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Deferasirox DFX, DT

Phase 2

Low and Int 1-risk Myelodysplastic Syndrome | Small molecule | Hematology |Novartis AG|Last Updated: Oct 31, 2018

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLED
Total Trials1
Total Enrollment28

FDA Designations

No designations recorded

Clinical trial landscape

Deferasirox DFX, DT · 1 trial · 1 indication

Phase 2 1
NCT01868477Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome.Low and Int 1-risk Myelodysplastic Syndrome
COMPLETED28 Analytics
PHASE2COMPLETED
Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome.
Low and Int 1-risk Myelodysplastic SyndromeUnlock trial analytics

Study Endpoints

Primary Endpoints

Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)
Baseline up to 12 weeks

Difference in percentage of patients achieving an erythroid response within 12 weeks of treatment between the two arms according to modified IWG 2006 criteria increase in hemoglobin (Hb) ≥ 1.5 g/dL. Erythroid response is defined as the increase in Hb from baseline ≥ 1.5 g/dL. Patients achieving erythroid response at least once within 12 weeks were considered responders

Secondary Endpoints

Absolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)
Baseline up to 24 weeks
Summary of Hematologic Improvement in Patients Randomized to EPO+DFX and EPO Alone, Within 24 Weeks of Treatment (Full Analysis Set)
Baseline up to 24 weeks
Absolute Change in Hemoglobin Values up to 24 Weeks
Baseline up to 24 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Erythropoietin alphaEXPERIMENTALPatients will receive erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be switched to the combination arm. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study.
Deferasirox + Erythropoietin alphaEXPERIMENTALPatients will receive deferasirox dispersible tablet (DT) 10 mg/kg/day or deferasirox film-coated tablet (FCT) 7 mg/kg/day in combination with erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, erythropoietin dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be discontinued from the study. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study. Patients will continue deferasirox treatment.

Interventions

NameTypeDescription
Deferasirox DFX, DTDRUGprovided as dispersible tablets for oral use in 125 and 250, 500 mg
Erythropoietin alphaDRUG -
Deferasirox DFX, FCTDRUGprovided as film-coated tablet for oral use in 90, 180, 360 mg strengths
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites30

Key Inclusion Criteria: * Patients who had low- and Int-1-risk myelodysplastic syndrome * Documented diagnosis of the following: Myelodysplastic syndrome that lasted ≥ 3 months and \< 3 years Disease must not have been secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy fo...

Countries:AlgeriaArgentinaCanadaChinaGermanyItalySouth KoreaSpainSwedenUnited Kingdom
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Frequently asked questions about Deferasirox DFX, DT

What is Deferasirox DFX, DT used for?

Deferasirox DFX, DT is an investigational small molecule being studied for the treatment of Low and Int 1-risk Myelodysplastic Syndrome, a group of blood cancers. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.

Who makes Deferasirox DFX, DT?

Deferasirox DFX, DT is being developed by Novartis AG, a multinational pharmaceutical company listed on the stock exchange under the ticker symbol NVS. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Low and Int 1-risk Myelodysplastic Syndrome.

What phase is Deferasirox DFX, DT in?

Deferasirox DFX, DT is currently in Phase 2 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory agencies. The drug is being studied for the treatment of Low and Int 1-risk Myelodysplastic Syndrome, and its safety and efficacy are still being evaluated in clinical trials.

What clinical trials is Deferasirox DFX, DT in?

Deferasirox DFX, DT has been studied in one completed clinical trial, identified as NCT01868477. This Phase 2 trial, titled "Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome," enrolled 28 participants across multiple countries including Algeria, Argentina, Canada, China, Germany, Italy, South Korea, Spain, Sweden, and the United Kingdom.

Is Deferasirox DFX, DT the same as Deferasirox?

Deferasirox DFX, DT is a formulation of deferasirox, an iron chelator. The DFX, DT designation indicates a specific formulation or delivery technology. In the context of the clinical trial NCT01868477, it is being studied in combination with erythropoietin for patients with Low and Int 1-risk Myelodysplastic Syndrome.