Safety, Tolerability and Potential Efficacy of AVT001 in Patients With Type 1 Diabetes

Prior treatment

• 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.
• Prior radiation therapy, immunotherapy (within 1 year of screening), or chemotherapy

Other illness

• 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
• 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)
• 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)
• Any condition that in the opinion of the investigator(s) would preclude the subject from participating in a clinical trial.

Lab values

• Screening hemoglobin <10.0 g/dL; leukocytes <3,000/uL; neutrophils <1,500/uL; lymphocytes <800/uL; platelets <100,000/uL
• Screening Urine Albumin Excretion > 300mg/gmCr
• Screening eGFR < 60 mL/min/1.73m2
• Screening ALT or AST > 1.5x upper limit of normal (ULN)
• Screening bilirubin > 2.0 mg / dL, or > 3.0 mg / dL for participants with Gilbert's Syndrome

Other

• Women who are pregnant (pregnancy testing during screening), breastfeeding, or planning pregnancy during the study period
• Inadequate venous access to support leukapheresis
• Abnormal screening ECG that in the opinion of the investigator or sponsor would pose a safety risk.

• 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.