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Emtricitabine /Tenofovir Disoproxil

Phase 2

Primary Biliary Cholangitis | Small molecule | Other |Merck & Company, Inc.|Last Updated: Feb 16, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment37
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03954327Combination Antiretroviral Therapy (cART) for PBCPHASE2 COMPLETED 37Mar 1, 2021Jan 1, 2024Feb 16, 20246 Canada
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Study Endpoints
Primary Endpoints
Change in alkaline phosphatase levels
12 months

Mean changes in alkaline phosphatase levels after 12 months treatment with combination antiretroviral therapy or placebo.

Secondary Endpoints
Serial changes in alkaline phosphatase
Evaluation baseline, 3 months, 6 months and end of RCT; then 3 months, 6 monthly to end of open label therapy]
Serial changes in ALT
Evaluation baseline, 3 months, 6 months and end of RCT; then 3 months, 6 monthly to end of open label therapy]
Serial changes in bilirubin
Evaluation baseline, 3 months, 6 months and end of RCT; then 3 months, 6 monthly to end of open label therapy]
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Emtricitabine (FTC)/Tenofovir Disoproxil (TDF) & RaltegravirEXPERIMENTALone Emtricitabine 200mg and Tenofovir Disoproxil 300mg tablet two Raltegravir 600mg tablets
PlaceboPLACEBO_COMPARATORIdentical tablets resembling one Emtricitabine 200mg and Tenofovir Disoproxil 300mg tablet two Raltegravir 600mg tablets
Interventions
NameTypeDescription
Emtricitabine (FTC)/Tenofovir Disoproxil (TDF)DRUGEmtricitabine (FTC) 200 mg/Tenofovir Disoproxil (TDF) 300 mg by mouth once per day
RaltegravirDRUGRaltegravir (RTF) 600 mg two tablets by mouth once per day
Placebo Oral Capsule [CEBOCAP]DRUGTwo capsules identical to Raltegravir and one capsule identical to Truvada with no active ingredients by mouth once per day
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: * over 18 years old of either sex, * Anti-mitochondrial antibody +ve or liver histology compatible with PBC, * stable UDCA dose of 13-15 mg/kg for \> 12 months or intolerant to UDCA, * ALP at least 1.67 x ULN or abnormal bilirubin less than 2x ULN * able to read and sign informe...

Countries:Canada
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