Phase 2
Completed N=25
Safety, Tolerability and Potential Efficacy of AVT001 in Patients With Type 1 Diabetes
Source: ClinicalTrials.gov NCT03895996 ↗Enrolled (actual)
25
Serious AEs
0.0%
Results posted
Mar 2025
Primary outcomePrimary: Number of Participants With Treatment-emergent Adverse Events (TEAE) — 9; 6; 4; 1 participants
Summary
This is a double-blind, randomized , placebo-controlled study to evaluate the safety and tolerability of AVT001, and to assess AVT001 as a potential treatment for type 1 diabetes (T1D). The trial will involve approximately 24 new-onset T1D subjects.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAE) |
9; 6; 4; 1; 3; 1 | — |
| PRIMARY Number of Participants and Severity of Local i.v.-Site Reactions, |
0; 0 | — |
| PRIMARY Changes From Baseline of Creatinine |
1.9338; 0.4911 | — |
| PRIMARY Changes From Baseline of Aspartate Aminotransferase |
-1.9; 2.2 | — |
| PRIMARY Changes From Baseline of Alanine Aminotransferase |
-0.1; 1.1 | — |
| PRIMARY Changes From Baseline of Total Bilirubin |
-0.114; 0.000 | — |
| SECONDARY Assessment of the HLA-E-restricted CD8+ T Cell Regulatory Activity ("Potency Assay") |
-11.2; 3.5; 9.3; 1.8 | — |
| SECONDARY Changes From Baseline in the Area Under the Curve (AUC) of the Stimulated C-peptide Levels Over a 4-hour Mixed Meal Tolerance Test (MMTT) |
0.531; 0.611; 0.518; 0.472 | — |
| SECONDARY Changes From Baseline in HbA1c |
6.0375; 5.6556; 6.1500; 5.8222 | — |
Eligibility Criteria
Key Inclusion Criteria
- Diagnosis of type 1 diabetes, within 12 months of first dosing, confirmed by positive lab result for one or more of the following types of autoantibodies:
- Glutamic acid decarboxylase (GAD65)
- Insulinoma associated protein 2 (IA-2, also known as ICA-512)
- Zinc transporter 8 (ZnT8).
- Age 16 or older and able to provide informed consent/assent.
- If a participant is female with reproductive potential, willing to avoid pregnancy through the duration of the trial.
- Signed and dated written informed consent/assent.
Key Exclusion Criteria
- Poorly controlled diabetes despite insulin therapy, who in the opinion of the investigator would not be a good candidate for participation in a clinical trial
- Screening hemoglobin 300mg/gmCr
- Screening eGFR 1.5x upper limit of normal (ULN)
- Screening bilirubin > 2.0 mg / dL, or > 3.0 mg / dL for participants with Gilbert's Syndrome
- Current use of immunosuppressive or immunomodulatory therapies, including pharmacologic doses of systemic steroids. However, topical steroidal creams and inhaled steroids without large systemic absorption are allowed.
- Coincident medical condition likely to require immunosuppressive or immunomodulatory therapies.
- Coincident medical condition likely to limit short term (5 year) life expectancy (malignancy, symptomatic coronary artery disease, recent stroke)
- Prior radiation therapy, immunotherapy (within 1 year of screening), or chemotherapy
- Serologic evidence of current HIV-1 or HIV-2 infection
- Serologic evidence of hepatitis C infection
- Serologic evidence of acute or chronic active hepatitis B as measured by Core Ab positive and / or Surface Antibody antigen positive
- Subjects with other autoimmune conditions (except compensated or treated autoimmune thyroid, celiac, alopecia, or vitiligo diseases)
- Women who are pregnant (pregnancy testing during screening), breastfeeding, or planning pregnancy during the study period
- Inadequate venous access to support leukapheresis
- Any condition that in the opinion of the investigator(s) would preclude the subject from participating in a clinical trial.
- Abnormal screening ECG that in the opinion of the investigator or sponsor would pose a safety risk.
Data sourced from ClinicalTrials.gov (NCT03895996). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.