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PF-06649751

Phase 1

Idiopathic Parkinson Disease | Small molecule | Neurology |Pfizer, Inc.|Last Updated: Sep 22, 2023

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-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment18
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02373072A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of PF-06649751 in Subjects With Idiopathic Parkinson's DiseasePHASE1 COMPLETED 18Mar 1, 2015Mar 1, 2016Sep 22, 20238 United States, Belgium
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Study Endpoints
Primary Endpoints
Number and proportion of subjects with Adverse Events (AEs)
Day 1 through 61
Number of participants with vital signs data that meet criteria of potential clinical concern
Day 1 through 61
Number of participants with ECG data that meet criteria of potential clinical concern
Day 1 through 61
Number of participants with abnormal clinically significant laboratory measurements
Day 1 through 61
C-SSRS (suicidality assessment)
Day 1 through 61
Secondary Endpoints
MDS-UPDRS part III
Day 1, Periods 1-3
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeBASIC_SCIENCE
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTALThree single doses over three periods. For each of the three periods, subjects will be crossed-over from placebo or PF-06649751. Each PF-06649751 dose is separated by at least one week.
Cohort 2EXPERIMENTALThree single doses over three periods. For each of the three periods, subjects will be crossed-over from placebo or PF-06649751. Each PF-06649751 dose is separated by at least one week.
Interventions
NameTypeDescription
PF-06649751DRUGSubjects completing all three treatment periods will be administered two doses. Doses: 0.75mg, 1.5mg, 3mg, 6mg, 9mg. Tablets in the form of 0.25mg or 1 mg.
Trimethobenzamide HydrochlorideDRUG300mg TID, Capsules. Optional in both Cohorts.
PlaceboDRUGSubjects completing all three treatment periods will be receiving placebo once.
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Eligibility Criteria
Age Range30 Years — 85 Years
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * L-DOPA-responsiveness * Hoehn \& Yahr Stage II-III inclusive * Experiencing motor fluctuations * Stable daily dose of L-DOPA of at least 300 mg * Females on non-childbearing potential and male subjects Exclusion Criteria: * History of troublesome dyskinesias * History of sur...

Countries:United StatesBelgium
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