Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)
A Prospective Randomized Placebo-Controlled Double Blind Clinical Trial to Evaluate the Safety and Efficacy of CLBS03 (Autologous Ex Vivo Expanded Polyclonal Regulatory T-cells) in Adolescents With Recent Onset Type 1 Diabetes Mellitus (T1DM)
1 other identifier
interventional
113
1 country
15
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2 diabetes-mellitus
Started Feb 2016
Typical duration for phase_2 diabetes-mellitus
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2016
CompletedFirst Submitted
Initial submission to the registry
February 12, 2016
CompletedFirst Posted
Study publicly available on registry
February 25, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
January 1, 2020
CompletedResults Posted
Study results publicly available
January 8, 2021
CompletedJanuary 8, 2021
December 1, 2020
3.1 years
February 12, 2016
November 18, 2020
December 14, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at 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.
Week 52
Secondary Outcomes (3)
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104
Week 104
Change in Hemoglobin A1c (HbA1c)
Week 104
Change From Baseline in Mean Daily Dose of Insulin
Week 104
Study Arms (3)
CLBS03 Low Dose
EXPERIMENTALA single infusion of CLBS03 Low Dose, a cell product comprised of autologous, ex vivo expanded regulatory T-cells resuspended in sterile USP (United States Pharmacopoeia) infusion solution.
CLBS03 High Dose
EXPERIMENTALA single infusion of CLBS03 High Dose, a cell product comprised of autologous, ex vivo expanded regulatory T-cells resuspended in sterile USP infusion solution.
Placebo
PLACEBO COMPARATORA single infusion of placebo, consisting of the infusion solution only
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
- +2 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Lisata Therapeutics, Inc.lead
- Sanford Healthcollaborator
- California Institute for Regenerative Medicine (CIRM)collaborator
Study Sites (15)
Rady Children's Hospital
San Diego, California, 92123, United States
University of California, San Francisco
San Francisco, California, 94143, United States
Barbara Davis Center for Diabetes
Aurora, Colorado, 80045, United States
Yale University School of Medicine
New Haven, Connecticut, 06519, United States
University of Florida
Gainesville, Florida, 32610, United States
University of Miami, Diabetes Research Institute
Miami, Florida, 33136, United States
Indiana University
Indianapolis, Indiana, 46202, United States
Joslin Diabetes Center
Boston, Massachusetts, 02215, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Children's Mercy Kansas City
Kansas City, Missouri, 64108, United States
Sanford Research
Fargo, North Dakota, 58122, United States
Oregon Health Science University
Portland, Oregon, 97239, United States
Sanford Research
Sioux Falls, South Dakota, 57104, United States
Vanderbilt Eskind Diabetes Clinic
Nashville, Tennessee, 37232, United States
Baylor College of Medicine / Texas Children's Hospital
Houston, Texas, 77030, United States
Related Publications (1)
Bender C, Wiedeman AE, Hu A, Ylescupidez A, Sietsema WK, Herold KC, Griffin KJ, Gitelman SE, Long SA; T-Rex Study Groupdagger; T-Rex Clinical Study Group. A phase 2 randomized trial with autologous polyclonal expanded regulatory T cells in children with new-onset type 1 diabetes. Sci Transl Med. 2024 May 8;16(746):eadn2404. doi: 10.1126/scitranslmed.adn2404. Epub 2024 May 8.
PMID: 38718135DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- William Sietsema
- Organization
- Caladrius Biosciences
Study Officials
- STUDY DIRECTOR
Douglas Losordo, MD
Caladrius Biosciences
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- FACTORIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 12, 2016
First Posted
February 25, 2016
Study Start
February 1, 2016
Primary Completion
March 1, 2019
Study Completion
January 1, 2020
Last Updated
January 8, 2021
Results First Posted
January 8, 2021
Record last verified: 2020-12
Data Sharing
- IPD Sharing
- Will not share