AVT001: randomized Phase 1/2 trial of spHsp60-pulsed autologous dendritic cells in recent-onset T1D
What this study tests
Double-blind, randomized (2:1), placebo-controlled Phase 1/2 study at Joslin Diabetes Center evaluating three monthly intravenous infusions of autologous spHsp60-pulsed dendritic cells (AVT001, n = 16) versus placebo (n = 9) in 25 newly diagnosed T1D patients aged 16 and older. Published in NEJM Evidence (2024), showing 0 SAEs, restored HLA-E CD8+ Treg potency, and less C-peptide decline at Day 150.
Editorial review: .
Registry checked: 2026-10-26. Registry’s own update: 2025-03-05.
Most recent recorded citation date: 2025-03-05. Only explicit date metadata is included; an undated citation may be newer. This does not mean every claim was reviewed on that date.
Trial status, labels and access can change between reviews. How we review the evidence · How to read the evidence
Evidence at a glance
- Who can enter the study?
- Patients aged 16 and older diagnosed with Type 1 Diabetes within the preceding 12 months with residual endogenous C-peptide. Registered eligibility ↗Enrollment criteria describe who may enter. They do not establish who was analyzed or the denominator for a reported result.
- Reported benefit and results
- Met all primary safety endpoints (0 SAEs, 0 local IV-site reactions). On secondary efficacy measures posted to ClinicalTrials.gov and published in NEJM Evidence (Gaglia et al. 2024, PMID 38916421), HLA-E-restricted CD8+ Treg inhibitory potency improved from -11.2% at baseline to +9.3% at Day 150 on AVT001 (vs 3.5% to 1.8% on placebo), and 4-hour MMTT C-peptide AUC declined by only 2.4% (0.531 to 0.518 nmol/L) on AVT001 versus 22.7% (0.611 to 0.472 nmol/L) on placebo at Day 150 (estimated log C-peptide AUC treatment difference +0.09, 95% CI 0.03 to 0.15).Read the result sources and their limitations →
- Important harms
- Read the reported results, safety discussion and original sources. A separate harms summary is not available; this does not establish safety.
- Research access and approval
- Study regions: United States. A trial listing does not establish product approval or current recruitment at a particular site. Check the study’s current entry requirements.
- What remains uncertain?
- Read the full discussion and original sources for study limitations. Planned endpoints and completion dates are not results.
Research status alone does not establish approval, clinical benefit or local availability.
Primary endpoints
- Incidence of treatment-emergent adverse events, local IV-site reactions, and hepatic/renal laboratory changes through Day 150
The full picture
Study design and results
Randomized (2:1), double-blind, placebo-controlled Phase 1/2 trial at Joslin Diabetes Center evaluating three monthly intravenous infusions of autologous spHsp60-pulsed immature dendritic cells (AVT001, n = 16) versus matched placebo (n = 9) in 25 adolescents and adults aged 16 and older within 1 year of Stage 3 Type 1 Diabetes diagnosis. Published in NEJM Evidence (Gaglia et al. 2024, PMID: 38916421), demonstrating clean safety (0 SAEs), restoration of HLA-E-restricted CD8+ Treg inhibitory potency (+9.3% at Day 150 vs -11.2% at baseline), and less decline in 4-hour MMTT C-peptide AUC at Day 150 (+0.09 log AUC treatment difference, 95% CI 0.03 to 0.15).
Sources
- [1]NCT03895996: A Double-Blind, Randomized, Placebo-Controlled Study of AVT001 in Patients With Type 1 Diabetes · Trial registry · 2025-03-05
- [2]Novel Autologous Dendritic Cell Therapy AVT001 for Type 1 Diabetes · Peer-reviewed study · 2024-07 (month only) — Gaglia JL et al., NEJM Evidence 2024;3(7):EVIDoa2300238 (PMID 38916421; DOI 10.1056/EVIDoa2300238).