary surgeries are common.
Research in mesenchymal stem cells has had a rapid acceleration over the past decade and MSC-based therapy has become one of the objects of investigation for a new branch of medicine termed regenerative medicine. This emerging technology shows great promise for producing transplantable cartilage constructs to restore the function of degenerated joints.
CARTISTEM®, a combination of human umbilical cord blood-derived mesenchymal stem cells and sodium hyaluronate, is intended to be used as a single-dose cellular therapeutic agent for cartilage regeneration in human subjects with cartilage defects of the knee as a result of ageing, trauma, or degenerative diseases.
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
- •Patients with an intended- to- treat single focal, full-thickness cartilage defect (ICRS [International Cartilage Repair Society] Grade 3 or 4) of the knee as a result of ageing, trauma, or degenerative diseases.
- •Age ≥ 18 years old
- •Size of the articular cartilage lesion is ≥ 2 cm2
- •Swelling, tenderness and active range of motion ≤ Grade II
- •Joint pain : 20-mm - 60-mm on VAS (Visual Analog Scale) at the time of Screening
- •Appropriate blood coagulation, kidney and liver function laboratory parameters: PT(INR) < 1.5, APTT <1.5×control Creatinine ≤ 2.0 mg/dL Albumin ≤ trace in urine dipstick test Bilirubin ≤ 2.0 mg/dL, AST/ALT ≤ 100 IU/L
- •Ligament instability ≤ Grade II
- •Lower extremity alignment within 5 degrees of the neutral weight bearing axis
- •No meniscal surgery within the past 3 months and more than 5mm of meniscal rim remaining
- •Ability and willingness to fully participate in the post-operative rehabilitation program
- •Subject is informed of the investigational nature of this study, voluntarily agrees to participate in the study, and signs an IRBapproved informed consent prior to performing any of the screening procedures
- •Body Mass Index (BMI) ≤ 35 kg/m2
排除标准
- •Patients who have been treated previously and are asymptomatic
- •Avascular necrosis/ osteonecrosis
- •Autoimmune or inflammatory joint disease
- •History of infection within the past 6 weeks
- •Surgery or radiation therapy within the past 6 weeks
- •Serious medical co-morbidities, which would otherwise contraindicate surgery, as determined by the investigator
- •Currently pregnant or nursing
- •Psychotic diseases, epilepsy, or any history of such diseases
- •Current abuse of alcohol (> 10 drinks weekly) and/or regular exposure to other substances of abuse, currently an active smoker
- •Chronic inflammatory articular diseases such as rheumatoid arthritis
- •Enrolled in any other clinical trials within the past 4 weeks
- •Administered immunosuppressants such as Cyclosporin A or azathioprine within the past 6 weeks
- •Ligament instability > Grade II
- •Uncorrected significant lower extremity malalignment (i.e. > 5 degrees)
- •(sub-) Total meniscectomy (<5mm rim remaining)
- •Corticosteroid or viscosupplementation injection to the affected knee in the past 3 months
- •Principal investigator considers inappropriate for the clinical trial due to any reasons other than those listed above
研究组 & 干预措施
CARTISTEM®
Drug name and ingredients: CARTISTEM [allogeneic-unrelated, umbilical cord blood-derived mesenchymal stem cells, ex vivo cultured, combined with sodium hyaluronate] Dosage: Administer 0.5 mL of the combination product per cm^2 of the cartilage defect
干预措施: CARTISTEM® (Biological)
结局指标
主要结局
IKDC Score
时间窗: 12 months
Subjects underwent a self-assessment of knee function using the IKDC (International Knee Documentation Committee Assessment)subjective knee evaluation before and after the administration of the IP. The assessment is designed to detect improvement or deterioration in symptoms, function, and sports activities due to knee impairment. The possible score ranges from 0 to 100, where 100 = no limitation with daily or sporting activities and the absence of symptoms. The IKDC assessment was performed before IP administration and at Months 12 after IP administration in an exploratory fashion. The IKDC score from the Month 12 visit was used as a primary indicator of the post operative change. The outcome was presented with the post-operative change amount from the baseline.
次要结局
- Lysholm Score(24 months)
- IKDC Score(24 months)
- VAS(24 months)
- KOOS Score(24 months)