A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
- 试验地点
- 5
- 主要终点
- Complete Wound Closure of the First Wound Pair at Week 24
研究概览
简要总结
The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa.
Funding Source - FDA OOPD
详细描述
DEFI-RDEB is a multi-center, intra-patient randomized, controlled, open-label, Phase 3 study of FCX-007 for the treatment of persistent non-healing and recurrent RDEB wounds in approximately 24 subjects. Each subject will serve as his/her own control. Each subject's target wounds will be paired then randomized to receive FCX-007 (treatment wound) or remain untreated (control wound). Up to three target wound pairs will be identified for each subject.
Subjects will receive intradermal injections of FCX-007 in each specified treatment wound in two or more treatment sessions. The first treatment session occurs at Day 1 and the second at Week 12/Month 3. Additional treatment sessions may occur at Week 24/Month 6 and Week 36/Month 9 when unclosed treatment wounds may be re-treated, and unclosed control wounds may be treated. Safety and efficacy assessments will occur at scheduled intervals through Week 48/Month 12, when the treatment period is completed, and a long-term safety follow-up period (through 15 years) commences for subjects who have received one or more FCX-007 injections.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female ≥2 years of age at the Screening visit.
- •Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.
排除标准
- •Medical instability limiting ability to travel to the investigative site.
- •Active infection with human immunodeficiency virus, hepatitis B or hepatitis C.
- •The presence of COL7 antibodies.
- •Evidence of systemic infection.
- •Evidence or history of squamous cell carcinoma at the site to be injected.
- •Evidence of or history of metastatic squamous cell carcinoma.
- •Known allergy to any of the constituents of the product.
- •Female who is pregnant or breastfeeding.
- •Receipt of a chemical or biological intervention for the specific treatment of RDEB in the past three (3) months prior to screening or anticipated/planned during the screening and treatment period for this study.
研究组 & 干预措施
FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts
Intra-subject randomized (paired wounds in each subject receive experimental treatment, FCX-007, or remain untreated). Up to three target wound pairs will be identified for each subject. Following pairing, target wounds will be randomly assigned as the treatment wound (FCX-007 is administered) or control wound. Subjects will receive intradermal injections of FCX-007 in each specified treatment wound in two or more treatment sessions. The first treatment session occurs at Day 1 and the second at Week 12/Month 3. Additional treatment sessions may occur at Week 24/Month 6 and Week 36/Month 9 when unclosed treatment wounds may be re-treated, and unclosed control wounds may be treated.
干预措施: FCX-007 (dabocemagene autoficel; see below for FCX-007 description) (Biological)
结局指标
主要结局
Complete Wound Closure of the First Wound Pair at Week 24
时间窗: Week 24
Complete wound closure of the first wound pair (treated vs. control)
次要结局
- Complete Wound Closure of All Wound Pairs at Week 12(Week 12)
- Complete Wound Closure of the First Wound Pair at Week 12(Week 12)
- Complete Wound Closure of All Wound Pairs at Week 24(Week 24)