Phase 2
Completed N=110
Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)
Source: ClinicalTrials.gov NCT02691247 ↗Enrolled (actual)
110
Serious AEs
8.2%
Results posted
Jan 2021
Primary outcomePrimary: Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52 — -0.2863; -0.3895; -0.2670 pmol*hr/mL
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52 |
-0.2863; -0.3895; -0.2670 | — |
| SECONDARY Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104 |
-0.4492; -0.5224; -0.4412 | — |
| SECONDARY Change in Hemoglobin A1c (HbA1c) |
1.56; 1.05; 1.77 | — |
| SECONDARY Change From Baseline in Mean Daily Dose of Insulin |
0.3840; 0.4619; 0.2874 | — |
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
Data sourced from ClinicalTrials.gov (NCT02691247). 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.