A Phase 2 Open-Label Study of PTR-01 in Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Incidence of anti-drug antibodies (ADA)
研究概览
简要总结
Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.
详细描述
Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.
In Part 1, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 2 in which patients will receive a dose of 3.0 mg/kg every other week for a total of 7 doses. In Part 3, patients will be followed for 12 weeks. No investigational therapy will be administered during this time. At the end of each dosing period, an efficacy assessment will be performed. Safety will be assessed continuously throughout the study.
Following the end of Part 3, patients may be eligible for a potential long-term extension to further refine the dosing regimen, depending upon study drug availability.
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
盲法说明
Open-label
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must meet all of the following criteria to be eligible for study participation in the three month run in period of the study:
- •Willing to provide informed consent form, or if 12 to <18 years of age, legal guardian has provided informed consent form and the minor has signed an assent form acknowledging that they understand and agree to study procedures.
- •Has a diagnosis of RDEB based on genetic analysis and consistent with a recessive inheritance pattern.
- •Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.
- •Agrees to use contraception as follows:
- •For women of childbearing potential (WOCBP) agrees to use highly effective contraceptive (including abstinence) methods from Screening, through the study, and for at least 10 weeks after the last dose of study drug. Non-childbearing potential is defined as a female who meets either of the following criteria: age ≥50 years and no menses for at least 1 year or documented hysterectomy, bilateral tubal ligation, or bilateral oophorectomy.
- •For males, agrees to use a condom with any WOCBP sexual partner from Day 1 of study treatment, through the study, and at least 10 weeks after the last dose of study drug.
- •Be willing and able to comply with this protocol.
排除标准
- •Patients with any of the following will be excluded from participation in the study:
- •Has known systemic hypersensitivity to any of the inactive ingredients in PTR-
- •Has previously had an anaphylactic reaction to PTR-
- •Is pregnant or nursing.
- •Has received in the last six months any investigational gene therapy product or in the last three months any non-gene therapy investigational products.
- •Is anticipated to receive new regimens of antibiotics or other anti-infectives during the trial.
- •Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the patient, or may preclude the patient's
研究组 & 干预措施
PTR-01 3 mg/kg
All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg every other week for a total of 7 doses.
干预措施: PTR-01 (Drug)
结局指标
主要结局
Incidence of anti-drug antibodies (ADA)
时间窗: Up to 162 days
Safety and tolerability, as assessed by immunogenicity through anti-drug antibody (ADA) testing
Wound healing
时间窗: Up to 162 days
Change in a majority of target lesions of at least 2 levels using a 7-point (1-7) Global Impression of Change instrument (7 being the worst)
Incidence of treatment-emergent adverse events
时间窗: Up to 162 days
Safety and tolerability, as assessed by treatment-emergent adverse events
Incidence of infusion-associated reactions
时间窗: Up to 162 days
Safety and tolerability, as assessed by infusion-associated reactions (IAR)
次要结局
- Change in amount of wound care(Up to 162 days)
- Formation of anchoring fibrils(Up to 162 days)
- Change in wound surface area, as assessed by Investigator Global Impression of Change (IGIC)(Up to 162 days)
- Change in total body wound surface area(Up to 162 days)
- Change in skin integrity, as assessed by suction blister time(Up to 162 days)
- Change in itch severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains(Up to 162 days)
- Change in itch severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)(Up to 162 days)
- Change in the impact of itch on quality of life(Up to 162 days)
- Change in pain severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)(Up to 162 days)
- Change in the impact of pain on quality of life(Up to 162 days)
- Change in corneal symptoms(Up to 162 days)
- Rate of change in nutritional markers (total protein/albumin)(Up to 162 days)
- Rate of change in nutritional markers (C-reactive protein)(Up to 162 days)
- Change in skin integrity, as assessed by time to re-blistering(Up to 162 days)
- Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire(Up to 162 days)
- Stabilization of corneal symptoms(Up to 162 days)
- Rate of stabilization of nutritional markers (hemoglobin/hematocrit)(Up to 162 days)
- Rate of stabilization of nutritional markers (iron/TIBC)(Up to 162 days)
- Change in time for wound care(Up to 162 daysUp to 162 days)
- Delivery of PTR-01 to skin(Up to 162 days)
- Change in wound surface area, as assessed by wound imaging(Up to 162 days)
- Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Scale(Up to 162 days)
- Rate of change in nutritional markers (iron/TIBC)(Up to 162 days)
- Rate of stabilization of nutritional markers (C-reactive protein)(Up to 162 days)
- Change in Investigator Global Impressions of Change (IGIC)(Up to 162 days)
- Change in Investigator Patient Impressions of Change (PGIC)(Up to 162 days)
- Change in mental health(Up to 162 days)
- Change in pain severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) pain domains(Up to 162 days)
- Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire(Up to 162 days)
- Change in overall health(Up to 162 days)
- Change in social function(Up to 162 days)
- Change in cost of wound care(Up to 162 days)
- Change in overall patient impression of quality of life(Up to 162 days)
- Change in overall patient impression of disability(Up to 162 days)
- Change in dysphagia, as assessed by volume of oral nutritional intake(Up to 162 days)
- Stabilization of dysphagia, as assessed by volume oral nutritional intake(Up to 162 days)
- Rate of change in nutritional markers (hemoglobin/hematocrit)(Up to 162 days)
- Rate of stabilization of nutritional markers (total protein/albumin)(Up to 162 days)
- Change in disease activity and scarring(Up to 162 days)