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RJX

Phase 1

Critical Limb Ischemia | Small molecule | Other |Icon Plc|Last Updated: Apr 27, 2020

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 Enrollment76
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03680105A Safety and Tolerability Study of RJX Drug Product in Healthy ParticipantsPHASE1 COMPLETED 76Aug 24, 2018Jan 15, 2019Apr 27, 20201 United States
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Study Endpoints
Primary Endpoints
Treatment-related Adverse Events (TEAE) Reporting of RJX
Up to Day 5 for Part 1 and Up to Day 12 for Part 2

Number of participants with indicated AEs receiving RJX as assessed by CTCAE v4 03

Safety and Tolerability of RJX as Assessed by Electrocardiograms (ECGs).
Up to Day 2 for Part 1 and Up to Day 8 for Part 2

Number of participants with abnormal and clinically significant findings based on ECG.

Safety and Tolerability of RJX as Assessed by Neurological Examinations.
Up to Day 5 for Part 1 and Up to Day 12 for Part 2

Number of participants with clinically significant values and actual changes from baseline of continuous neurological assessments.

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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeBASIC_SCIENCE
Treatment Arms
ArmTypeDescription
Part 1; Cohort 1; RJX or PlaceboEXPERIMENTALParticipants in Part 1; Cohort 1 will receive a single 0.024 mL/kg dose of RJX or matching placebo on Day 1.
Part 1; Cohort 2; RJX or PlaceboEXPERIMENTALParticipants in Part 1; Cohort 2 will receive a single 0.076 mL/kg dose of RJX or matching placebo on Day 1.
Part 1; Cohort 3; RJX or PlaceboEXPERIMENTALParticipants in Part 1; Cohort 3 will receive a single 0.240 mL/kg dose of RJX or matching placebo on Day 1.
Part 1; Cohort 4; RJX or PlaceboEXPERIMENTALParticipants in Part 1; Cohort 4 will receive a single 0.5 mL/kg dose of RJX or matching placebo on Day 1.
Part 1; Cohort 5; RJX or PlaceboEXPERIMENTALParticipants in Part 1; Cohort 5 will receive a single 0.759 mL/kg dose of RJX or matching placebo on Day 1.
Part 1; Cohort 6; RJX or PlaceboEXPERIMENTALParticipants in Part 1; Cohort 6 will receive a single dose of RJX or matching placebo, to be determined following review of safety and PK data from Cohorts 1 to 5, on Day 1.
Part 2; Cohort 1; RJX or PlaceboEXPERIMENTALParticipants in Part 2; Cohort 1 will receive a dose of RJX or matching placebo, determined based on the findings of Part 1, every day for 7 days. The Part 2; Cohort 1 dose will be 1 log down from the maximum tolerated dose determined in Part 1 or at a dose determined to be safe and well tolerated in Part 1 with an acceptable PK profile.
Part 2; Cohort 2; RJX or PlaceboEXPERIMENTALParticipants in Part 2; Cohort 2 will receive an escalated dose of RJX or matching placebo, determined based on the findings of Part 1, every day for 7 days.
Part 2; Cohort 3; RJX or PlaceboEXPERIMENTALParticipants in Part 2; Cohort 3 will receive an escalated dose of RJX or matching placebo, determined based on the findings of Part 1, every day for 7 days.
Interventions
NameTypeDescription
RJXDRUGRJX is an IV infusion formulation. The appropriate RJX dose is calculated based on mL/kg. The administration dose is prepared by adding the appropriate RJX dose to 0.9% sterile saline such that a constant volume of 100 mL per IV infusion is obtained. Infusion time is 100 mL over 45 minutes (+/- 5 minutes).
PlaceboDRUGMatching placebo will be normal saline. To maintain the blind, a sleeve will cover the infusion bag and line such that the appearance will be identical to RJX administration.
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Healthy male and female participants aged 18 to 50 years inclusive (Cohort 1 to Cohort 5) or 51 to 70 years inclusive (Cohort 6 only),at the time of screening; 2. Have a body mass index of 18 kg/m2 to 35 kg/m2, inclusive, at screening. In addition, weight cannot exceed 132 kg...

Countries:United States
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