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

Sponsor
Genenta Science
Study ID
NCT06716853
Phase
PHASE1/PHASE2
Status
Terminated

Conditions

  • Clear Cell RCC

Eligibility Criteria

Sex
ALL
Age
18 Years - 70 Years
Healthy Volunteers
Not accepted

Interventions

  • Temferon — GENETIC
    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.
  • Pembrolizumab — BIOLOGICAL
    Pembrolizumab 400mg IV every 6 weeks commencing at D+30
  • Cabozantinib — DRUG
    40mg QD once PD occurs as assessed at D+30 or at subsequent visits

Study Details

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.

Key Dates

Start date
Oct 22, 2024
Status verified
Feb 2026
Primary completion
Jan 23, 2026
Completion
Jan 23, 2026

Study Design

Enrollment
10 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Pembrolizumab cohort
    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.
  • Experimental: Cabozantinib cohort
    At D+30 after Temferon, in the event that a patient has received ICI in the 6 months prior to study entry and in case PD occurs (as assessed at D+30 or at subsequent visits), they will be receiving cabozantinib. Patients allocated to cabozantinib cohort will initiate treatment with 40mg QD

Primary Outcome Measure

Tolerability and safety of conditioning and Temferon, over the first 90 days following administration, as determined by the incidence of adverse events [ Time Frame: First 90 days ]

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