Recent Updates
Recently added Catalysts

CNSA-001

Phase 2

Gastroparesis | Small molecule | Other |PTC Therapeutics, Inc.|Last Updated: Jan 5, 2022

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment21
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03712124A Study of CNSA-001 in Women With Diabetic GastroparesisPHASE2 COMPLETED 21 — — Feb 27, 2019Oct 8, 2019Jan 5, 20226 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Change From Baseline (Day 1) in Maximal Tolerated Volume Consumed During the Nutrient Satiety Test at Day 14
Baseline (Day 1), Day 14

Participants consumed 150 milliliters (mL) of Ensure™ every 5 minutes for the nutrient satiety test. At 5-minute intervals, participants scored their fullness using a rating scale that combines verbal descriptors on a scale graded 0 to 5 (0 = no symptoms, 1 = first sensation of fullness \[threshold\], 2 = mild, 3 = moderate, 4 = severe and 5 = maximum or unbearable fullness). Participants were told to stop when a score of 5 was obtained. The actual volume of Ensure™ consumed at this point was the maximum tolerated volume.

Change From Baseline (Day 1) in Maximal Tolerated Volume Consumed During the Nutrient Satiety Test at Day 28
Baseline (Day 1), Day 28

Participants consumed 150 mL of Ensure™ every 5 minutes for the nutrient satiety test. At 5-minute intervals, participants scored their fullness using a rating scale that combines verbal descriptors on a scale graded 0 to 5 (0: no symptoms, 1 = first sensation of fullness \[threshold\], 2 = mild, 3 = moderate, 4 = severe and 5 = maximum or unbearable fullness). Participants were told to stop when a score of 5 was obtained. The actual volume of Ensure™ consumed at this point was the maximum tolerated volume.

Secondary Endpoints
Change From Baseline (Day 1) in the Gastroparesis Cardinal Symptom Index (GCSI) Total Score at Day 14/15 and Day 28
Baseline (Day 1), Days 14/15 and 28
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Baseline (Day 1), Days 14 and 28
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Baseline (Day 1), Days 14 and 28
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CNSA-001EXPERIMENTALParticipants will receive CNSA-001 20 mg/kg/day (10 mg/kg twice daily \[BID\]) as an oral suspension for 14 days.
PlaceboPLACEBO_COMPARATORParticipants will receive placebo matching to CNSA-001 BID for 14 days.
Interventions
NameTypeDescription
CNSA-001DRUGCNSA-001 Powder for Suspension
PlaceboDRUGPlacebo Suspension
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — 75 Years
SexFEMALE
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: * Informed consent * Diagnosis of diabetes mellitus * Documentation of delayed gastric emptying on gastric emptying scintigraphy or gastric emptying breath test (GEBT) (within 2 year of enrollment) * Symptoms of gastroparesis for at least 6 months with GCSI score \>21 indicating...

Countries:United States
Unlock Eligibility Criteria
Competitive Landscape -Gastroparesis 2 trials
CompanyTickerTrialsLead PhaseDrugs
Vanda Pharmaceuticals Inc.VNDA1PHASE4Tradipitant
United Therapeutics CorporationUTHR1PHASE4Undisclosed
Unlock Competitive Intelligence