A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Safety Endpoint (number of patients and wounds that have an infection or any related adverse event.)
研究概览
简要总结
To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen [C7] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older.
详细描述
A multicenter, open-label, single-arm Phase 3b safety study of one-time surgical application of up to 12 autologous, LZRSE-Col7A1 gene-corrected keratinocyte sheets with C7 expression (EB-101) for the treatment of large, chronic DEB wounds in each of approximately 10-12 patients. Patients enrolled will also contribute biopsies to support manufacturing requirements. All patients will be followed through 24 weeks post-treatment.
Patients will be evaluated at their Screening Visit (D -60 to D -25), a phone call approximately 14 days after screening, and at Baseline (Day -1) prior to treatment on Day 0. Patients will remain in the hospital, or other suitable inpatient medical facility that will allow appropriate immobilization of treated wounds, as determined by the Investigator, for observation for up to 7 days (±2 days) following treatment. They will be evaluated by phone on Day 14; by telehealth visits on Weeks 4, 8, and 18; and by clinic visits on Weeks 12 and 24.
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
- 12 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of DEB.
- •Age 12 months and older.
- •Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent.
- •(This inclusion criterion was deleted as of Amendment 1.)
- •Confirmation of DEB diagnosis (either dominant [DDEB] or recessive [RDEB] forms) by genetic testing.
- •Able to undergo adequate anesthesia during EB-101 treatment.
- •All women of childbearing potential must have a negative urine pregnancy test and use a reliable birth control method throughout the duration of the study.
- •On stable pain medication regimen for at least 30 days prior to Screening (and through Baseline).
- •Must have at least one wound site that meets all of the following criteria:
- •An area ≥20 cm2,
- •Present for ≥6 months, and
- •Stage 2 wound defined as an open skin wound with partial thickness loss of dermis that has not extended through the dermis into subcutaneous tissue.
- •For patients with RDEB, they must have had Vyjuvek exposure by the time of screening or positive anti-C7 antibodies at baseline.
- •Patients must be willing to discontinue Vyjuvek and Filsuvez use on EB-101 treated sites until completion of the Week 24 visit.
排除标准
- •Medical instability limiting ability to travel to the study site or undergo EB-101 treatment.
- •The presence of medical illness expected to complicate participation and/or compromise the safety of this technique, such as active infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C.
- •(This exclusion criterion was deleted as of Amendment 3.)
- •Evidence of systemic infection.
- •Current evidence or a history of SCC in the area that will undergo EB-101 application.
- •Active drug or alcohol addiction.
- •Hypersensitivity to vancomycin or amikacin.
- •Receipt of chemical or biological investigational therapy for the specific treatment of DEB in the 3 months prior to EB-101 application.
- •Breast-feeding.
- •Inability to properly follow protocol assessments and protect keratinocyte sheet sites as determined by the PI.
- •Grade 3 clinical event or laboratory abnormality at Day
- •Abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch are expected in severe DEB patients, and these abnormalities will not exclude a patient.
- •Unwillingness or inability to provide 4 skin biopsies, or patient's keratinocytes cannot be manufactured for use in EB-101 application.
- •Any other circumstance where the PI believes that the patient may not be appropriate for participation in the study.
研究组 & 干预措施
EB-101 Surgical application of RDEB wounds
New or Previously Treated RDEB Patients
干预措施: EB-101 Surgical application of RDEB wounds (Biological)
结局指标
主要结局
Safety Endpoint (number of patients and wounds that have an infection or any related adverse event.)
时间窗: 6 months
The number of patients and wounds that have an infection or any related adverse event (AE).
Safety Endpoint (number of patients and wounds that result in hospitalization (serious adverse event )
时间窗: 6 months
The number of patients and wounds that result in hospitalization (SAE).
Safety Endpoint RCR status
时间窗: 6 months
Replication-competent retrovirus (RCR) status.
Safety Endpoint (number of treatment-related and treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
时间窗: 6 months
The number of treatment-related and treatment-emergent adverse events (TEAEs), treatment-emergent wound adverse events (TEWAEs), and serious adverse events (SAEs), including systemic and wound-specific adverse events.
Safety Endpoint (Incidence of squamous cell carcinoma)
时间窗: 6 months
The incidence of squamous cell carcinoma (SCC).
次要结局
未报告次要终点