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Genz-644282

Phase 1

Solid Tumors | Small molecule | Oncology |Sanofi|Last Updated: Mar 19, 2014

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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment87
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00942799Dose Escalation Study to Assess the Safety and Tolerability of Genz-644282 in Patients With Solid TumorsPHASE1 COMPLETED 87Jul 1, 2009Jan 1, 2013Mar 19, 20144 United States
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Study Endpoints
Primary Endpoints
Determine the Maximum Tolerated Doses for the 28-day and 21-day dosing schedules
24 Months
Secondary Endpoints
Determine the recommended Phase 2 doses for the 28-day and 21-day dosing schedules
36 Months
Establish the pharmacokinetic (PK) profile through calculation of area under the curve of blood and urine concentrations.
36 Months
Pharmacokinetics (PK) as measured by maximal observed concentration (Cmax)
36 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Study Drug: Genz-644282 (28-day dosing schedule)EXPERIMENTALGenz-644282 (Each cohort will be based on dose-escalation)
Study Drug: Genz-644282 (21-day dosing schedule)EXPERIMENTALGenz-644282 (Each cohort will be based on dose-escalation)
Interventions
NameTypeDescription
Genz-644282 (28-day dosing schedule)DRUGGenz-644282 will be administered as a 60-minute IV infusion. The initial dose of Genz-644282 will be 0.5 mg/m2 and will be administered once weekly for 3 consecutive weeks on Day 1, Day 8, and Day 15 of the 28 day cycle. Each 28-day period will represent one treatment cycle. Study drug will continue to be given until disease progression or unacceptable toxicity is observed.
Genz-644282 (21-day dosing schedule)DRUGThe starting dose of Genz-644282 for the 21-day schedule will be prescriptive and will be based on review of available safety data from the 28-day dosing schedule by the Investigator and Sponsor. Patients will receive Genz-644282 once-weekly for 2 consecutive weeks on Day 1 and Day 8 of a 21 day cycle. Each 21-day period will represent 1 treatment cycle. Patients enrolled in subsequent cohorts will receive higher doses until the MTD is established.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Signed written informed consent * Patients enrolled in dose-escalation phase: Histologically or cytologically confirmed advanced malignant solid tumor for which no standard therapeutic option exists. * For the disease indications evaluated in the expansion phase, the following...

Countries:United States
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