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VC-02 Combination Product

Phase 1

Type 1 Diabetes Mellitus With Hypoglycemia | Small molecule | Metabolic |Vertex Pharmaceuticals Incorporated|Last Updated: Dec 3, 2024

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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment49
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03163511A Safety, Tolerability, and Efficacy Study of VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus and Hypoglycemia UnawarenessPHASE1 COMPLETED 49Jul 6, 2017Oct 19, 2023Dec 3, 202410 United States, Belgium +1
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Study Endpoints
Primary Endpoints
Incidence of All AEs in Cohort 1 Participants
Enrollment (Visit 3, Day 1) through the Month 4 Visit

Incidence of all AEs and evaluation of causality related to VC-02 combination product, the surgical procedures, and the immunosuppressive drug regimen.

Change in C-peptide for Cohort 2 Subjects
Baseline to Week 26 Visit

Change from baseline to Week 26 in C-peptide area under the curve from 0 to 4 hours following a mixed meal tolerance test.

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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeOTHER
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTALVC-02 Combination Product: Up to ten (10) VC-02-20 implants and up to two (2) VC-02-300 implants
Cohort 2EXPERIMENTALVC-02 Combination Product; Up to twelve units implanted of which up to ten (10) are VC-02-300 implants and the rest are VC-02-20 implants.
Interventions
NameTypeDescription
VC-02 Combination ProductCOMBINATION_PRODUCTPEC-01 cells loaded into a Delivery Device
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Men and non-pregnant women * Diagnosis of T1DM for a minimum of five (5) years * Hypoglycemia unawareness or significant glycemic lability * Stable diabetic treatment * Willingness to use a continuous glucose meter * Acceptable candidate for implantation Exclusion Criteria: ...

Countries:United StatesBelgiumCanada
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