Trial Outcomes & Findings for Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study) (NCT NCT02691247)
NCT ID: NCT02691247
Last Updated: 2021-01-08
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
113 participants
Primary outcome timeframe
Week 52
Results posted on
2021-01-08
Participant Flow
Participant milestones
| Measure |
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.
CLBS03 Low Dose
|
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.
CLBS03 High Dose
|
Placebo
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
|---|---|---|---|
|
Overall Study
STARTED
|
40
|
24
|
46
|
|
Overall Study
COMPLETED
|
37
|
22
|
43
|
|
Overall Study
NOT COMPLETED
|
3
|
2
|
3
|
Reasons for withdrawal
| Measure |
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.
CLBS03 Low Dose
|
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.
CLBS03 High Dose
|
Placebo
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
2
|
2
|
|
Overall Study
Lost to Follow-up
|
2
|
0
|
1
|
Baseline Characteristics
Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)
Baseline characteristics by cohort
| Measure |
CLBS03 Low Dose
n=40 Participants
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
|
CLBS03 High Dose
n=24 Participants
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
|
Placebo
n=46 Participants
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
Total
n=110 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
40 Participants
n=39 Participants
|
24 Participants
n=41 Participants
|
46 Participants
n=35 Participants
|
110 Participants
n=31 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=39 Participants
|
5 Participants
n=41 Participants
|
13 Participants
n=35 Participants
|
28 Participants
n=31 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=39 Participants
|
19 Participants
n=41 Participants
|
33 Participants
n=35 Participants
|
82 Participants
n=31 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
9 Participants
n=31 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
34 Participants
n=39 Participants
|
22 Participants
n=41 Participants
|
44 Participants
n=35 Participants
|
100 Participants
n=31 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
3 Participants
n=35 Participants
|
5 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
2 Participants
n=31 Participants
|
|
Race (NIH/OMB)
White
|
36 Participants
n=39 Participants
|
23 Participants
n=41 Participants
|
42 Participants
n=35 Participants
|
101 Participants
n=31 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Region of Enrollment
United States
|
40 participants
n=39 Participants
|
24 participants
n=41 Participants
|
46 participants
n=35 Participants
|
110 participants
n=31 Participants
|
PRIMARY outcome
Timeframe: Week 52The 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.
Outcome measures
| Measure |
CLBS03 Low Dose
n=40 Participants
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
|
CLBS03 High Dose
n=24 Participants
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
|
Placebo
n=46 Participants
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
|---|---|---|---|
|
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52
|
-0.2863 pmol*hr/mL
Standard Deviation 0.25988
|
-0.3895 pmol*hr/mL
Standard Deviation 0.31425
|
-0.2670 pmol*hr/mL
Standard Deviation 0.25745
|
SECONDARY outcome
Timeframe: Week 104The 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.
Outcome measures
| Measure |
CLBS03 Low Dose
n=40 Participants
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
|
CLBS03 High Dose
n=24 Participants
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
|
Placebo
n=46 Participants
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
|---|---|---|---|
|
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104
|
-0.4492 pmol*hr/mL
Standard Deviation 0.33135
|
-0.5224 pmol*hr/mL
Standard Deviation 0.33903
|
-0.4412 pmol*hr/mL
Standard Deviation 0.28923
|
SECONDARY outcome
Timeframe: Week 104Outcome measures
| Measure |
CLBS03 Low Dose
n=39 Participants
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
|
CLBS03 High Dose
n=24 Participants
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
|
Placebo
n=46 Participants
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
|---|---|---|---|
|
Change in Hemoglobin A1c (HbA1c)
|
1.56 HbA1c %
Standard Deviation 1.660
|
1.05 HbA1c %
Standard Deviation 1.474
|
1.77 HbA1c %
Standard Deviation 2.162
|
SECONDARY outcome
Timeframe: Week 104Outcome measures
| Measure |
CLBS03 Low Dose
n=39 Participants
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
|
CLBS03 High Dose
n=24 Participants
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
|
Placebo
n=46 Participants
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
|---|---|---|---|
|
Change From Baseline in Mean Daily Dose of Insulin
|
0.3840 U/kg body weight
Standard Deviation 0.39727
|
0.4619 U/kg body weight
Standard Deviation 0.40220
|
0.2874 U/kg body weight
Standard Deviation 0.33602
|
Adverse Events
CLBS03 Low Dose
Serious events: 4 serious events
Other events: 38 other events
Deaths: 0 deaths
CLBS03 High Dose
Serious events: 2 serious events
Other events: 24 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 46 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CLBS03 Low Dose
n=40 participants at risk
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
|
CLBS03 High Dose
n=24 participants at risk
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
|
Placebo
n=46 participants at risk
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
|---|---|---|---|
|
Gastrointestinal disorders
Gastritis
|
2.5%
1/40 • Number of events 1 • 2 years
|
0.00%
0/24 • 2 years
|
0.00%
0/46 • 2 years
|
|
Infections and infestations
Gastroenteritis viral
|
2.5%
1/40 • Number of events 1 • 2 years
|
0.00%
0/24 • 2 years
|
0.00%
0/46 • 2 years
|
|
Infections and infestations
Groin abscess
|
0.00%
0/40 • 2 years
|
4.2%
1/24 • Number of events 1 • 2 years
|
0.00%
0/46 • 2 years
|
|
Metabolism and nutrition disorders
Diabetic ketoacidosis
|
7.5%
3/40 • Number of events 3 • 2 years
|
0.00%
0/24 • 2 years
|
4.3%
2/46 • Number of events 5 • 2 years
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
2.5%
1/40 • Number of events 1 • 2 years
|
0.00%
0/24 • 2 years
|
0.00%
0/46 • 2 years
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/40 • 2 years
|
4.2%
1/24 • Number of events 1 • 2 years
|
0.00%
0/46 • 2 years
|
|
Psychiatric disorders
Major depression
|
0.00%
0/40 • 2 years
|
0.00%
0/24 • 2 years
|
2.2%
1/46 • Number of events 1 • 2 years
|
|
Psychiatric disorders
Suicidal ideation
|
0.00%
0/40 • 2 years
|
4.2%
1/24 • Number of events 1 • 2 years
|
2.2%
1/46 • Number of events 1 • 2 years
|
|
Psychiatric disorders
Suicide attempt
|
0.00%
0/40 • 2 years
|
4.2%
1/24 • Number of events 1 • 2 years
|
0.00%
0/46 • 2 years
|
Other adverse events
| Measure |
CLBS03 Low Dose
n=40 participants at risk
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
|
CLBS03 High Dose
n=24 participants at risk
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
|
Placebo
n=46 participants at risk
A single infusion of placebo, consisting of the infusion solution only
Placebo
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/40 • 2 years
|
0.00%
0/24 • 2 years
|
8.7%
4/46 • 2 years
|
|
Gastrointestinal disorders
Abdominal pain upper
|
12.5%
5/40 • 2 years
|
12.5%
3/24 • 2 years
|
2.2%
1/46 • 2 years
|
|
Gastrointestinal disorders
Diarrhoea
|
7.5%
3/40 • 2 years
|
12.5%
3/24 • 2 years
|
4.3%
2/46 • 2 years
|
|
Gastrointestinal disorders
Nausea
|
10.0%
4/40 • 2 years
|
4.2%
1/24 • 2 years
|
4.3%
2/46 • 2 years
|
|
Gastrointestinal disorders
Vomiting
|
12.5%
5/40 • 2 years
|
8.3%
2/24 • 2 years
|
21.7%
10/46 • 2 years
|
|
General disorders
Influence like illness
|
5.0%
2/40 • 2 years
|
8.3%
2/24 • 2 years
|
4.3%
2/46 • 2 years
|
|
General disorders
Pyrexia
|
7.5%
3/40 • 2 years
|
8.3%
2/24 • 2 years
|
6.5%
3/46 • 2 years
|
|
Immune system disorders
Seasonal allergy
|
7.5%
3/40 • 2 years
|
0.00%
0/24 • 2 years
|
4.3%
2/46 • 2 years
|
|
Infections and infestations
Gastroenteritis
|
2.5%
1/40 • 2 years
|
0.00%
0/24 • 2 years
|
6.5%
3/46 • 2 years
|
|
Infections and infestations
Gastroenteritis viral
|
5.0%
2/40 • 2 years
|
4.2%
1/24 • 2 years
|
10.9%
5/46 • 2 years
|
|
Infections and infestations
Influenza
|
10.0%
4/40 • 2 years
|
12.5%
3/24 • 2 years
|
8.7%
4/46 • 2 years
|
|
Infections and infestations
Nasopharyngitis
|
17.5%
7/40 • 2 years
|
33.3%
8/24 • 2 years
|
15.2%
7/46 • 2 years
|
|
Infections and infestations
Pharyngitis streptococcal
|
10.0%
4/40 • 2 years
|
20.8%
5/24 • 2 years
|
4.3%
2/46 • 2 years
|
|
Infections and infestations
Upper respiratory tract infection
|
30.0%
12/40 • 2 years
|
54.2%
13/24 • 2 years
|
39.1%
18/46 • 2 years
|
|
Infections and infestations
Viral infection
|
7.5%
3/40 • 2 years
|
4.2%
1/24 • 2 years
|
6.5%
3/46 • 2 years
|
|
Injury, poisoning and procedural complications
Concussion
|
5.0%
2/40 • 2 years
|
4.2%
1/24 • 2 years
|
6.5%
3/46 • 2 years
|
|
Injury, poisoning and procedural complications
Limb injury
|
7.5%
3/40 • 2 years
|
0.00%
0/24 • 2 years
|
0.00%
0/46 • 2 years
|
|
Metabolism and nutrition disorders
Diabetic ketoacidosis
|
7.5%
3/40 • 2 years
|
0.00%
0/24 • 2 years
|
4.3%
2/46 • 2 years
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
7.5%
3/40 • 2 years
|
4.2%
1/24 • 2 years
|
8.7%
4/46 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.0%
2/40 • 2 years
|
0.00%
0/24 • 2 years
|
6.5%
3/46 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
2.5%
1/40 • 2 years
|
8.3%
2/24 • 2 years
|
0.00%
0/46 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.5%
3/40 • 2 years
|
0.00%
0/24 • 2 years
|
6.5%
3/46 • 2 years
|
|
Nervous system disorders
Headache
|
20.0%
8/40 • 2 years
|
20.8%
5/24 • 2 years
|
23.9%
11/46 • 2 years
|
|
Nervous system disorders
Migraine
|
0.00%
0/40 • 2 years
|
8.3%
2/24 • 2 years
|
2.2%
1/46 • 2 years
|
|
Nervous system disorders
Presyncope
|
0.00%
0/40 • 2 years
|
8.3%
2/24 • 2 years
|
2.2%
1/46 • 2 years
|
|
Nervous system disorders
Somnolence
|
7.5%
3/40 • 2 years
|
0.00%
0/24 • 2 years
|
0.00%
0/46 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
5/40 • 2 years
|
16.7%
4/24 • 2 years
|
13.0%
6/46 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.5%
3/40 • 2 years
|
0.00%
0/24 • 2 years
|
0.00%
0/46 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
12.5%
5/40 • 2 years
|
16.7%
4/24 • 2 years
|
8.7%
4/46 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
2.5%
1/40 • 2 years
|
8.3%
2/24 • 2 years
|
0.00%
0/46 • 2 years
|
|
Skin and subcutaneous tissue disorders
Lipohypertrophy
|
5.0%
2/40 • 2 years
|
8.3%
2/24 • 2 years
|
8.7%
4/46 • 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place