A Phase 2b Open-Label Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01 in Study PTR-01-002
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 2
- 试验地点
- 2
- 主要终点
- Incidence of treatment-emergent adverse events
研究概览
简要总结
A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria.
详细描述
Protocol PTR-01-003 is a 4-part Phase 2, open-label study of PTR-01 in patients who satisfactorily completed study PTR-01-002 and meet current enrollment criteria.
In Part 1, patients will be monitored monthly for up to 2 months with patient-reported and Investigator assessments. In Part 2, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 3 in which patients will receive a dose of 3.0 mg/kg monthly for a total of 5 additional doses. At the end of each dosing period, efficacy assessments will be performed. During Part 4, patients will be evaluated at Months 1 and 3 after completion of dosing to assess the durability of wound healing and other efficacy parameters. Safety will be assessed continuously throughout the study.
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
- •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 satisfactorily completed participation in PTR-01-
- •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
- •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 (other than PTR-01).
- •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
研究组 & 干预措施
PTR-01
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 once monthly for a total of 5 doses.
干预措施: PTR-01 (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events
时间窗: Up to 246 days
Safety and tolerability, as assessed by treatment-emergent adverse events
Sustained wound healing
时间窗: Up to 246 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)
次要结局
- Change in wound surface area(Up to 246 days)
- Delivery of recombinant collagen 7 (PTR-01) to skin(Up to 246 days)
- Formation of anchoring fibrils(Up to 246 days)
- Change in skin integrity, as assessed by suction blister time(Up to 246 days)
- Change in skin integrity, as assessed by time to re-blistering(Up to 246 days)
- Change in the impact of itch on quality of life(Up to 246 days)
- Stabilization of corneal symptoms(Up to 246 days)
- Change in itch severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains(Up to 246 days)
- Change in pain severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) pain domains(Up to 246 days)
- Rate of change in nutritional markers (hematocrit)(Up to 246 days)
- Change in pain severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)(Up to 246 days)
- Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire(Up to 246 days)
- Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Instrument(Up to 246 days)
- Rate of change in nutritional markers (hemoglobin)(Up to 246 days)
- Rate of change in nutritional markers (total protein/albumin)(Up to 246 days)
- Rate of change in nutritional markers (Fe/TIBC)(Up to 246 days)
- Rate of change in nutritional markers (C-reactive protein)(Up to 246 days)
- Change in corneal symptoms(Up to 246 days)
- Change in Investigator Global Impressions of Change (IGIC)(Up to 246 days)
- Change in Investigator Patient Impressions of Change (PGIC)(Up to 246 days)
- Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire(Up to 246 days)
- Change in amount of wound care(Up to 246 days)
- Change in overall health(Up to 246 days)
- Change in mental health(Up to 246 days)
- Change in social function(Up to 246 days)
- Change in time for wound care(Up to 246 days)
- Change in cost of wound care(Up to 246 days)
- Change in overall patient impression of quality of life(Up to 246 days)
- Change in overall patient impression of disability(Up to 246 days)
- Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of clearance of PTR-01(Up to 246 days)
- Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of PTR-01 half-life(Up to 246 days)
- Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Cmax(Up to 246 days)
- Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Tmax(Up to 246 days)
- Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Area Under the Curve (AUC)(Up to 246 days)