Status:
NOT_YET_RECRUITING
A First-in-human Clinical Trial Using a Gene Therapy With Patient's Own Stem Cells to Treat Early Type 1 Diabetes
Lead Sponsor:
Altheia Science
Collaborating Sponsors:
Evidenze Health S.r.l.
Conditions:
Diabetes, Type I
Eligibility:
All Genders
18-40 years
Phase:
PHASE1
PHASE2
Brief Summary
Purpose: The purpose of the trial is to assess the safety profile of the study treatment and to evaluate its efficacy in terms of improvement in key diabetes management parameters, including insulin ...
Eligibility Criteria
Inclusion
- Capable of giving signed informed consent, compliance with the requirements and restrictions listed in the Informed Consent Form and the protocol.
- Male and or female patients.
- Age ≥18 and ≤40 years
- Patient able to comply with all protocol procedures for the duration of the study.
- Recent T1D onset/diagnosis (patients should receive the DP within 180 days from the 1st insulin administration).
- HbA1c ≥53 and ≤150 mmol/mol
- 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).
- 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.
Exclusion
- Unwillingness to sign the informed consent.
- Type 2 diabetes
- Any other unstable chronic disease
- Significant systemic infection during the four weeks before requiring hospitalisation, administration of intravenous antibiotics, surgery
- Present administration of chemotherapeutic anti-neoplastic drugs.
- QTcF \>470 msec.
- Occurrence of an episode of ketoacidosis or hypoglycaemic coma in the past two weeks.
- Presence of a ≥grade 3 adverse event (including laboratory analyses) according to CTCAE version 5.0.
- Evidence of clinically significant abnormalities at bone-marrow aspirate
- Body Mass Index (body weight\*height2 )\>27 kg⁄m2
- 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).
- Active SARS-CoV-2 infection.
- Allergy to mobilizing agents (G-CSF and plerixafor).
- Pregnancy or lactation
- Absence of an efficacious method of contraception
- Any condition that in the opinion of investigator contraindicate apheresis or infusion of transduced HSPCs or affects patient's compliance.
Key Trial Info
Start Date :
August 15 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 15 2029
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT06938334
Start Date
August 15 2025
End Date
June 15 2029
Last Update
April 22 2025
Active Locations (1)
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1
Azienda Ospedale-Università Padova
Padua, Italia, Italy