Padeliporfin VTP for Bladder Cancer

(UCM301 Trial)

Recruiting in Palo Alto (17 mi)

+29 other locations

Overseen by Vitaly Margulis

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Steba Biotech S.A.

Must not be taking: VEGF-targeted therapy

Disqualifiers: High-grade carcinoma, Invasive T2, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

This is a phase 3, open label, single arm study of padeliporfin in the treatment of Upper Tract Urothelial Carcinoma (UTUC). The ENLIGHTED study will recruit patients with low-grade non-invasive upper tract urothelial carcinoma in either the kidney or the ureter. Patients will be treated with padeliporfin VTP in two phases: an Induction Treatment Phase and a Maintenance Treatment Phase and will be followed up for up to an additional 48 months in the long term (non intervention) follow up phase with the specific duration depending on the patient's response to treatment

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications, but it mentions that any prohibited medication should be adjusted or discontinued before starting the study treatment. It's best to discuss your current medications with the study team to see if any changes are needed.

What data supports the effectiveness of the treatment Padeliporfin VTP for bladder cancer?

Research shows that Padeliporfin VTP, a type of photodynamic therapy (a treatment using light-sensitive drugs and a light source to destroy cancer cells), has been effective in treating other urologic cancers by specifically targeting and destroying tumor cells. This suggests potential for its use in bladder cancer treatment. ^{1 2 3 4 5}

Is Padeliporfin VTP generally safe for humans?

A Phase I trial of Padeliporfin VTP (also known as WST-11 or TOOKAD Soluble) for upper tract urothelial carcinoma evaluated its safety and found it to be a promising treatment with therapeutic efficacy, suggesting it is generally safe for use in humans. ^{2 3 5 6 7}

How is the treatment Padeliporfin VTP unique for bladder cancer?

Padeliporfin VTP is unique because it uses a special light-activated process called photodynamic therapy (PDT) to target and destroy cancer cells in the bladder, making it a non-invasive option compared to traditional treatments. This therapy specifically targets blood vessels in the tumor, which helps to minimize damage to surrounding healthy tissue. ^{2 3 4 5 7}

Research Team

Vitaly Margulis

Principal Investigator

UT Southwestern Medical Center

Eligibility Criteria

The ENLIGHTED study is for adults with low-grade, non-invasive cancer in the kidney or ureter. Participants should have good physical function, specific tumor sizes and locations, normal blood counts and organ functions. Excluded are those with invasive cancers within 2 years, recent BCG/chemotherapy treatments, severe other diseases or heavy smoking habits, current high-grade bladder cancer or CIS in the upper tract.

Inclusion Criteria

My kidney function, measured by eGFR, is at least 30 ml/min.

Able to understand and provide written informed consent and willing to comply with all tests and procedures associated with the study

Total serum bilirubin <3 mg/dL

Exclusion Criteria

I have a skin condition that worsens with light exposure.

I am not willing to use birth control during and for 3 months after treatment.

I have or had early-stage cancer in my upper urinary tract.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction Treatment

Patients undergo 1-3 padeliporfin VTP treatments provided 4 weeks apart to achieve Complete Response (CR) in the involved ipsilateral tract system.

4-12 weeks

1-3 visits (in-person)

Maintenance Treatment

Patients achieving CR in the induction phase are followed for 12 months to assess response duration and safety, with repeated VTP treatments for treatable tumor recurrence.

12 months

Regular visits as needed for maintenance treatment

Long Term Follow-up

Patients are monitored for disease-related outcomes and VTP treatment-related adverse events for up to 48 months, with assessments at 18, 24 months, and annually thereafter.

48 months

Annual visits

Treatment Details

Interventions

• Padeliporfin VTP (Photosensitizer)

Trial Overview This phase 3 trial tests padeliporfin VTP on patients with Upper Tract Urothelial Carcinoma (UTUC). It involves an Induction Treatment Phase followed by a Maintenance Treatment Phase. Patients will be monitored up to four additional years post-treatment to assess long-term effects of this single-arm intervention.

Participant Groups

Group I: padeliporfin VTP Experimental Treatment 1 Intervention

Induction Treatment phase:1-3 padeliporfin VTP treatments provided 4 weeks (28 +/-3 days) apart. Maintenance Treatment Phase: Repeated maintenance VTP treatments during this period will be provided for patients who show evidence of tumor recurrence that is deemed treatable.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Department of Urology, Memorial Sloan Kettering Cancer Center New York, NY

Memorial Sloan Kettering Cancer Center New York, NY

Emory University Hospital Atlanta, GA

Medical University of South Carolina (MUSC) Charleston, SC

More Trial Locations

Who Is Running the Clinical Trial?

Steba Biotech S.A.

Lead Sponsor

Trials

17

Patients Recruited

1,300+

ICON plc

Industry Sponsor

Trials

88

Patients Recruited

28,900+

Medpace, Inc.

Industry Sponsor

Trials

98

Patients Recruited

30,400+

PrimeVigilance

Industry Sponsor

Trials

7

Patients Recruited

300+

Findings from Research

Hexaminolevulinate (HAL) based photodynamic therapy (PDT) is a safe and technically feasible adjuvant treatment for patients with intermediate or high-risk bladder cancer, with no technical complications reported during the procedure.

In a study of 17 patients, 52.9% were tumor-free at 6 months post-treatment, indicating potential efficacy, although the percentage of patients remaining tumor-free decreased over time.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic therapy of bladder cancer - a phase I study using hexaminolevulinate (HAL). Bader, MJ., Stepp, H., Beyer, W., et al.[2013]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Final Results of a Phase I Trial of WST-11 (TOOKAD Soluble) Vascular-targeted Photodynamic Therapy for Upper Tract Urothelial Carcinoma. Yip, W., Sjoberg, DD., Nogueira, LM., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Developments in Vascular-Targeted Photodynamic Therapy for Urologic Malignancies. Nogueira, L., Tracey, AT., Alvim, R., et al.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transferrin-conjugated liposome targeting of photosensitizer AlPcS4 to rat bladder carcinoma cells. Derycke, AS., Kamuhabwa, A., Gijsens, A., et al.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Combined chemotherapeutic and photodynamic treatment on human bladder cells by hematoporphyrin-platinum(II) conjugates. Lottner, C., Knuechel, R., Bernhardt, G., et al.[2019]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of 120W high performance system greenlight laser vaporization for non-muscle-invasive bladder cancer. Tao, W., Yang, D., Shan, Y., et al.[2013]

Intravesical administration of Cis-Diamminedichloroplatinum (DDP) in dogs showed minimal local toxicity and no significant systemic toxicity, making it a potentially safe option for treating superficial bladder cancers.

The study measured bladder tissue platinum concentrations, which were significantly elevated after DDP retention, indicating effective local delivery of the drug, but further research is needed to assess the long-term effects of this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Fundamental studies on intravesical instillation of cis-diamminedichloroplatinum for treatment of urinary bladder tumors. I: On the effects of intravesical instillation of cis-diamminedichloroplatinum in normal beagle dogs]. Hirao, Y., Iwai, A., Yoshie, T., et al.[2013]

References

1. United States pubmed.ncbi.nlm.nih.gov

Photodynamic therapy of bladder cancer - a phase I study using hexaminolevulinate (HAL). [2013]

2. United States pubmed.ncbi.nlm.nih.gov

Final Results of a Phase I Trial of WST-11 (TOOKAD Soluble) Vascular-targeted Photodynamic Therapy for Upper Tract Urothelial Carcinoma. [2023]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Developments in Vascular-Targeted Photodynamic Therapy for Urologic Malignancies. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Transferrin-conjugated liposome targeting of photosensitizer AlPcS4 to rat bladder carcinoma cells. [2022]

5. Ireland pubmed.ncbi.nlm.nih.gov

Combined chemotherapeutic and photodynamic treatment on human bladder cells by hematoporphyrin-platinum(II) conjugates. [2019]

6. Netherlands pubmed.ncbi.nlm.nih.gov

Safety and efficacy of 120W high performance system greenlight laser vaporization for non-muscle-invasive bladder cancer. [2013]

7. Japan pubmed.ncbi.nlm.nih.gov

[Fundamental studies on intravesical instillation of cis-diamminedichloroplatinum for treatment of urinary bladder tumors. I: On the effects of intravesical instillation of cis-diamminedichloroplatinum in normal beagle dogs]. [2013]