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ICL670

Phase 3

Beta-Thalassemia | Small molecule | Hematology |Novartis AG|Last Updated: Apr 19, 2012

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
RandomizedACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment595
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00061750Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood TransfusionsPHASE3 COMPLETED 595May 1, 2003 -Apr 19, 20127 United States
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Study Endpoints
Primary Endpoints
Demonstrate non-inferiority to deferoxamine in its effects on liver iron content (LIC)
Secondary Endpoints
Evaluate tolerability profile
Estimate absolute and relative change of LIC and Total body iron excretion
Evaluation relationship between LIC and potential surrogate markers
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ICL670EXPERIMENTAL -
DeferoxamineACTIVE_COMPARATOR -
Interventions
NameTypeDescription
ICL670DRUG -
deferoxamineDRUG -
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Eligibility Criteria
Age Range2 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: * Beta-thalassemia patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day * Liver iron content greater than 2 mg iron/g dw as measured by liver biopsy * Need for regular transfusions 8 or more times per year Exclusion Criteria: * Non-trans...

Countries:United States
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