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Deferasirox and Deferoxamine

Phase 2

Cardiac Iron Overload | Small molecule | Other |Novartis AG|Last Updated: Jul 26, 2021

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
UNCONTROLLED
Total Trials1
Total Enrollment60
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01254227Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron OverloadPHASE2 COMPLETED 60 — — Jan 1, 2011Nov 1, 2013Jul 26, 202115 Canada, Egypt +6
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Study Endpoints
Primary Endpoints
Change in Cardiac Iron Content From Baseline to Month 12
From Baseline to Month 12

Cardiac T2\* is the most sensitive and reproducible test in detecting myocardial iron load. A cardiac T2\* value of \<10 ms is defined as severe cardiac iron overload. Participants who do not have baseline T2\* or do not have any post-baseline T2\* are excluded from the analysis.

Secondary Endpoints
Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24
From the Months 6, 12, 18 and 24
Change in Cardiac Iron Content From Baseline to Month 6,18 and 24
From Baseline to Months 6, 18 and 24
Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24
From the Months 6, 12, 18 and 24
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
DeferasiroxEXPERIMENTALDeferoxamine combination followed by Deferasirox monotherapy
Interventions
NameTypeDescription
Deferasirox and DeferoxamineDRUG -
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Eligibility Criteria
Age Range10 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites15

Inclusion Criteria: * Patients with β-thalassemia major or Diamond-Blackfan anemia (DBA) or congenital sideroblastic anemia on chronic transfusion therapy * Myocardial T2\* value that is ≥ 5 and \< 10 ms * Left ventricular ejection fraction (LVEF) ≥ 56% as determined by Magnetic resonance imaging (...

Countries:CanadaEgyptGreeceItalyTaiwanThailandTurkey (Türkiye)United Kingdom
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