A Double-Blind, Randomized, Placebo-Controlled Phase II Clinical Trial to Evaluate the Safety and Efficacy of L2-01 in Subjects With Crohn's Disease
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 120
- 主要终点
- Incidence of adverse events occurring in less than 5% of study participants as a measure of safety.
研究概览
简要总结
This clinical trial is testing a new treatment called L2-01 in patients with Crohn's Disease, a condition that causes ongoing inflammation of the digestive tract. Current treatments for Crohn's Disease do not work for everyone, and some patients still experience symptoms even with standard therapies.
The purpose of this study is to determine if a single intravenous infusion of L2-01, which uses cells called mesenchymal stem cells derived from a patient's own or their close relative's body fat, is safe and can help improve symptoms of Crohn's Disease. Researchers will compare the effects of L2-01 to a placebo (an inactive substance) to see if L2-01 helps reduce inflammation and improves quality of life in patients.
The main questions the study aims to answer are:
Is L2-01 safe for patients with Crohn's Disease?
Can L2-01 effectively reduce symptoms and improve the health of people living with Crohn's Disease compared to a placebo?
The researchers believe that L2-01 will be safe and more effective than placebo in reducing inflammation, decreasing disease activity, and improving patients' quality of life.
详细描述
This Phase II clinical trial is a randomized, double-blind, placebo-controlled study designed to evaluate the safety and efficacy of L2-01, a human adipose-derived mesenchymal stem cell (MSC) product, in patients with Crohn's Disease (CD). L2-01 consists of MSCs harvested from the patient's own adipose tissue or that of a first-degree relative, expanded in culture, and administered as a single intravenous infusion at a dose of 1 × 10^6 cells per kilogram of body weight.
The study enrolls 120 adult subjects with non-active Crohn's Disease, defined by a Crohn's Disease Activity Index (CDAI) of 200 or less. Patients are randomized in a 1:1 ratio to receive either L2-01 or placebo (sterile normal saline). The initial 20 patients are enrolled in a staggered manner to closely monitor safety signals, followed by enrollment of an additional 100 subjects if no significant adverse drug reactions are observed.
Subjects undergo liposuction to collect approximately 25 ml of lipoaspirate from the lower abdomen or flanks under local anesthesia. The MSCs are isolated, expanded under Good Manufacturing Practice (GMP) conditions, cryopreserved, and then thawed and prepared as a ready-to-infuse suspension prior to administration. Placebo infusions contain an identical volume of lactated Ringer's solution mixed with saline, maintaining blinding integrity.
The infusion is administered intravenously over approximately 30 minutes with close monitoring for adverse events, vital sign changes, and infusion reactions during and after the procedure. Safety assessments include monitoring treatment-emergent adverse events (TEAEs), laboratory tests (including comprehensive metabolic panel, complete blood count, coagulation studies, and inflammatory markers), physical examinations, vital signs, and adverse event reporting per MedDRA coding.
Efficacy assessments focus on changes in systemic disease activity, inflammatory markers, imaging (pelvic MRI), and quality of life metrics (using the IBDQ-32 questionnaire). Follow-up visits occur at scheduled intervals up to 24 weeks post-infusion to assess both safety and therapeutic effects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Study monitors will also be blinded
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 Crohn Disease (CD) diagnosed within 12 months before acceptance of clinical, endoscopical, anatomopathological and/or radiological criteria and have a non-active CD. (Crohn´s Disease Activity Index (CDAI)≤ 200)
- •Any gender, > 18 Years of age
- •Negative pregnancy test in female subjects
- •Patients voluntarily sign the informed consent before performance of any study-related procedures
- •Patient is, in the investigator's opinion, willing and able to comply with the protocol requirements
排除标准
- •Patients who have received infliximab or any other anti-TNF agent within 8 weeks before the cell treatment administration.
- •Patients who have received tacrolimus or cyclosporine within 4 weeks before cell treatment.
- •Patients with a history of alcohol or other addictive substance abuse within 6 months before inclusion.
- •Severe uncontrolled diseases (chronic renal failure, cardio, pulmonary, etc.).
- •Any type of medical or psychiatric disease which are considered an exclusion criterion, in the investigator's opinion.
- •Patients with a diagnosis of malignant neoplasia, except basal cell or epidermoid carcinoma of the skin, or a previous history of malignant tumors, except those that have no evidence of relapse for at least 5 years.
- •Subjects with congenital or acquired immunodeficiency.
- •Subjects who tested positive for HIV 1/2, HBV, HCV, HTLV I/II, WNV, and syphilis
- •Flu symptoms
- •Patient had major surgery or serious trauma within 6 weeks before enrolment.
- •Pregnant or breastfeeding women.
- •Physical or psychological impossibility of following the protocol requirements
- •Patients who are receiving or have received another investigational drug within 30 days prior to the first visit.
研究组 & 干预措施
L2-01 Treatment Arm
Participants in this arm receive a single intravenous infusion of L2-01, a human adipose-derived mesenchymal stem cell product. The dose is 1 × 10^6 MSC cells per kilogram of body weight, suspended in 10 ml of lactated Ringer's solution mixed with 100 ml of saline. The infusion is administered over approximately 30 minutes. MSCs are derived from the patient's own adipose tissue or from that of a first-degree relative.
干预措施: L2-01 adipose-derived mesenchymal stem cells (MSC) (Biological)
Placebo Comparator Arm
Participants in this arm receive a single intravenous infusion of placebo, consisting of 10 ml of lactated Ringer's solution mixed with 100 ml of saline. The infusion is administered over approximately 30 minutes. The placebo product is packaged identically to the active product to maintain study blinding.
干预措施: Placebo saline solution (Other)
结局指标
主要结局
Incidence of adverse events occurring in less than 5% of study participants as a measure of safety.
时间窗: From the day of infusion through 12 weeks post-infusion.
次要结局
- Relapse rate among patients who achieved treatment success with adipose-derived mesenchymal stem cells.(Up to 24 weeks post-infusion.)
- Change in Crohn's Disease Activity Index (CDAI) from baseline to 12 weeks post-infusion.(Baseline and 12 weeks post-infusion.)
- Improvement in inflammatory markers and MRI results from baseline to 12 weeks post-infusion.(Baseline and 12 weeks post-infusion.)
- Change in quality of life as assessed by the Inflammatory Bowel Disease Questionnaire (IBDQ-32) from baseline to 12 weeks post-infusion.(Baseline and 12 weeks post-infusion.)
- Biological characterization of the cell product (phenotype study, suppressor capacity, cytokine production) and its correlation with therapeutic effect.(At infusion and during follow-up assessments up to 24 weeks.)