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Sargramostim

Phase 3

Crohn Disease | Small molecule | Immunology |Sanofi|Last Updated: Dec 4, 2013

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-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment413
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00206674Efficacy (Induction of Response/Remission) and Safety Study in Patients With Moderate to Severe Crohn's DiseasePHASE3 COMPLETED 286Sep 1, 2004Jun 1, 2006Dec 4, 201370 Argentina, Australia +7
NCT00206596Study in Patients With Crohn's Disease Who Are Steroid Dependent, Despite Previous Unsuccessful Attempts to Reduce Steroids Due to Worsening of Crohn's DiseasePHASE2 COMPLETED 127Jul 1, 2003Oct 1, 2005Dec 4, 2013 -
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Study Endpoints
Primary Endpoints
Induction of clinical response and/or remission as defined by decrease in CDAI score
After 8 weeks of treatment
Corticosteroid-free remission
At the end of the study treatment
Secondary Endpoints
Time and duration to response/remission
During the 8 weeks of treatment
QoL (different methods)
During the whole study
Safety Profile ( Adverse Event, Serious Adverse Event collection, laboratories especially AB test etc.)
About 20 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Arm 1EXPERIMENTAL -
Arm 2PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
Sargramostim (Leukine)DRUG6µg/kg SARGRAMOSTIM subcutaneous daily for 8 weeks
PlaceboDRUGSterile liquid solution, inactive excipient only subcutaneous daily for 8 weeks
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites70

Inclusion Criteria: * Stable but active Crohn's disease at the time of screening * Must be able to self-inject or have another person who can help with the injection Exclusion Criteria: * Not have a colostomy or ileostomy * Not be taking prohibited medications as defined in the protocol * Not hav...

Countries:ArgentinaAustraliaBrazilCanadaNew ZealandRussiaSwitzerlandUkraineUnited Kingdom
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