MedDataX Logo

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

NCT ID: NCT06716853

Last Updated: 2024-12-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-22

Study Completion Date

2026-09-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Clear Cell RCC

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

metastatic renal cell carcinoma Temferon Gene Solid tumor Immunotherapy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

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.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

Temferon

Intervention Type 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

Intervention Type BIOLOGICAL

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

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

Group Type EXPERIMENTAL

Temferon

Intervention Type 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.

Cabozantinib

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type GENETIC

Pembrolizumab

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

Intervention Type BIOLOGICAL

Cabozantinib

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

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Alira Health

OTHER

Sponsor Role collaborator

Arithmos srl

UNKNOWN

Sponsor Role collaborator

Ospedale San Raffaele

OTHER

Sponsor Role collaborator

Genenta Science

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Ospedale San Raffaele

Milan, Italy, Italy

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Italy

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Carlo Russo, MD

Role: CONTACT

Phone: +39 02 2643 3982

Email: [email protected]

Zambanini Andrew, MD

Role: CONTACT

Phone: +39 02 2643 3982

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Necchi Andrea, MD

Role: primary

Andrea Necchi, MD

Role: backup

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2024-512898-27-00

Identifier Type: CTIS

Identifier Source: secondary_id

TEM-GU

Identifier Type: -

Identifier Source: org_study_id