Authorised Phase I and Phase II (Integrated)- First administration to humans Type 1 diabetes

A first-in-human clinical trial using a gene therapy with patient’s own stem cells to treat early type 1 diabetes

EU CTIS ID: 2025-521304-21-00

What this study is testing

The primary objective of the clinical trial is to evaluate study treatment safety.

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

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

1. Capable of giving signed informed consent as described in Section 13.2, which includes compliance with the requirements and restrictions listed in the Informed Consent Form and this protocol.
2. Male or female patients.
3. Age ≥18 and ≤40 years (≤25 years at Padova University Hospital due to feasibility constraints of the phase I clinical centre).
4. Patient able to comply with all protocol procedures for the duration of the study.
5. Recent T1D onset/diagnosis (patients should receive the DP within 180 days from the 1st insulin administration).
6. HbA1c ≥53 and ≤150 mmol/mol.

You likely can't join if

1. Unwillingness to sign the informed consent.
3. Any other unstable chronic disease.
4. Significant systemic infection during the four weeks before requiring hospitalisation, administration of intravenous antibiotics, surgery.
5. Present administration of anti-neoplastic drugs.
6. QTcF >470 msec.
7. Occurrence of an episode of ketoacidosis or hypoglycaemic coma in the past two weeks.

See the full eligibility criteria

Who can join

1. Capable of giving signed informed consent as described in Section 13.2, which includes compliance with the requirements and restrictions listed in the Informed Consent Form and this protocol.
2. Male or female patients.
3. Age ≥18 and ≤40 years (≤25 years at Padova University Hospital due to feasibility constraints of the phase I clinical centre).
4. Patient able to comply with all protocol procedures for the duration of the study.
5. Recent T1D onset/diagnosis (patients should receive the DP within 180 days from the 1st insulin administration).
6. HbA1c ≥53 and ≤150 mmol/mol.
7. Positivity to at least 2 autoantibodies (i.e., anti-insulin, IAA; anti-glutamic acid decarboxylase 65, GAD65; anti-islet antigen 2, IA-2A; anti-zinc transporter 8, ZnT8; anti-islet cell antibody, ICA).
8. Basal C-peptide levels ≥0.2 nmol/L or ≥0.6 ng/mL; if basal C-peptide levels <0.2 nmol/L, stimulated C-peptide peak ≥0.2 nmol/L or ≥0.6 ng/mL during a 2-hour MMTT; MMTT should not be performed within one week of resolution of a diabetic ketoacidosis event.

What rules you out

1. Unwillingness to sign the informed consent.
3. Any other unstable chronic disease.
4. Significant systemic infection during the four weeks before requiring hospitalisation, administration of intravenous antibiotics, surgery.
5. Present administration of anti-neoplastic drugs.
6. QTcF >470 msec.
7. Occurrence of an episode of ketoacidosis or hypoglycaemic coma in the past two weeks.
8. Presence of a ≥grade 3 adverse event (including laboratory analyses) according to CTCAE version 5.0.
9. Evidence of clinically significant abnormalities at bone-marrow aspirate.
12. Active CMV or EBV infection, defined as EBV DNA or CMV DNA >1,000 copies/mL.
10. Body Mass Index (body weight*height2)>27 kg⁄m2.
11. A positive result to Biological Screening testing for Anti-HCV Antibody (Ab), HCV nucleic acid test (NAT) (if anti-HCV Ab positive), HIV-1/-2 p24 Ab and antigen (Ag), HIV RNA NAT, anti-Treponema pallidum total Ig, HbsAg (Australia Ag), HBV DNA NAT, total anti-HB core Ab (if HBV DNA NAT positive), anti-HTLV I, and anti-HTLV II (if applicable).
13. Active SARS-CoV-2 infection.
14. Allergy to mobilizing agents (G-CSF and plerixafor).
15. Pregnancy or lactation.
16. Absence of an efficacious method of contraception.
17. Any condition that in the opinion of investigator contraindicates apheresis or infusion of transduced HSPCs or affects patient’s compliance.
2. Type 2 diabetes.

The study team makes the final eligibility decision.

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 18-64 years. The study team makes the final eligibility decision.

Explore other conditions

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.