Gene therapy using Temferon for metastatic renal cell carcinoma treatment
An Open-label Phase 1/2 Study to Evaluate the Safety, Biological Response and Efficacy of a Single Dose of Temferon (autologous CD34+-enriched Hematopoietic Stem and Progenitors Cells Genetically Modified with Human Interferon-α2) in Patients with Metastatic Renal Cell Carcinoma
PHASE1; PHASE2 · Genenta Science · NCT06716853
This study is testing a new gene therapy called Temferon to see if it can help people with advanced kidney cancer who haven't responded to previous treatments.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Genenta Science (industry) |
| Drugs / interventions | pembrolizumab, cabozantinib |
| Locations | 1 site (Milan) |
| Trial ID | NCT06716853 on ClinicalTrials.gov |
What this trial studies
This clinical trial is an open-label, single-center study evaluating the safety and efficacy of Temferon, an investigational gene therapy, in patients with metastatic clear cell renal cell carcinoma who have experienced disease progression after at least two lines of standard treatments. The study is divided into two phases: Phase 1 involves administering Temferon to up to 12 patients, who will be categorized based on their prior immune checkpoint inhibitor therapy. Following a 30-day observation period, patients will receive either pembrolizumab or cabozantinib, depending on their treatment history. The goal is to assess the therapeutic potential of this novel approach in a challenging cancer population.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18-70 with histologically confirmed metastatic clear cell renal cell carcinoma who have progressed after at least two lines of standard care.
Not a fit: Patients who have not received prior standard treatments or those with other types of renal cell carcinoma may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced renal cell carcinoma who have limited treatment choices.
How similar studies have performed: While gene therapy approaches are emerging, this specific combination of Temferon with pembrolizumab or cabozantinib is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patient aged between 18 - 70 years old * Women of childbearing potential must have a negative pregnancy test at screening and agree to use two distinct acceptable methods of contraception (which may include partner contraception) for the duration of the study * Men with partners of childbearing potential must be willing to use acceptable barrier contraceptive method during the trial or have undergone a vasectomy at least 6 months prior to study entry and confirmed by semen analysis * Adequate cardiac, renal, hepatic, pulmonary, and hematologic function * Patient able and willing to provide written informed consent and comply with study protocol and procedures * Histologically confirmed diagnosis of unresectable, locally advanced/metastatic RCC with clear cell component, with or without sarcomatoid features * Presence of a disease burden sufficiently large to permit biopsy * Disease progression following approved standard of care treatments for metastatic disease * ECOG PS 0-1 * Measurable disease at physical examination or at imaging assessment according to RECIST 1.1 criteria Exclusion Criteria: * Use of investigational agents or procedures in the 4 weeks prior to study enrolment (6 weeks for long-acting agents) or receipt of an experimental gene therapy product in the past 2 years * History of current evidence of neuropsychiatric illness * History of severe cardiovascular disease * Evidence of haematological neoplasm * Active alcohol or substance abuse within 6 months of the study * Current pregnancy or lactation * Expected to undergo a surgical intervention during the first 3 months of the study * Known bleeding diathesis or history of abnormal/severe bleeding or any other known coagulation abnormalities that would contraindication a tissue biopsy, active treatment with anticoagulants. * New CNS or rapidly growing metastases or carcinomatous meningitis * Presence of hepatic metastases * Previous allogenic bone marrow, renal, liver transplant * Prior use of immunosuppressives in the previous 4 weeks prior to enrolment * Clinically relevant active viral, bacterial or fungal infection * Active autoimmune disease requiring disease modifying treatment.
Where this trial is running
Milan
- Ospedale San Raffaele — Milan, Italy (RECRUITING)
Study contacts
- Study coordinator: Carlo Russo, MD
- Email: info-trial@genenta.com
- Phone: +39 02 2643 3982
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Clear Cell RCC, metastatic renal cell carcinoma, Temferon, Gene, Solid tumor, Immunotherapy