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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.
 
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.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Factorial Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Diabetes Mellitus
Interventions Biological: CLBS03 Low Dose
Biological: CLBS03 High Dose
Biological: Placebo
Enrollment 113
Recruitment Details  
Pre-assignment Details  
Arm/Group Title CLBS03 Low Dose CLBS03 High Dose Placebo
Hide Arm/Group 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.

CLBS03 Low 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.

CLBS03 High Dose

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

Placebo
Period Title: Overall Study
Started 40 24 46
Completed 37 22 43
Not Completed 3 2 3
Reason Not Completed
Withdrawal by Subject             1             2             2
Lost to Follow-up             2             0             1
Arm/Group Title CLBS03 Low Dose CLBS03 High Dose Placebo Total
Hide Arm/Group 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.

CLBS03 Low 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.

CLBS03 High Dose

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

Placebo

 Total of all reporting groups
Overall Number of Baseline Participants 40 24 46 110
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants 24 participants 46 participants 110 participants
<=18 years
40
 100.0%
24
 100.0%
46
 100.0%
110
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants 24 participants 46 participants 110 participants
Female
10
  25.0%
5
  20.8%
13
  28.3%
28
  25.5%
Male
30
  75.0%
19
  79.2%
33
  71.7%
82
  74.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants 24 participants 46 participants 110 participants
Hispanic or Latino
5
  12.5%
2
   8.3%
2
   4.3%
9
   8.2%
Not Hispanic or Latino
34
  85.0%
22
  91.7%
44
  95.7%
100
  90.9%
Unknown or Not Reported
1
   2.5%
0
   0.0%
0
   0.0%
1
   0.9%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants 24 participants 46 participants 110 participants
American Indian or Alaska Native
1
   2.5%
0
   0.0%
0
   0.0%
1
   0.9%
Asian
1
   2.5%
0
   0.0%
0
   0.0%
1
   0.9%
Native Hawaiian or Other Pacific Islander
1
   2.5%
1
   4.2%
3
   6.5%
5
   4.5%
Black or African American
1
   2.5%
0
   0.0%
1
   2.2%
2
   1.8%
White
36
  90.0%
23
  95.8%
42
  91.3%
101
  91.8%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 40 participants 24 participants 46 participants 110 participants
40 24 46 110
1.Primary Outcome
Title Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title CLBS03 Low Dose CLBS03 High Dose Placebo
Hide Arm/Group 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.

CLBS03 Low 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.

CLBS03 High Dose

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

Placebo
Overall Number of Participants Analyzed 40 24 46
Mean (Standard Deviation)
Unit of Measure: pmol*hr/mL
-0.2863  (0.25988) -0.3895  (0.31425) -0.2670  (0.25745)
2.Secondary Outcome
Title Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title CLBS03 Low Dose CLBS03 High Dose Placebo
Hide Arm/Group 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.

CLBS03 Low 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.

CLBS03 High Dose

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

Placebo
Overall Number of Participants Analyzed 40 24 46
Mean (Standard Deviation)
Unit of Measure: pmol*hr/mL
-0.4492  (0.33135) -0.5224  (0.33903) -0.4412  (0.28923)
3.Secondary Outcome
Title Change in Hemoglobin A1c (HbA1c)
Hide Description [Not Specified]
Time Frame Week 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title CLBS03 Low Dose CLBS03 High Dose Placebo
Hide Arm/Group 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.

CLBS03 Low 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.

CLBS03 High Dose

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

Placebo
Overall Number of Participants Analyzed 39 24 46
Mean (Standard Deviation)
Unit of Measure: HbA1c %
1.56  (1.660) 1.05  (1.474) 1.77  (2.162)
4.Secondary Outcome
Title Change From Baseline in Mean Daily Dose of Insulin
Hide Description [Not Specified]
Time Frame Week 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title CLBS03 Low Dose CLBS03 High Dose Placebo
Hide Arm/Group 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.

CLBS03 Low 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.

CLBS03 High Dose

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

Placebo
Overall Number of Participants Analyzed 39 24 46
Mean (Standard Deviation)
Unit of Measure: U/kg body weight
0.3840  (0.39727) 0.4619  (0.40220) 0.2874  (0.33602)
Time Frame 2 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title CLBS03 Low Dose CLBS03 High Dose Placebo
Hide Arm/Group 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.

CLBS03 Low 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.

CLBS03 High Dose

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

Placebo
All-Cause Mortality
CLBS03 Low Dose CLBS03 High Dose Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/40 (0.00%)      0/24 (0.00%)      0/46 (0.00%)    
Hide Serious Adverse Events
CLBS03 Low Dose CLBS03 High Dose Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/40 (10.00%)      2/24 (8.33%)      3/46 (6.52%)    
Gastrointestinal disorders       
Gastritis †  1/40 (2.50%)  1 0/24 (0.00%)  0 0/46 (0.00%)  0
Infections and infestations       
Gastroenteritis viral †  1/40 (2.50%)  1 0/24 (0.00%)  0 0/46 (0.00%)  0
Groin abscess †  0/40 (0.00%)  0 1/24 (4.17%)  1 0/46 (0.00%)  0
Metabolism and nutrition disorders       
Diabetic ketoacidosis †  3/40 (7.50%)  3 0/24 (0.00%)  0 2/46 (4.35%)  5
Hyperglycaemia †  1/40 (2.50%)  1 0/24 (0.00%)  0 0/46 (0.00%)  0
Hypoglycaemia †  0/40 (0.00%)  0 1/24 (4.17%)  1 0/46 (0.00%)  0
Psychiatric disorders       
Major depression †  0/40 (0.00%)  0 0/24 (0.00%)  0 1/46 (2.17%)  1
Suicidal ideation †  0/40 (0.00%)  0 1/24 (4.17%)  1 1/46 (2.17%)  1
Suicide attempt †  0/40 (0.00%)  0 1/24 (4.17%)  1 0/46 (0.00%)  0
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
CLBS03 Low Dose CLBS03 High Dose Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   38/40 (95.00%)      24/24 (100.00%)      46/46 (100.00%)    
Blood and lymphatic system disorders       
Neutropenia †  0/40 (0.00%)  0/24 (0.00%)  4/46 (8.70%) 
Gastrointestinal disorders       
Abdominal pain upper †  5/40 (12.50%)  3/24 (12.50%)  1/46 (2.17%) 
Diarrhoea †  3/40 (7.50%)  3/24 (12.50%)  2/46 (4.35%) 
Nausea †  4/40 (10.00%)  1/24 (4.17%)  2/46 (4.35%) 
Vomiting †  5/40 (12.50%)  2/24 (8.33%)  10/46 (21.74%) 
General disorders       
Influence like illness †  2/40 (5.00%)  2/24 (8.33%)  2/46 (4.35%) 
Pyrexia †  3/40 (7.50%)  2/24 (8.33%)  3/46 (6.52%) 
Immune system disorders       
Seasonal allergy †  3/40 (7.50%)  0/24 (0.00%)  2/46 (4.35%) 
Infections and infestations       
Gastroenteritis †  1/40 (2.50%)  0/24 (0.00%)  3/46 (6.52%) 
Gastroenteritis viral †  2/40 (5.00%)  1/24 (4.17%)  5/46 (10.87%) 
Influenza †  4/40 (10.00%)  3/24 (12.50%)  4/46 (8.70%) 
Nasopharyngitis †  7/40 (17.50%)  8/24 (33.33%)  7/46 (15.22%) 
Pharyngitis streptococcal †  4/40 (10.00%)  5/24 (20.83%)  2/46 (4.35%) 
Upper respiratory tract infection †  12/40 (30.00%)  13/24 (54.17%)  18/46 (39.13%) 
Viral infection †  3/40 (7.50%)  1/24 (4.17%)  3/46 (6.52%) 
Injury, poisoning and procedural complications       
Concussion †  2/40 (5.00%)  1/24 (4.17%)  3/46 (6.52%) 
Limb injury †  3/40 (7.50%)  0/24 (0.00%)  0/46 (0.00%) 
Metabolism and nutrition disorders       
Diabetic ketoacidosis †  3/40 (7.50%)  0/24 (0.00%)  2/46 (4.35%) 
Hypoglycaemia †  3/40 (7.50%)  1/24 (4.17%)  4/46 (8.70%) 
Musculoskeletal and connective tissue disorders       
Arthralgia †  2/40 (5.00%)  0/24 (0.00%)  3/46 (6.52%) 
Back pain †  1/40 (2.50%)  2/24 (8.33%)  0/46 (0.00%) 
Pain in extremity †  3/40 (7.50%)  0/24 (0.00%)  3/46 (6.52%) 
Nervous system disorders       
Headache †  8/40 (20.00%)  5/24 (20.83%)  11/46 (23.91%) 
Migraine †  0/40 (0.00%)  2/24 (8.33%)  1/46 (2.17%) 
Presyncope †  0/40 (0.00%)  2/24 (8.33%)  1/46 (2.17%) 
Somnolence †  3/40 (7.50%)  0/24 (0.00%)  0/46 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Cough †  5/40 (12.50%)  4/24 (16.67%)  6/46 (13.04%) 
Epistaxis †  3/40 (7.50%)  0/24 (0.00%)  0/46 (0.00%) 
Nasal congestion †  5/40 (12.50%)  4/24 (16.67%)  4/46 (8.70%) 
Sinus congestion †  1/40 (2.50%)  2/24 (8.33%)  0/46 (0.00%) 
Skin and subcutaneous tissue disorders       
Lipohypertrophy †  2/40 (5.00%)  2/24 (8.33%)  4/46 (8.70%) 
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: William Sietsema
Organization: Caladrius Biosciences
Phone: 9495352391
EMail: bsietsema@caladrius.com
Layout table for additonal information
Responsible Party: Caladrius Biosciences, Inc.
ClinicalTrials.gov Identifier: NCT02691247    
Other Study ID Numbers: CLBS03-P01
First Submitted: February 12, 2016
First Posted: February 25, 2016
Results First Submitted: November 18, 2020
Results First Posted: January 8, 2021
Last Update Posted: January 8, 2021