NCT06716853

A Clinical Gene Therapy Study With Hematopoietic Stem Cells for the Treatment, With Single Dose of Temferon, of Patients Suffering From Metastatic Renal Cell Carcinoma

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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Genenta Science · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is an open label, single-centre phase 1/2 study involving a single dose of Temferon, an investigational Advanced Therapy Medicinal Product (ATMP), to treat patients with metastatic clear cell renal cell carcinoma (RCC) with evidence of disease progression following at least two lines of standard of care (SoC) treatments.

Detailed description

During Part A (Phase 1) of the study, Temferon will be administered to up to 12 patients, who are going to be split into two cohorts according to immune checkpoint inhibitor -ICI- therapy received in the six months prior to entry into the study, with a histologically confirmed diagnosis of metastatic RCC and evidence of disease progression following at least two lines of SoC treatments. At D+30 after Temferon, patients will start to receive pembrolizumab providing they have not received ICI in the 6 months prior to entry into the study. Patients allocated to pembrolizumab will receive pembrolizumab 400mg IV every 6 weeks commencing at D+30. In the event that a patient has received ICI in the 6 months prior to study entry, they will be allocated to the cabozantinib arm. Patients allocated to cabozantinib, will initiate treatment with 40mg QD once PD occurs as assessed at D+30 or at subsequent visits.

Conditions

• Clear Cell RCC

Interventions

Type	Name	Description
GENETIC	Temferon	Autologous CD34+-enriched hematopoietic progenitor cells exposed in vitro to specific lentiviral vector encoding for the human interferon-alpha 2 gene. Its expression is tightly controlled by the human TIE2 enhancer/promoter sequence and by a post-transcriptional regulation layer represented by target miRNA sequences. This enables suppression of interferon-alpha2 expression in HSPCs, thereby further increasing the specificity of the delivery strategy for their Tie2 expressing myeloid cell progeny.
BIOLOGICAL	Pembrolizumab	Pembrolizumab 400mg IV every 6 weeks commencing at D+30
DRUG	Cabozantinib	40mg QD once PD occurs as assessed at D+30 or at subsequent visits

Timeline

• Start date: 2024-10-22
• Primary completion: 2026-01-23
• Completion: 2026-01-23
• First posted: 2024-12-04
• Last updated: 2026-02-09

Locations

1 site across 1 country: Italy