Novel Autologous Dendritic Cell Therapy AVT001 for Type 1 Diabetes
Authors' conclusion
In this phase 1/2 trial, AVT001 did not result in dose-limiting adverse events. Potential signals of efficacy observed here warrant further evaluation in a fully powered trial.
Study context
- Population
- Participants aged ≥16 years, within 1 year of T1D diagnosis, with ex vivo evidence of a defect in Q/E CD8+ T regulatory (Treg) function
- Participants
- 16 vs 9
- Comparison
- Autologous dendritic cell therapy AVT001 vs placebo
- AVT001n = 16
- Placebon = 9reference
- Primary endpoint
- Safety
Methodological detailsphase 1/2, double-blind, placebo-controlled, randomized 2:1
- Phase 1/2
- Double-blind
- Placebo-controlled
- Randomized 2:1
Primary result
As reported in the abstract
Safety (adverse events) · through day 360
| Group | Value | Serious adverse events |
|---|---|---|
| AVT001 | 0 events | 0 |
AVT001
Value0 events
Serious adverse events0
Further findings
| Outcome | Estimate |
|---|---|
| Change from baseline in log-transformed C-peptide AUC (nmol/l), 4-hour mixed meal · day 150Less decline with AVT001 | treatment effect 0.09 (95% CI 0.03–0.15) |
| Change from baseline in log-transformed C-peptide AUC (nmol/l), 4-hour mixed meal · day 360Less decline with AVT001 | treatment effect 0.10 (95% CI 0.04–0.15) |
| Change from baseline in HbA1c · day 360 | estimated treatment effect −0.17 % (95% CI −0.60 to 0.26%) |
| Change from baseline in daily insulin dose · day 360 | estimated treatment effect −0.06 U/kg/day (95% CI −0.14 to 0.02) |
Change from baseline in hemoglobin A1c (HbA1c) · day 360
- No clear differences in change in HbA1c between groups
Change from baseline in daily insulin dose · day 360
- No clear differences in change in insulin dose between groups
Safety
Adverse events · through day 360
- Serious adverse events with AVT001
- 0
- Similar rates and severity of adverse events in both groups
- No dose-limiting adverse events with AVT001
Original publication
Novel Autologous Dendritic Cell Therapy AVT001 for Type 1 Diabetes. NEJM Evid. Published 25 June 2024. doi:10.
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