Phase1 Open-label, Non-controlled,Dose-escalation Clinical Trial of RM-0256 Photoimmunotherapy for the Treatment of Solid Tumors in Patients Who are Refractory to or Ineligible for Standard Treatment
Phase1 Open-label Clinical Trial of RM-0256 Photoimmunotherapy for the Treatment of Solid Tumors by Rakuten Medical, K.K.
Daisuke Okuda
Rakuten Medical, K.K.
Futakotamagawa Rise Office, 2-21-1 Tamagawa, Setagaya-ku, Tokyo
+81-3-4405-2187
daisuke.okuda@rakuten-med.com
Daisuke Okuda
Rakuten Medical, K.K.
Futakotamagawa Rise Office, 2-21-1 Tamagawa, Setagaya-ku, Tokyo
+81-3-4405-2187
daisuke.okuda@rakuten-med.com
Recruiting
May. 22, 2026
Aug. 17, 2026
24
Interventional
single arm study
open(masking not used)
uncontrolled control
single assignment
treatment purpose
1.Signed the informed consent form (ICF) to participate in this study by him/herself.
2.Male and female aged 18 years or above at the time of informed consent.
3.Have histologically confirmed solid tumor.
4.Refractory to standard treatment for advanced or recurrent solid tumors, or the Investigator/Sub-investigator considered that there are no standard or equivalent treatments available for the patient.
5.Have at least 1 lesion that can be illuminated with the laser system, and the tumor falls under one of the cancer types and sites which completed risk assessment of treatment with the investigational device in this study.
6. Completed PD-L1 testing (by IHC assay) and the result in PD-L1 expression (either PD-L1 positive or negative) is available. The results of previous PD-L1 testing (IHC assay) can be used if the sample was collected within 6 months prior to enrollment and provided that no systemic therapy or local therapy to the sample collection site has been administered during that period.
7.Can provide sufficient formalin-fixed, paraffin-embedded (FFPE) tumor tissue samples for PD-L1 IHC testing and other exploratory biomarker testing.
8.Have sufficient function of major organs (hematopoietic organ, liver, kidney, heart, and lung).
9.Eastern Cooperative Oncology Group Performance Status Scale (ECOG PS) is 0 or 1.
10.Expected to survive at least 3 months.
11.Can be hospitalized from D1 of the run-in phase in Part A until completion of observations, tests, and assessments specified on D8 of the run-in phase in Part A and from C1D1 in all Parts until completion of observations, tests, and assessments specified on C1D9 in all Parts.
12.Female patients must not be pregnant (i.e., must be tested negative for urine or serum pregnancy test at screening) or breastfeeding. They must agree to use 2 methods of highly effective birth control, or practice abstinence, as well as not to provide any eggs (ova and oocytes) for reproduction purposes throughout the study period and for at least 180 days after the final RM-0256 dose.
Women who are breastfeeding may be enrolled after stopping breastfeeding. However, they must wait at least 120 days to restart breastfeeding after treatment completion.
13.Male patients must be sterile or agree to use a highly effective method of contraception or practice abstinence, as well as not to provide sperm for reproductive purposes throughout the study period and for at least 120 days after the final RM-0256 dose.
Medical history
1. Malignant tumor other than the target disease within the past 2 years.
This criterion does not apply to diseases with a low recurrence risk, including adequately treated intraepithelial cancer, intramucosal cancer, low-grade non-melanoma skin cancer, focal prostate cancer, and ductal carcinoma in situ.
Other diseases with similar low recurrence risk or diseases which require no treatment may be deemed eligible based on discussions between the Investigator/Sub-investigator and the Medical Monitor.
2. Active metastasis in the central nervous system (CNS) or carcinomatous meningitis. However, patients with CNS metastasis that are considered clinically stable, and confirmed by imaging not to be progressing for more than 4 weeks, may be deemed eligible based on discussions between the Investigator/Sub-investigator and the Medical Monitor.
3. Pleural or ascites or pericardial effusion requiring drainage.
4. Current or past medical history of interstitial lung disease. However, localized pulmonary fibrosis occurring in the irradiated area is permitted.
5. Uncontrolled current medical history of cardiovascular disorders including the following but not limited to acute or chronic congestive heart failure, unstable angina pectoris, atrial fibrillation, other cardiac arrhythmias, myocardial infarction, coronary/peripheral artery bypass graft surgery, cardiomyopathies, cerebrovascular accident, transient ischemic attack, deep vein thrombosis, or symptomatic pulmonary embolism.
6. Current or past medical history of chronic or recurrent autoimmune disease requiring systemic treatment. However, if the disease is controlled with replacement therapy, the patient may be deemed eligible based on discussions between the Investigator/Sub-investigator and the Medical Monitor.
7. Current medical history of active infection requiring systemic treatment such as antibiotic(s) or antifungal/antiviral drug administration.
8. Active or latent hepatitis B (HBs antigen positive; or HBs antigen negative and HBs or HBc antibody positive; and HBV-DNA assay positive) and/or hepatitis C (HCV antibody positive and HCV-RNA assay positive).
9. Current or past medical history of testing positive for human immunodeficiency virus (HIV) infection or acquired immunodeficiency syndrome (AIDS) related diseases.
10. Known history of testing positive for qualitative HTLV-1 (human T-cell lymphotropic virus type 1).
11. Known history of Grade 3 or higher irAEs caused by immune checkpoint inhibitors, leading to treatment discontinuation.
12. Known to have Grade 3 or higher hypersensitivity to the components of RM-0256 (AB-256 [antiPD-L1 antibody], IR700 and excipients included in this drug).
13. The tumor to be illuminated is invading any areas where there is a risk of functional impairment or significant hemorrhage.
14. Current or past medical history of Grade 3 or higher tumor hemorrhage within the past 12 weeks. However, if the patient has received appropriate treatment such as transfusion or hemostatic therapy and currently shows no signs of active bleeding with stable general condition, the patient may be deemed eligible based on discussions between the Investigator/Sub-investigator and the Medical Monitor.
15. Prior allogeneic tissue/solid organ transplant.
Prior/Concomitant medications
16. Treated with anti-cancer treatment for a specific period, as described below. However, this criterion does not apply to zoledronic acid hydrate or denosumab initiated before participation in this study to treat bone metastasis.
a. Received chemotherapy (excluding immunotherapies such as anti-PD-1 antibody, antiPD-L1 antibody, and anti-CTLA-4 antibody) within 4 weeks prior to study treatment initiation.
b. Received immunotherapy (including antibodies such as anti-PD-1 antibody, anti-PD-L1 antibody, and anti-CTLA-4 antibody) within 4 weeks or 5 times the half-life of the drug prior to study treatment initiation (whichever is shorter).
c. Received radiation therapy (including palliative radiation therapy to relieve pain associated with bone metastasis) or other local therapy within 4 weeks prior to study treatment initiation.
d. Received surgery (i.e., radical operation) within 4 weeks prior to study treatment initiation.
17. Experienced toxicity in prior therapy and the toxicity has not improved to Grade 1 or below. However, the following toxicities are excluded from this criterion.
a. Grade 2 alopecia
b. Grade 2 peripheral nerve disorder
c. Autoimmune hypothyroidism controlled with a stable dose of thyroid hormone replacement therapy
d. Endocrine disorders controlled with systemic steroid therapy equivalent to 10mg/day or less of prednisolone as replacement therapy
18. Received long-term systemic steroid (exceeding 10 mg/day equivalent of systemic prednisolone) or other immunosuppressant administration within 14 days prior to study treatment initiation.
19. Received a live vaccine within 4 weeks prior to study treatment initiation.
Prior/Concomitant treatments
20. Underwent a major surgery or experienced a severe injury within 4 weeks prior to study treatment initiation; or expected to require a major surgery during the study treatment period.
21. Expected to have tests or treatments involving exposure to strong light (e.g., ophthalmology test, surgical treatment, or endoscopy) that are not related to RM-0256 PIT.
22. Require concomitant drug administration such as antiplatelet, anticoagulant, and thrombolytic drugs, which may increase risks of bleeding, during the study treatment period.
Others
23. Will not or cannot comply with the provisions defined in the protocol.
24. Female patients are considered as potentially pregnant based on medical interviews or examinations from the investigator or sub-investigator.
25. The Investigator/Sub-investigator deemed the patient inappropriate for study participation due to reasons not mentioned above.
18age old over
No limit
Both
Solid Tumors in Patients Who are Refractory to or Ineligible for Standard Treatment
The designated RM-0256 dose specified in the registered Dose Level (per Dose Level, 10 to 40 mg/kg) is infused one-time over 2 hours (window: +60 minutes). The designated laser light dose specified in the registered Dose Level is illuminated within 24 (window: plus or minus 4) hours after completion of RM-0256 infusion.
Incidence of DLTs during the protocol-defined assessment period.
Classification, incidence, and severity of AEs.
Classification, incidence, and severity of adverse device effects (ADEs) associated with the use of PIT690.4-1A3B Laser System, FD, SD, CD,and NC.
Best overall response.
Best percent change from baseline in the sum of diameters of target lesions (only for applicable patients).
Pharmacokinetic parameters (including CL, VSS, T1/2, and Ctrough at 3 weeks ).
