Clinical trial NCT02691247: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Diabetes Mellitus
Keywords: T1DM
Type 1 Diabetes Mellitus
T regulatory cell
Immunotherapy
Cell therapy
T-Reg
Lead sponsor: Lisata Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Sanford Health
name: California Institute for Regenerative Medicine (CIRM)
First posted: 2016-02-25
Enrollment: 113
Sex: ALL
Minimum age (years): 8
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: FACTORIAL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52
timeFrame: Week 52
Secondary outcomes:
measure: Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104
timeFrame: Week 104
measure: Change in Hemoglobin A1c (HbA1c)
timeFrame: Week 104
measure: Change From Baseline in Mean Daily Dose of Insulin
timeFrame: Week 104
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Aurora
country: United States
facility: Barbara Davis Center for Diabetes
state: Colorado
city: New Haven
country: United States
facility: Yale University School of Medicine
state: Connecticut
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Miami
country: United States
facility: University of Miami, Diabetes Research Institute
state: Florida
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Boston
country: United States
facility: Joslin Diabetes Center
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Kansas City
country: United States
facility: Children's Mercy Kansas City
state: Missouri
city: Fargo
country: United States
facility: Sanford Research
state: North Dakota
city: Portland
country: United States
facility: Oregon Health Science University
state: Oregon
city: Sioux Falls
country: United States
facility: Sanford Research
state: South Dakota
city: Nashville
country: United States
facility: Vanderbilt Eskind Diabetes Clinic
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine / Texas Children's Hospital
state: Texas
Study officials:
affiliation: Caladrius Biosciences
name: Douglas Losordo, MD
role: STUDY_DIRECTOR
References: