Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)
Condition: Diabetes Mellitus
MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 1
Intervention: CLBS03 Low Dose; CLBS03 High Dose; Placebo
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Factorial Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 113.0
Sex: All
Minimum age: 8 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: T1DM; Type 1 Diabetes Mellitus; T regulatory cell; Immunotherapy; Cell therapy; T-Reg

Summary:

      This clinical trial will explore the safety and effect of autologous ex vivo expanded
      polyclonal regulatory T-cells on beta cell function in patients, aged 8 to 17, with recent
      onset T1DM. Other measures of diabetes severity and the autoimmune response underlying T1DM
      will also be explored. Eligible subjects will receive a single infusion of CLBS03 (high or
      low dose) or placebo.
    

Eligibility criteria:

        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