Treatment of skin cancer using REM-001 photodynamic therapy

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) That is Refractory or Not Eligible for Radiotherapy or Surgery

This study is testing a new light-based treatment called REM-001 for people with skin cancer from breast cancer to see if it helps shrink tumors and improve their quality of life.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Kintara Therapeutics, Inc. (Industry-sponsored)
• Drugs / interventions: trastuzumab, sacituzumab, pembrolizumab, chemotherapy, radiation
• Locations: 2 sites (Bronx, New York and 1 other locations)
• Trial ID: NCT05374915 on ClinicalTrials.gov

What this trial studies

This open-label, single cohort study aims to evaluate the efficacy and safety of REM-001 photodynamic therapy for patients with cutaneous metastatic breast cancer. Participants will receive an intravenous infusion of REM-001 followed by light treatment to targeted lesions. The study will assess tumor response through photographic imaging and patient-reported outcomes over a 24-week period. The primary goal is to confirm the treatment's effectiveness and support future studies.

Who should consider this trial

Good fit: Ideal candidates are adults with histopathologically confirmed cutaneous breast cancer metastasis that is symptomatic and not suitable for surgical resection.

Not a fit: Patients with non-symptomatic lesions or those who have not progressed on systemic therapy may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for patients with cutaneous metastatic breast cancer who are not candidates for surgery.

How similar studies have performed: Other studies have shown promise with photodynamic therapy for various cancers, suggesting potential success for this approach in treating cutaneous metastatic breast cancer.

Eligibility criteria

Inclusion Criteria:

* Adult participants 18 years of age or greater.
* Participants able and willing to sign informed consent.
* Histopathologically confirmed breast cancer metastasis to the skin.
* Cutaneous metastasis not suitable for surgical resection.
* Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.
* Symptomatic lesions (including discomfort, pain, discharge, ulceration).
* Cutaneous, subcutaneous soft tissue, or superficial lymphatic metastasis that is amenable to PDT:

  * Lesion(s) \> 10 mm and \< 60 mm in longest dimension.
  * Lesion(s) exhibit at least one of the following symptoms: ulcerated, bleeding, discharging, itchy, painful.
  * Judged by investigator as eligible for PDT.
* Participants are radiotherapy refractory (have received a radiation dose of 60 gray (Gy) or greater to the ipsilateral thorax) or are not otherwise amenable to radiotherapy.
* Disease progression on at least 2 courses of systemic therapy:

  * HR positive/HER2 negative participants: should be refractory to endocrine therapy (at least 2 different regimens including at least one CDK4/6 inhibitor). Maintenance endocrine therapy at the clinician's discretion is allowed.
  * HER2 positive participants should have had disease progression on at least 2 different regimens of HER2 targeted therapies. Maintenance therapy on trastuzumab (HERCEPTIN®) is allowed.
* If participants are on systemic therapy at enrollment, they must meet the following:

  * Participants must have undergone a minimum of two cycles of systemic therapy prior to enrollment.
  * The systemic therapy must be one from the Treatment of Physician's Choice (TPC) list, as follows: : eribulin mesylate (Halaven®); capecitabine (Xeloda®); Gemcitabine (Gemzar®); a taxane \[either docetaxel (Taxotere®), nab-paclitaxel (Abraxane®), or paclitaxel (Taxol®)\]; vinorelbine (Navelbine®); an antibody-drug conjugate \[either sacituzumab-govitecan (Trodelvy®), trastuzumab-deruxtecan (Enhertu®), or ado-trastuzumab emtansine (KADCYLA®)\]; pembrolizumab (Keytruda®); or carboplatin (Paraplatin®).
  * Patients who are not on any chemotherapy will also be eligible.
* At the time of enrolment, participants should have no known plans to change or modify their TPC regimen while receiving study treatment. Note: TPC regimen may be changed or modified after treatment with REM-001 therapy, but this should be done in consultation with the Sponsor Medical Monitor.
* Adequate renal function, as evidenced by estimated glomerular filtration rate (eGFR) \> 45 mL/min/1.73 m2 using the CKD-EPI Creatinine Equation without race.
* Adequate bone marrow function as evidenced by absolute neutrophil count (ANC) ≥ 1.5 × 10\^9/L, hemoglobin ≥ 8.5 g/dL and platelet count ≥ 100 × 10\^9/L; INR \< 1.5.
* Adequate liver function as evidenced by bilirubin ≤ 2.0 times the upper limits of normal (ULN) and alkaline phosphatase, alanine aminotransferase (ALT), and aspartate aminotransferase (AST) ≤ 3 × ULN (in the case of liver metastases ≤ 5 × ULN) \[Participants with known Gilbert's Syndrome who have serum bilirubin \< 1.5 x ULN (NCI CTCAE v5.0 Gr 2) may be enrolled\].
* QTCF \< 470msec on baseline ECG
* Woman of childbearing potential (WOCBP) must have a negative serum pregnancy test documented within 7 days prior to registration and must agree to practice adequate contraception
* Male patients must be sterile or willing to use an approved method of contraception from the time of treatment with REM-001 until 90 days after study drug treatment.

Exclusion Criteria:

* Participants who have received local cryotherapy, radiotherapy, intra-lesional chemotherapy, systemic or topical PDT, or surgery to study lesion fields within the past 12 weeks.
* Participants with progressive brain or subdural metastases, or leptomeningeal disease.
* Participants with previously treated brain or subdural metastases may participate provided:

  * Previously treated brain metastases are stable and without evidence of progression, as determined by contrast-enhanced CT or MRI brain scan, for at least 4 weeks prior to the first dose of study treatment.
  * There is no evidence of new brain metastases
  * They have completed local therapy and discontinued the use of corticosteroids for this indication for at least 4 weeks prior to first dose of study treatment.
  * Any neurologic symptoms attributed to brain metastases must have been stable for at least 4 weeks prior to study enrollment
* History of allergic or hypersensitivity reactions to light, egg proteins or egg yolk; history of porphyria, systemic lupus erythematosus, or xeroderma pigmentosum.
* Known disorder of lipoprotein metabolism or clearance (cholesterol\> 400 mg/dl, and/or triglycerides \> 500 mg/dl).
* Participants who have received investigational agents within the past 4 weeks or within 4 half-lives of the investigational agent (whichever is shorter) before the first study drug dose.
* Participants with inflammatory breast cancer.
* Known human immunodeficiency virus (HIV) infection with detectable virus titer.
* Active or chronic hepatitis B or C infection.
* Active or ongoing infection requiring systemic treatment.
* Participants who have undergone major surgery within 4 weeks of study treatment, or have planned surgery within 4 weeks of anticipated initiation of treatment with REM-001 therapy.
* Participants with otherwise unexplained weight loss (\> 10% body weight) in the last 30 days prior to Screening.
* History of other malignancy treated with curative intent within the last 3-5 years. Exceptions are: Curatively treated basal cell/squamous cell skin cancer; carcinoma in situ of the cervix; superficial transitional cell bladder carcinoma
* Patients with other major or uncontrolled medical conditions, e.g., myocardial infarction or New York Heart Association (NYHA) Class III/IV heart failure within the last 6 months, stroke, uncontrolled diabetes, uncontrolled autoimmune disease.
* WOCBP that is pregnant or breast-feeding, or planning to become pregnant or breast-feed during protocol treatment and for 3 months after last dose of REM-001 therapy.
* WOCBP unwilling to use effective contraception during protocol treatment and for 3 months after last dose of REM-001 therapy.

Where this trial is running

Bronx, New York and 1 other locations

• Montefiore Einstein Center for Cancer Care — Bronx, New York, United States (Recruiting)
• Memorial Sloan Kettering Cancer Center — New York, New York, United States (Recruiting)

Study contacts

• Principal investigator: Alina Markova, MD — Memorial Sloan Kettering Cancer Center
• Study coordinator: Manahil Sadiq, MHA

Conditions Cutaneous Breast Cancer; metastatic; cutaneous