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

Primary Purpose

Diabetes Mellitus

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
CLBS03 Low Dose
CLBS03 High Dose
Placebo
Sponsored by
Lisata Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Diabetes Mellitus focused on measuring T1DM, Type 1 Diabetes Mellitus, T regulatory cell, Immunotherapy, Cell therapy, T-Reg

Eligibility Criteria

8 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Rady Children's Hospital
  • University of California, San Francisco
  • Barbara Davis Center for Diabetes
  • Yale University School of Medicine
  • University of Florida
  • University of Miami, Diabetes Research Institute
  • Indiana University
  • Joslin Diabetes Center
  • University of Minnesota
  • Children's Mercy Kansas City
  • Sanford Research
  • Oregon Health Science University
  • Sanford Research
  • Vanderbilt Eskind Diabetes Clinic
  • Baylor College of Medicine / Texas Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

CLBS03 Low Dose

CLBS03 High Dose

Placebo

Arm Description

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.

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.

A single infusion of placebo, consisting of the infusion solution only

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at 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)
Change From Baseline in Mean Daily Dose of Insulin

Full Information

First Posted
February 12, 2016
Last Updated
December 14, 2020
Sponsor
Lisata Therapeutics, Inc.
Collaborators
Sanford Health, California Institute for Regenerative Medicine (CIRM)
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1. Study Identification

Unique Protocol Identification Number
NCT02691247
Brief Title
Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
February 2016 (Actual)
Primary Completion Date
March 2019 (Actual)
Study Completion Date
January 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Lisata Therapeutics, Inc.
Collaborators
Sanford Health, California Institute for Regenerative Medicine (CIRM)

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diabetes Mellitus
Keywords
T1DM, Type 1 Diabetes Mellitus, T regulatory cell, Immunotherapy, Cell therapy, T-Reg

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Factorial Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
113 (Actual)

8. Arms, Groups, and Interventions

Arm Title
CLBS03 Low Dose
Arm Type
Experimental
Arm Description
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.
Arm Title
CLBS03 High Dose
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
A single infusion of placebo, consisting of the infusion solution only
Intervention Type
Biological
Intervention Name(s)
CLBS03 Low Dose
Intervention Type
Biological
Intervention Name(s)
CLBS03 High Dose
Intervention Type
Biological
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52
Description
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.
Time Frame
Week 52
Secondary Outcome Measure Information:
Title
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104
Description
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.
Time Frame
Week 104
Title
Change in Hemoglobin A1c (HbA1c)
Time Frame
Week 104
Title
Change From Baseline in Mean Daily Dose of Insulin
Time Frame
Week 104

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Douglas Losordo, MD
Organizational Affiliation
Caladrius Biosciences
Official's Role
Study Director
Facility Information:
Facility Name
Rady Children's Hospital
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
University of California, San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Barbara Davis Center for Diabetes
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Yale University School of Medicine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06519
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
University of Miami, Diabetes Research Institute
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Indiana University
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Joslin Diabetes Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Children's Mercy Kansas City
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Sanford Research
City
Fargo
State/Province
North Dakota
ZIP/Postal Code
58122
Country
United States
Facility Name
Oregon Health Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Sanford Research
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57104
Country
United States
Facility Name
Vanderbilt Eskind Diabetes Clinic
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Baylor College of Medicine / Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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