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CLBS03 Low Dose

Phase 2

Diabetes Mellitus | Monoclonal antibody | Metabolic |Lisata Therapeutics, Inc.|Last Updated: Jan 8, 2021

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment113
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02691247Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)PHASE2 COMPLETED 113Feb 1, 2016Jan 1, 2020Jan 8, 202115 United States
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Study Endpoints
Primary Endpoints
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 52
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 Endpoints
Change From Baseline in 4-hour Mixed Meal Tolerance Test (MMTT)-Stimulated C-peptide Area Under the Curve (AUC) at Week 104
Week 104
Change in Hemoglobin A1c (HbA1c)
Week 104
Change From Baseline in Mean Daily Dose of Insulin
Week 104
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelFACTORIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CLBS03 Low DoseEXPERIMENTALA 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 High DoseEXPERIMENTALA single infusion of CLBS03 High Dose, a cell product comprised of autologous, ex vivo expanded regulatory T-cells resuspended in sterile USP infusion solution.
PlaceboPLACEBO_COMPARATORA single infusion of placebo, consisting of the infusion solution only
Interventions
NameTypeDescription
CLBS03 Low DoseBIOLOGICAL -
CLBS03 High DoseBIOLOGICAL -
PlaceboBIOLOGICAL -
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Eligibility Criteria
Age Range8 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites15

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 r...

Countries:United States
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