Study of IMMUNOSTEM gene therapy using modified stem cells for newly diagnosed patients with type 1 diabetes who still produce insulin

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What is this study about?

This clinical trial focuses on treating Type 1 diabetes using a new gene therapy approach. The treatment involves using the patient’s own hematopoietic stem and progenitor cells (blood-forming stem cells) that are modified in a laboratory. These cells are altered using a special carrier called a lentiviral vector to introduce a gene that produces a protein known as human programmed death-ligand 1. This treatment is called IMMUNOSTEM.

The main purpose of this study is to evaluate if this new treatment is safe for patients who have been recently diagnosed with Type 1 diabetes and still have some functioning insulin-producing cells. The treatment is given as a single intravenous injection of the modified cells back into the patient’s body.

During the study, patients will be monitored closely for any side effects and changes in their diabetes condition. The monitoring includes regular blood tests, checking blood sugar levels, and measuring how well the body is producing insulin. The study will track how the modified cells perform in the body and observe any changes in the patient’s need for insulin treatment over time.

1 Initial evaluation

Your eligibility for the study will be assessed based on specific criteria, including:

– Age between 18 and 40 years

– Recent diagnosis of type 1 diabetes (within 180 days of first insulin use)

– Presence of at least 2 diabetes-related antibodies in your blood

– Specific blood sugar control measurements

2 Cell collection procedure

A procedure will be performed to collect stem cells from your blood

These cells will be modified in a laboratory to include a special protein called PD-L1

The modified cells will be prepared as a suspension for injection

3 Treatment administration

The modified cells will be given back to you through an intravenous injection

This is a one-time treatment administration

4 Follow-up period

Regular monitoring will occur over 24 months to assess:

– Safety through vital signs and laboratory tests

– Blood sugar control through continuous monitoring

– Insulin requirements

– Blood tests to measure the presence of modified cells

The total duration of the study is from August 2025 to August 2029

5 Testing procedures

Throughout the study period, you will undergo:

– Regular blood tests

– Mixed meal tolerance tests to measure your body’s insulin production

– Continuous blood sugar monitoring

– Regular health assessments

Who Can Join the Study?

You must be able to understand and sign an informed consent form
You can be either male or female
Your age must be between 18 and 40 years old
You must be able to follow all study procedures throughout the entire duration of the study
You must have been recently diagnosed with Type 1 diabetes (within 180 days of starting insulin treatment)
Your HbA1c (blood test that measures average blood sugar levels over the past 2-3 months) must be between 53 and 150 mmol/mol
You must test positive for at least 2 autoantibodies (proteins in your blood that indicate your immune system is attacking insulin-producing cells)
Your C-peptide (a substance released with insulin that shows how much insulin your body produces) must be either:
- At least 0.2 nmol/L or 0.6 ng/mL when measured at rest, or
- At least 0.2 nmol/L or 0.6 ng/mL during a special meal test (if your resting level is lower)

Who Cannot Join the Study?

Persons who have not been diagnosed with Type 1 diabetes
Children under 18 years of age
Pregnant or breastfeeding women
Persons with severe allergic reactions in the past
Participation in other clinical trials within the last 30 days
Persons with serious medical conditions that could interfere with the study (such as severe heart, liver, or kidney problems)
People unable to provide informed consent
Persons with a history of non-compliance with medical treatments
Those with unstable mental health conditions
People with conditions that might affect their ability to follow study procedures
Individuals taking medications that could interact with the study treatment
Those with uncontrolled blood sugar levels (extremely high or low glucose readings)
People with severe complications from diabetes
Those who cannot commit to regular study visits and follow-up appointments

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Azienda Ospedaliera di Padova	Padua	Italy

Trial status

Country	Status	Recruitment Start
Italy	Recruiting	01.12.2025

Trial locations

Autologous hematopoietic stem and progenitor cells (HSPCs) are cells taken from the patient’s own body, specifically from their bone marrow or blood. In this trial, these cells are modified in a laboratory using a special genetic technique. This is not a traditional medication, but rather a cell therapy using the patient’s own modified cells.

Lentiviral vector (LVV) is a tool used to modify the patient’s cells. It’s designed to deliver genetic material that helps the cells produce a protein called PD-L1, which plays a role in controlling immune responses. This is not taken as a medication but is used in the laboratory to prepare the cell therapy.

These modified cells are intended to help treat type 1 diabetes by potentially protecting the remaining insulin-producing cells in the pancreas from being destroyed by the immune system.

Type 1 diabetes – A chronic autoimmune condition where the body’s immune system attacks and destroys insulin-producing beta cells in the pancreas. This results in the body being unable to produce sufficient insulin, which is essential for controlling blood sugar levels. The condition typically develops during childhood or adolescence, but can occur at any age. People with Type 1 diabetes experience high blood sugar levels, and their bodies cannot properly convert glucose from food into energy. The disease progresses as more beta cells are destroyed over time, leading to complete or near-complete dependency on external insulin. Common symptoms include increased thirst, frequent urination, extreme hunger, unintended weight loss, and fatigue.

Trial ID:

2025-521304-21-00

Protocol code:

Immunostem

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study of IMMUNOSTEM gene therapy using modified stem cells for newly diagnosed patients with type 1 diabetes who still produce insulin

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?