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L-PZQ ODT /kg

Phase 3

Schistosomiasis | Small molecule | Other |Merck & Company, Inc.|Last Updated: Mar 21, 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
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment288
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03845140L-PZQ ODT in Schistosoma Infected ChildrenPHASE3 COMPLETED 288Sep 2, 2019Oct 11, 2021Mar 21, 20242 Côte d’Ivoire, Kenya
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Study Endpoints
Primary Endpoints
Cohort 1a and Cohort 1b: Number of Participants With Clinical Cure Determined by Kato-Katz Method
at Week 3

Clinical cure was defined as no parasite egg in the stool at Week 3 as determined by the Kato-Katz method. Number of participants with clinical cure were reported.

Secondary Endpoints
Cohort 2 and Cohort 3: Number of Participants With Clinical Cure Determined by Kato-Katz Method
at Week 3
Cohort 4a and Cohort 4b: Number of Participants With Clinical Cure Determined by Urine Filtration Technique
Week 3 and Week 5
Cohort 1a, Cohort 1b, Cohort 2 and Cohort 3: Egg Reduction Rate (Percent [%]) Determined by Kato-Katz Method
Pre-treatment, Week 3 post-treatment
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1a: 4 to 6 years L-PZQ ODT 50 mg/kgEXPERIMENTALParticipants aged 4 to 6 years infected with Schistosoma (S.) mansoni received Levorotatory enantiomer of praziquantel (L-PZQ) orodispersible tablets (ODT) (150 milligrams \[mg\]) orally at a dose of 50 milligram per kilogram (mg/Kg) as a single oral dose after food-intake on Day 1.
Cohort 1b: 4 to 6 years Biltricide® 40 mg/kgACTIVE_COMPARATORParticipants aged 4 to 6 years infected with S. mansoni received Racemate Praziquantel tablets (Biltricide®) (600 mg) orally at a dose of 40 mg/kg as a single oral dose after food-intake on Day 1.
Cohort 2: 2 to 3 years L-PZQ ODT 50 mg/kgEXPERIMENTALParticipants aged 2 to 3 years infected with S. mansoni received L-PZQ ODT (150 mg) tablet orally at a dose of 50 mg/kg as a single oral dose after food-intake on Day 1.
Cohort 3: 3 to 24 months L-PZQ ODT 50 mg/kgEXPERIMENTALParticipants aged 3 to 24 months infected with S. mansoni received L-PZQ ODT (150 mg) tablet orally at a dose of 50 mg/kg as a single oral dose after food-intake on Day 1.
Cohort 4a: 3 months to 6 years L-PZQ ODT 50 mg/kgEXPERIMENTALParticipants aged 3 months to 6 years infected with S. haematobium received L-PZQ ODT (150 mg) tablet orally at a dose of 50 mg/kg as a single oral dose after food-intake on Day 1.
Cohort 4b: 3 months to 6 years L-PZQ ODT 60 mg/kgEXPERIMENTALParticipants aged 3 months to 6 years infected with S. haematobium received L-PZQ ODT (150 mg) tablet orally at a dose of 60 mg/kg as a single oral dose after food-intake on Day 1.
Interventions
NameTypeDescription
L-PZQ ODT 50 mg/kgDRUGParticipants received single oral dose of L-PZQ ODT 50 mg/Kg on Day 1.
Biltricide®DRUGParticipants received single oral dose of Biltricide® 40 mg/kg on Day 1.
L-PZQ ODT 60 mg/kgDRUGParticipant received single oral dose of L-PZQ ODT 60 mg/kg on Day 1.
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Eligibility Criteria
Age Range3 Months — 6 Years
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Age of the participant is 4 to 6 years of age (Cohorts 1 and 4), 2 to 3 years of age (Cohorts 2 and 4) 3 to less than 24 months of age (Cohorts 3 and 4) * Participants are; Schistosoma (S.) mansoni positive (Cohorts 1, 2, and 3); diagnosis defined as positive egg counts in sto...

Countries:Côte d’IvoireKenya
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