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ICL670, deferoxamine

Phase 2

Anemia, Sickle Cell | Small molecule | Hematology |Novartis AG|Last Updated: Aug 22, 2017

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
RandomizedUNCONTROLLEDBiomarker
Total Trials1
Total Enrollment195
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00067080Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood TransfusionsPHASE2 COMPLETED 195 — — May 1, 2003 -Aug 22, 201734 United States
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Study Endpoints
Primary Endpoints
Evaluate the safety and tolerability of multiple doses of ICL670
1 year
Secondary Endpoints
Estimate the absolute and relative change of liver iron content (LIC) and total body iron excretion (TBIE)
at baseline, after 24 weeks and at 1year (end of study)
Evaluate the pharmacokinetics
24 hours post-dose @ 4, 12, 24 and 52 weeks
Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variables
at 24 and 52 weks pre-dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ICL670 + deferoxamineEXPERIMENTAL -
Interventions
NameTypeDescription
ICL670, deferoxamineDRUG -
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Eligibility Criteria
Age Range2 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites34

Inclusion Criteria: * Age greater than or equal to 2 years * Sickle cell disease patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day * Serum ferritin greater than 1000 mg/ml * Liver iron content greater than 2 mg iron/g dw assessed by means of superconductin...

Countries:United States
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