An Open-Labeled, Single Arm Phase 2 Efficacy and Safety Study of REM-001 Photodynamic Therapy (PDT) for Treatment of Cutaneous Metastatic Breast Cancer (CMBC)

REM-001 光动力疗法 (PDT) 治疗皮肤转移性乳腺癌 (CMBC) 的开放标记单臂 2 期疗效和安全性研究

• 批准号: 10699535
• 负责人: Dennis Brown
• 金额: $ 125.42万
• 依托单位: KINTARA THERAPEUTICS, INC.
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-07-01 至 2025-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10699535
• 关键词: 3-Dimensional; Acute; Adverse event; Aftercare; Area; Calibration; Clinical; Clinical Research; Cohort Studies; Common Terminology Criteria for Adverse Events; Coupled; Custom; Cutaneous; Dermal; Digital Photography; Dimensions; Dose; Electrocardiogram; Eligibility Determination; Event; HIV; Heart Function Tests; Hemorrhage; Hour; Image; Incidence; Infection; Kaposi Sarcoma; Laboratories; Lasers; Lesion; Light; Local Therapy; Malignant Neoplasms; Malignant neoplasm of prostate; Measures; Metastatic breast cancer; Monitor; Myelogenous; Normal tissue morphology; Operative Surgical Procedures; Outcome; PUVA Photochemotherapy; Pain; Participant; Patients; Pharmaceutical Preparations; Phase; Phase II/III Trial; Photosensitivity; Photosensitizing Agents; Phototherapy; Physicians; Population; Progression-Free Survivals; Pruritus; Psoriasis; Quality of life; Radiation therapy; Recovery; Recurrent tumor; Refractory; Relapse; Reporting; Research; Research Design; Research Personnel; Safety; Severities; Site; Source; Special Event; Specificity; Standardization; Sum; Symptoms; Systemic Therapy; Systemic disease; Testing; Therapeutic Effect; Therapy trial; Time; Tissues; Treatment Efficacy; Treatment-related toxicity; Ulcer; Visit; arm; associated symptom; design; dosage; efficacy evaluation; efficacy study; experimental study; follow-up; healing; improved; interest; light intensity; minimal risk; objective response rate; open label; phase III trial; response; safety study; side effect; standard of care; symptom treatment; treatment response; tumor

ABSTRACT Cutaneous metastatic breast cancer (CMBC) develops in up to 24% of patients with metastatic breast cancer. Systemic therapy often has limited efficacy in such cases, and surgery and radiotherapy offer only transient relief and their use may be limited in the CMBC population due to the side-effects involved and the extent of cutaneous lesions. CMBC can cause infection, bleeding, malodor and disfigurement and can progress to occupy large areas of the body. Kintara proposes a photodynamic therapy (PDT) known as REM-001 Therapy, wherein a photosensitive drug is injected into the body to collect at tumor sites, and is activated by a specially-designed laser focused on the selected lesions. The activated drug destroys tumors with minimal effects to surrounding tissue. REM-001 has been proven to be effective for many conditions, including CMBC. However, previous trials using a higher-than-threshold dose also reported high rates of unwanted side effects and longer healing time. In previous trials, symptoms such as eschar and ulceration increased recovery time and caused patients to leave studies but was not associated with better lesion outcomes. This project will reduce the treatment dosage to the previously identified threshold level – which demonstrated a 79% response rate in an initial study – in an effort to reduce side effects while still treating lesions. Kintara will test the reduced dose on 15 patients with symptomatic cutaneous lesions and stable systemic disease who are unable to receive surgery or radiotherapy at the time of treatment. These patients will be administered a single dose of REM-001 followed by light treatment and will be monitored for efficacy (lesion shrinking or disappearing, as well as pain and itch subsiding and improved quality of life) and safety/tolerability (eschar and ulceration, and bleeding and discharge from the lesions, treatment related photosensitivity, as well as standard clinical side-effect monitoring). Patients will continue on their existing standard of care systemic therapy and will receive it throughout the trial. If this trial is successful – the treated lesions shrink or disappear with the reduced treatment dose – Kintara will continue to a multi-site Phase III trial for CMBC and explore the use of REM-001 photodynamic therapy in other dermal indications.

抽象的 高达 24% 的转移性乳腺癌患者会发生皮肤转移性乳腺癌 (CMBC)。 在这种情况下，全身治疗通常疗效有限，手术和放疗只能提供短暂的缓解 由于所涉及的副作用和皮肤损伤的程度，它们在 CMBC 人群中的使用可能受到限制 CMBC 病变可引起感染、出血、恶臭和毁容，并可进展至大面积。 Kintara 提出了一种称为 REM-001 疗法的光动力疗法 (PDT)。 光敏药物被注入体内并聚集在肿瘤部位，并被特殊设计的药物激活 激光聚焦在选定的病灶上，激活的药物会破坏肿瘤，而对周围的影响最小。 REM-001 已被证明对许多疾病有效，包括 CMBC。 使用高于阈值的剂量还报告了不良副作用的高发生率和更长的愈合时间。 之前的试验中，焦痂和溃疡等症状会延长恢复时间并导致患者离开 研究但与更好的病变结果无关，该项目将减少治疗剂量。 先前确定的阈值水平（在一项初步研究中显示出 79% 的响应率） 为了在治疗病变的同时减少副作用，Kintara 将在 15 名患有此病的患者身上测试减少的剂量。 有症状的皮肤病变和稳定的全身性疾病而无法接受手术或放疗的 在治疗时，这些患者将接受单剂 REM-001，然后进行光治疗。 并将监测疗效（病变缩小或消失，以及疼痛和瘙痒消退和 改善生活质量）和安全性/耐受性（焦痂和溃疡、出血和排出物） 患者会 继续他们现有的护理标准全身治疗，并将在整个试验期间接受该治疗（如果该试验成功）。 成功 – 随着治疗剂量的减少，治疗的病灶缩小或消失 – Kintara 将继续 CMBC 多中心 III 期试验并探索 REM-001 光动力疗法在其他真皮中的应用 迹象。