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 皮肤转移性乳腺癌（CMBC）最多24％的转移性乳腺癌患者发展。 在这种情况下，全身治疗通常的效率通常有限，手术和放射疗法仅提供短暂的缓解 由于涉及的副作用和皮肤的程度，它们在CMBC人群中的使用可能受到限制 病变。 CMBC会引起感染，出血，疾病和毁容，并可以占据大区域 身体。 Kintara提案称为REM-001治疗的光动力疗法（PDT），其中a 光敏药物被注入体内，以在肿瘤部位收集，并被专门设计的 激光专注于选定的病变。激活的药物会破坏对周围的肿瘤 组织。 REM-001已被证明对包括CMBC在内的许多条件有效。但是，以前的试验 使用高于阈值较高的剂量还报告了不需要的副作用率高和更长的愈合时间。在 先前的试验，诸如Eschar和溃疡等症状增加了恢复时间，并导致患者离开 研究，但与更好的病变结局无关。该项目将将治疗剂量降低到 先前确定的阈值水平（在初始研究中证明了79％的响应率）。 减少副作用，同时仍在治疗病变。 Kintara将测试15例患者的减少剂量 有症状的皮肤病变和无法接受手术或放疗的稳定全身性疾病 在治疗时。这些患者将服用单剂量的rem-001，然后进行轻治疗 并将受到监测的效率（病变收缩或消失，疼痛和瘙痒缓解和 改善生活质量）和安全性/易学性（Eschar和溃疡，以及从 病变，相关的光敏性以及标准的临床副作用监测）。患者会 继续他们现有的护理系统治疗标准，并将在整个试验过程中接受。如果这个审判是 成功 - 治疗的病变随着治疗剂量减少而收缩或消失 - Kintara将继续进行 CMBC的多站点III期试验，并探索其他皮肤中Rem-001光动力疗法的使用 适应症。