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

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ClinicalTrials.gov Identifier: NCT02691247
Recruitment Status : Completed
First Posted : February 25, 2016
Results First Posted : January 8, 2021
Last Update Posted : January 8, 2021
Sponsor:
Collaborators:
Sanford Health
California Institute for Regenerative Medicine (CIRM)
Information provided by (Responsible Party):
Caladrius Biosciences, Inc.

Tracking Information
First Submitted Date  ICMJE February 12, 2016
First Posted Date  ICMJE February 25, 2016
Results First Submitted Date  ICMJE November 18, 2020
Results First Posted Date  ICMJE January 8, 2021
Last Update Posted Date January 8, 2021
Actual Study Start Date  ICMJE February 2016
Actual Primary Completion Date March 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 14, 2020)
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52 [ Time Frame: 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.
Original Primary Outcome Measures  ICMJE
 (submitted: February 20, 2016)
Mixed Meal Tolerance Test (MMTT)-stimulated C-peptide area under the curve (AUC) [ Time Frame: Week 52 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 14, 2020)
  • Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104 [ Time Frame: 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) [ Time Frame: Week 104 ]
  • Change From Baseline in Mean Daily Dose of Insulin [ Time Frame: Week 104 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: February 20, 2016)
  • MMTT-stimulated C-peptide AUC [ Time Frame: Week 104 ]
  • Change in Hemoglobin A1c (HbA1c) [ Time Frame: Week 104 ]
  • Change in insulin usage [ Time Frame: Week 104 ]
  • Proportion of subjects with adverse events [ Time Frame: Week 104 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)
Official Title  ICMJE 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)
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Factorial Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Diabetes Mellitus
Intervention  ICMJE
  • Biological: CLBS03 Low Dose
  • Biological: CLBS03 High Dose
  • Biological: Placebo
Study Arms  ICMJE
  • 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.
    Intervention: 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.
    Intervention: Biological: CLBS03 High Dose
  • Placebo Comparator: Placebo
    A single infusion of placebo, consisting of the infusion solution only
    Intervention: Biological: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 10, 2018)
113
Original Estimated Enrollment  ICMJE
 (submitted: February 20, 2016)
111
Actual Study Completion Date  ICMJE January 2020
Actual Primary Completion Date March 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02691247
Other Study ID Numbers  ICMJE CLBS03-P01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Caladrius Biosciences, Inc.
Study Sponsor  ICMJE Caladrius Biosciences, Inc.
Collaborators  ICMJE
  • Sanford Health
  • California Institute for Regenerative Medicine (CIRM)
Investigators  ICMJE
Study Director: Douglas Losordo, MD Caladrius Biosciences
PRS Account Caladrius Biosciences, Inc.
Verification Date December 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP