NCT05464381进行中（未招募）3 期
A Double-blind, Randomized, Placebo-controlled, Interventional, Multicenter, Phase III Clinical Trial to Investigate the Safety and Efficacy of ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs) on Epidermolysis Bullosa (EB)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 91
- 试验地点
- 16
- 主要终点
- Proportion of subjects with complete target wound closure
研究概览
简要总结
The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo. An additional baseline-controlled open-label arm will be included to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with JEB and to RDEB subjects < 1 year.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects with a diagnosis of RDEB or JEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM). A minimal body weight of at least 5 kg is required;
- •Subject is eligible to participate in this clinical trial based on general health condition;
- •Subject with a target wound meeting the following criteria: 5-50 cm2, and < 9 months, no signs of acute infection;
- •Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
- •Women of childbearing potential must have a negative urine pregnancy test at Visit
- •Women of childbearing potential, male participants, and their partner must be willing to use highly effective contraceptive methods during the course of the entire clinical trial.
排除标准
- •Signs and/or a history of skin precancerous and cancerous lesions at screening or any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
- •Known chronic lung disease;
- •Clinically significant laboratory values for coagulation and thrombocytes at screening;
- •Thromboembolic events of any grade in medical history;
- •Immunoreactions of any grade in medical history;
- •Any known allergies to components of the IP or premedication;
- •Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
- •Pregnant or lactating women;
- •Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
- •Previous participation in this clinical trial (except for screening failures);
- •Clinically significant or unstable concurrent disease or other clinical contraindications like an uncontrolled or poorly controlled mental health condition of the subject and/or his/her legal representative that could impact on patient's safety or interfere with study compliance such as inability to attend scheduled study visits;
- •Employees of the sponsor, or employees or relatives of the investigator.
研究组 & 干预措施
Verum
Experimental
干预措施: allo-APZ2-OTS (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Proportion of subjects with complete target wound closure
时间窗: 6 months
Proportion of subjects with complete healing of target wound, determined by the investigator, as compared to baseline
次要结局
未报告次要终点
研究者
研究点 (16)
Loading locations...
相似试验
进行中（未招募）
3 期
Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)Epidermolysis BullosaNCT05838092RHEACELL GmbH & Co. KG9
进行中（未招募）
3 期
CLINICAL STUDY TO TEST STEM CELLS FOR THE TREATMENT OF LONG-TERM, NON-HEALING VENOUS LEG ULCERS2024-512720-11-00Rheacell GmbH & Co. KG230
已完成
2 期
Phase II Study of STA-2 in Patients With Chronic Stable AnginaChronic Stable AnginaNCT01484912Sinphar Pharmaceutical Co., Ltd79
已完成
2 期
Study to Assess the Efficacy and Safety of Atuliflapon in Moderate-to-Severe Uncontrolled AsthmaAsthmaNCT05251259AstraZeneca670
已完成
3 期
Safety and Efficacy of AST-120 in Mild to Moderate Crohn's Patients With FistulasIntestinal FistulaInflammatory Bowel DiseaseNCT00321412Ocera Therapeutics191