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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02691247
Recruitment Status : Completed
First Posted : February 25, 2016
Last Update Posted : July 22, 2020
Sanford Health
California Institute for Regenerative Medicine (CIRM)
Information provided by (Responsible Party):
Caladrius Biosciences, Inc.

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.

Condition or disease Intervention/treatment Phase
Diabetes Mellitus Biological: CLBS03 Low Dose Biological: CLBS03 High Dose Biological: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 113 participants
Allocation: Randomized
Intervention Model: Factorial Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : February 2016
Actual Primary Completion Date : March 2019
Actual Study Completion Date : January 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arm Intervention/treatment
Experimental: CLBS03 Low Dose
A 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.
Biological: CLBS03 Low Dose
Experimental: CLBS03 High Dose
A single infusion of CLBS03 High Dose, a cell product comprised of autologous, ex vivo expanded regulatory T-cells resuspended in sterile USP infusion solution.
Biological: CLBS03 High Dose
Placebo Comparator: Placebo
A single infusion of placebo, consisting of the infusion solution only
Biological: Placebo

Primary Outcome Measures :
  1. Mixed Meal Tolerance Test (MMTT)-stimulated C-peptide area under the curve (AUC) [ Time Frame: Week 52 ]

Secondary Outcome Measures :
  1. MMTT-stimulated C-peptide AUC [ Time Frame: Week 104 ]
  2. Change in hemoglobin A1c (HbA1c) [ Time Frame: Week 104 ]
  3. Change in insulin usage [ Time Frame: Week 104 ]
  4. Proportion of subjects with adverse events [ Time Frame: Week 104 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   8 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02691247

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United States, California
Rady Children's Hospital
San Diego, California, United States, 92123
University of California, San Francisco
San Francisco, California, United States, 94143
United States, Colorado
Barbara Davis Center for Diabetes
Aurora, Colorado, United States, 80045
United States, Connecticut
Yale University School of Medicine
New Haven, Connecticut, United States, 06519
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
University of Miami, Diabetes Research Institute
Miami, Florida, United States, 33136
United States, Indiana
Indiana University
Indianapolis, Indiana, United States, 46202
United States, Massachusetts
Joslin Diabetes Center
Boston, Massachusetts, United States, 02215
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Children's Mercy Kansas City
Kansas City, Missouri, United States, 64108
United States, North Dakota
Sanford Research
Fargo, North Dakota, United States, 58122
United States, Oregon
Oregon Health Science University
Portland, Oregon, United States, 97239
United States, South Dakota
Sanford Research
Sioux Falls, South Dakota, United States, 57104
United States, Tennessee
Vanderbilt Eskind Diabetes Clinic
Nashville, Tennessee, United States, 37232
United States, Texas
Baylor College of Medicine / Texas Children's Hospital
Houston, Texas, United States, 77030
Sponsors and Collaborators
Caladrius Biosciences, Inc.
Sanford Health
California Institute for Regenerative Medicine (CIRM)
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Study Director: Douglas Losordo, MD Caladrius Biosciences
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Responsible Party: Caladrius Biosciences, Inc. Identifier: NCT02691247    
Other Study ID Numbers: CLBS03-P01
First Posted: February 25, 2016    Key Record Dates
Last Update Posted: July 22, 2020
Last Verified: July 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Caladrius Biosciences, Inc.:
Type 1 Diabetes Mellitus
T regulatory cell
Cell therapy
Additional relevant MeSH terms:
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Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases