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

Diabetes Mellitus Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02691247
• Other study ID #: CLBS03-P01
• Status: Completed
• Phase: Phase 2
• Start date: February 2016
• Est. completion date: January 2020

Study information

• Verified date: December 2020
• Source: Caladrius Biosciences, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 113
• Est. completion date: January 2020
• Est. primary completion date: March 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Diabetes Mellitus
• Diabetes Mellitus, Type 1

Intervention

Biological:
• CLBS03 Low Dose

• CLBS03 High Dose

• Placebo

Locations

Country	Name	City	State
United States	Barbara Davis Center for Diabetes	Aurora	Colorado
United States	Joslin Diabetes Center	Boston	Massachusetts
United States	Sanford Research	Fargo	North Dakota
United States	University of Florida	Gainesville	Florida
United States	Baylor College of Medicine / Texas Children's Hospital	Houston	Texas
United States	Indiana University	Indianapolis	Indiana
United States	Children's Mercy Kansas City	Kansas City	Missouri
United States	University of Miami, Diabetes Research Institute	Miami	Florida
United States	University of Minnesota	Minneapolis	Minnesota
United States	Vanderbilt Eskind Diabetes Clinic	Nashville	Tennessee
United States	Yale University School of Medicine	New Haven	Connecticut
United States	Oregon Health Science University	Portland	Oregon
United States	Rady Children's Hospital	San Diego	California
United States	University of California, San Francisco	San Francisco	California
United States	Sanford Research	Sioux Falls	South Dakota

Sponsors (3)

Lead Sponsor	Collaborator
Caladrius Biosciences, Inc.	California Institute for Regenerative Medicine (CIRM), Sanford Health

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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.	Week 104
Secondary	Change in Hemoglobin A1c (HbA1c)		Week 104
Secondary	Change From Baseline in Mean Daily Dose of Insulin		Week 104