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

Phase 2

Completed

Brief 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.

Recruitment & Eligibility

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

Primary Outcome Measures

Name	Time	Method
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52	Week 52	The area-under-curve of sequential C-peptide concentrations (AUC-Cpep) during the mixed-meal tolerance test (MMTT) is the gold-standard method to assess residual beta-cell (ie, insulin) secretion in type 1 diabetes.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Mean Daily Dose of Insulin	Week 104
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104	Week 104	The area-under-curve of sequential C-peptide concentrations (AUC-Cpep) during the mixed-meal tolerance test (MMTT) is the gold-standard method to assess residual beta-cell (ie, insulin) secretion in type 1 diabetes.
Change in Hemoglobin A1c (HbA1c)	Week 104

Locations (14): Rady Children's Hospital
🇺🇸
San Diego, California, United States
University of California, San Francisco
🇺🇸
San Francisco, California, United States
Barbara Davis Center for Diabetes
🇺🇸
Aurora, Colorado, United States
Yale University School of Medicine
🇺🇸
New Haven, Connecticut, United States
University of Florida
🇺🇸
Gainesville, Florida, United States
University of Miami, Diabetes Research Institute
🇺🇸
Miami, Florida, United States
Indiana University
🇺🇸
Indianapolis, Indiana, United States
Joslin Diabetes Center
🇺🇸
Boston, Massachusetts, United States
University of Minnesota
🇺🇸
Minneapolis, Minnesota, United States
Children's Mercy Kansas City
🇺🇸
Kansas City, Missouri, United States
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Rady Children's Hospital
🇺🇸San Diego, California, United States