CLINICAL TRIALS PROFILE FOR DEFERASIROX

505(b)(2) Clinical Trials for DEFERASIROX

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT02435212 ↗	Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload	Active, not recruiting	Novartis Pharmaceuticals	Phase 2	2015-10-21	This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox DT formulation in children and adolescents aged ≥ 2 and < 18 years at enrollment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden. Randomization will be stratified by age groups (2 to <10 years, 10 to <18 years) and prior iron chelation therapy (Yes/ No). There will be two study phases which include a 1 year core phase where patients will be randomized to a 48 week treatment period to either Deferasirox DT or granules, and an optional extension phase where all patients will receive the granules up to 5 years. Patients who demonstrated benefit to granules or DT in the core phase, and/or express the wish to continue in the optional extension phase on granules, will be offered this possibility until there is local access to the new formulation (granules or FCT) or up to 5 years, whichever occurs first.

All Clinical Trials for DEFERASIROX

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00061750 ↗	Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions	Completed	Novartis Pharmaceuticals	Phase 3	2003-05-01	The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
NCT00061763 ↗	Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias	Completed	Novartis Pharmaceuticals	Phase 2	2003-05-01	The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions. Beta-thalassemia patients unable to be treated with deferoxamine or patients with rare chronic anemias such as Myelodysplastic Syndrome, Fanconi's Syndrome, Blackfan-Diamond Syndrome, and Pure Red Blood Cell Anemia are eligible for this study. Liver iron content will be measured by liver biopsy at the beginning of the study and after one year of treatment. However, those patients living in the San Francisco/Oakland area may have a SQUID in place of the liver biopsy if the biopsy is not medically possible for them. The SQUID is a non-invasive magnetic means to measure liver iron content.
NCT00067080 ↗	Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions	Completed	Novartis Pharmaceuticals	Phase 2	2003-05-01	The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
NCT00110266 ↗	Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients	Completed	Novartis Pharmaceuticals	Phase 2	2005-07-25	The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.
NCT00110617 ↗	Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients	Completed	Novartis Pharmaceuticals	Phase 2	2005-05-01	This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
NCT00117507 ↗	Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients	Completed	Novartis Pharmaceuticals	Phase 4	2005-09-01	Thirty patients were to be enrolled and 24 patients were actually enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusion dependent myelodysplastic syndrome (MDS) patients with iron overload. Patients enrolled in this study had low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria. All patients initiated treatment with 20mg/kg/day deferasirox. Deferasirox were administered orally once per day for 12 months.

Clinical Trial Conditions for DEFERASIROX

Condition Name

Condition Name for DEFERASIROX
Intervention	Trials
Iron Overload	16
Myelodysplastic Syndromes	12
Beta-Thalassemia	7
Transfusional Iron Overload	7

Condition MeSH

Condition MeSH for DEFERASIROX
Intervention	Trials
Iron Overload	52
Thalassemia	36
beta-Thalassemia	25
Preleukemia	25

Clinical Trial Locations for DEFERASIROX

Trials by Country

Trials by Country for DEFERASIROX
Location	Trials
United States	206
Italy	71
Canada	21
United Kingdom	19
China	19

Trials by US State

Trials by US State for DEFERASIROX
Location	Trials
California	23
New York	18
Illinois	15
Pennsylvania	14
Texas	12

Clinical Trial Progress for DEFERASIROX

Clinical Trial Phase

Clinical Trial Phase for DEFERASIROX
Clinical Trial Phase	Trials
Phase 4	21
Phase 3	11
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for DEFERASIROX
Clinical Trial Phase	Trials
Completed	62
Terminated	14
Unknown status	11

Clinical Trial Sponsors for DEFERASIROX

Sponsor Name

Sponsor Name for DEFERASIROX
Sponsor	Trials
Novartis Pharmaceuticals	52
Novartis	11
National Cancer Institute (NCI)	2

Sponsor Type

Sponsor Type for DEFERASIROX
Sponsor	Trials
Other	88
Industry	69
NIH	3