Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)

Inclusion Criteria:

- Male and females aged 8 to 17 years of age

- Diagnosis of T1DM within 100 days of receipt of study drug

- Positive for at least one islet cell autoantibody

- Peak MMTT-stimulated C-peptide level > 0.2 pmol/mL (at the screening visit)

- Weight of ≥30 kg

- Must agree to use a reliable and acceptable method of contraception for the duration
of participation

- Willing and medically acceptable to postpone live vaccine immunizations for one year
after infusion

- Written informed consent and written assent

Exclusion Criteria:

- Hemoglobin less than the lower limit of normal

- Leukocytes <3,000/μL; neutrophils <1,500/μL; lymphocytes <800/μL; platelets
<100,000/μL

- Regulatory T-cells present in peripheral blood at <20 cells per μL

- Current or ongoing use of non-insulin pharmaceuticals (that may affect glycemic
control)

- Current or anticipated use of systemic corticosteroids or other immunomodulatory drugs

- Recent serious bacterial, viral, fungal, or other opportunistic infections

- History of malignancy or serious uncontrolled cardiovascular, nervous system,
pulmonary, renal, or gastrointestinal disease

- Serologic evidence of current or past viral infection: human immunodeficiency virus
(HIV), Hepatitis B, Hepatitis C, and human T-lymphotropic virus (HTLV) 1/2

- Positive QuantiFERON® tuberculosis (TB) test, purified protein derivative (PPD) skin
test, history of tuberculosis, or active TB infection

- Active infection with Epstein-Barr Virus or Cytomegalovirus

- Liver disease

- Pregnant or breast-feeding

- Vaccination with a live virus within 8 weeks of receipt of study drug

- Vaccination with a killed virus within 2 weeks of receipt of study drug

- Participation in an investigational drug study within 90 days prior to screening

- Previously treated with a T-Reg based cell therapy

- History of allergy to gentamicin