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Clear Cell RCCPhase 1/2TEM-GUNCT06716853

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

A Phase 1/2 study testing Temferon, Pembrolizumab and Cabozantinib in people with Clear Cell RCC, run by Genenta Science at 1 site in Italy.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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)… Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Clear Cell RCC
What is being tested?
Temferon, Pembrolizumab and Cabozantinib
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteItaly
Study dates
22 Oct 2024to 23 Jan 2026
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 10Show fewer
  • 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

Who cannot join

  • 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
Show all 14Show fewer
  • 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.

Trial locations and contacts

1 site in 1 country.

Italy1 site

  • Ospedale San Raffaele

    Milan, Italy, 20132

Other trials in Italy

About this study

Official title: 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

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.

Read the 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
Being tested
Temferon, Pembrolizumab, Cabozantinib
Keywords
metastatic renal cell carcinoma, Temferon, Gene, Solid tumor, Immunotherapy
Medical subject headings
Carcinoma, Renal Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TemferonGenetic

    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.

  • PembrolizumabBiological

    Pembrolizumab 400mg IV every 6 weeks commencing at D+30

  • CabozantinibDrug

    40mg QD once PD occurs as assessed at D+30 or at subsequent visits

Study groups in NCT06716853
Study groupRoleWhat they receive
Pembrolizumab cohortExperimentalAt 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.
Receives: Genetic: Temferon; Biological: Pembrolizumab
Cabozantinib cohortExperimentalAt 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
Receives: Genetic: Temferon; Drug: Cabozantinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Tolerability and safety of conditioning and Temferon, over the first 90 days following administration, as determined by the incidence of adverse events

    To assess tolerability and safety of conditioning and Temferon over the first 90 days following Temferon administration, as evaluated by the incidence of >=CTCAE Grade 2 adverse events.

    Measured: First 90 days

  2. Biological activity of Temferon, over the first 90 days following administration, as determined by the presence of Temferon-derived progeny in the tumor (IFN gene signature)

    To assess the biological activity of Temferon in the tumor of patients with metastatic renal cell carcinoma, over the first 90 days following Temferon administration, as determined by the change in IFN (interferon) gene signature in the tumor

    Measured: First 90 days

Secondary outcomes (5)

  1. Long term tolerability and safety of Temferon as determined by the incidence of adverse events up to 1 year following Temferon administration according to CTCAE v5.0 criteria

    Measured: 1 year following Temferon administration

  2. Incidence, severity and duration of adverse events of special interest as indicated on the study protocol

    Measured: Through study completion, an average of 1 year

  3. Proportion of patients achieving hematological recovery by Day +30

    Measured: Day 30

  4. Overall response rate per RECIST version 1.1

    Defined as the proportion of patients who achieved a complete or partial response as their best overall response

    Measured: RECIST criteria used at Screening, Baseline, Day +72, Day +120, Day +180, Day +210, Day +270, Day +360 or at any time disease progression or a secondary malignancy is suspected

  5. Disease control rate following Temferon infusion

    Measured: Through study completion, an average of 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Dec 2024
  2. Study start22 Oct 2024
  3. Primary completion23 Jan 2026
  4. Study completion23 Jan 2026

Why it stopped: Persistent challenges have been encountered in patient recruitment, difficulty in identifying a sufficient number of eligible subjects. The decision to terminate the study was not based on any safety concerns. Benefit-risk profile of IP unchanged.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
This is an open label, single-centre phase 1/2 therapeutic-exploratory prospective study where Temferon will be administered to up to 12 patients split into two cohorts according to immune checkpoint inhibitor -ICI- therapy received in the six months prior to entry into the study.
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06716853
Sponsor's protocol ID
TEM-GU
Other IDs
2024-512898-27-00
Acronym
TEM-GU
Status last verified
Feb 2026
First submitted
15 Nov 2024
Last updated
9 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Temferon, Pembrolizumab and Cabozantinib in people with Clear Cell RCC, run by Genenta Science at 1 site in Italy.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 9 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Persistent challenges have been encountered in patient recruitment, difficulty in identifying a sufficient number of eligible subjects. The decision to terminate the study was not based on any safety concerns. Benefit-risk profile of IP unchanged.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the 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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06716853.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2026.

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