Recent Updates
Recently added Catalysts

Deferitrin

Phase 1

Beta-Thalassemia | Small molecule | Hematology |Sanofi|Last Updated: Mar 5, 2015

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment25
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00069862Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-ThalassemiaPHASE1 COMPLETED 25Sep 1, 2003Nov 1, 2005Mar 5, 20151 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
safety and tolerability
iron excretion in urine and stool
pharmacokinetic measurements
Unlock Study Endpoints
Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeTREATMENT
Interventions
NameTypeDescription
Deferitrin (GT56-252)DRUG -
desferoxamine (DFO)DRUG -
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Beta-thalassemia patients, 18 years of age or older, currently undergoing chronic blood transfusion therapy and iron chelation therapy who weigh more than 40 kg. * No clinically significant findings on physical exam, medical history, or screening laboratories. * Serum ferritin...

Countries:United States
Unlock Eligibility Criteria