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Deferasirox dispersible

Phase 2

Chronic Iron Overload Due to Transfusion-dependant Anemias | Small molecule | Hematology |Novartis AG|Last Updated: Jul 25, 2017

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLED
Total Trials1
Total Enrollment173

FDA Designations

No designations recorded

Clinical trial landscape

Deferasirox dispersible · 1 trial · 1 indication

Phase 2 1
NCT02125877Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)Chronic Iron Overload Due to Transfusion-dependant Anemias
COMPLETED173 Analytics
PHASE2COMPLETED
Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
Chronic Iron Overload Due to Transfusion-dependant AnemiasUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall Safety as Measured by Frequency of Adverse Events
28 weeks

The percentage of participants with adverse events, serious adverse events and deaths was assessed.

Overall Safety as Measured by Changes in Laboratory Values From Baseline
baseline (BL), 30 weeks

The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary.

Secondary Endpoints

Frequency of Selected Gastro-intestinal (GI) Adverse Events
28 weeks
Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Palatability Questionnaire Score
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Deferasirox dispersible tablet (DFX-DT)ACTIVE_COMPARATORIron chelation naïve participants received DFX-DT 20 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 5 to 10 mg/kg/day, with a maximum dose of 40 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose.
Deferasirox film-coated tablet (DFX-FCT)EXPERIMENTALParticipants received DFX-FCT 14 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 3.5 to 7 mg/kg/day, with a maximum dose of 28 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose

Interventions

NameTypeDescription
Deferasirox dispersible tabletDRUGDeferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
Defearisox film-coated tabletDRUGDeferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
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Eligibility Criteria

Age Range10 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites51

Key Inclusion Criteria: * Male and female patients aged ≥ 10 years * Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic sy...

Countries:United StatesArgentinaAustriaFranceGermanyGreeceItalyLebanonMalaysiaMexicoRussiaSaudi ArabiaSpainThailandUnited Arab EmiratesUnited Kingdom
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Frequently asked questions about Deferasirox dispersible

What is Deferasirox dispersible used for?

Deferasirox dispersible is used for chronic iron overload due to transfusion-dependent anemias. It is a small molecule being developed by Novartis AG for this hematologic condition. The drug is currently in Phase 2 clinical development and is considered investigational.

What does Deferasirox dispersible target?

Deferasirox dispersible is an iron chelator that binds to iron in the body, helping to remove excess iron that accumulates from repeated blood transfusions. This mechanism addresses chronic iron overload in patients with transfusion-dependent anemias, reducing the risk of iron-related organ damage.

Who makes Deferasirox dispersible?

Deferasirox dispersible is developed by Novartis AG, a multinational pharmaceutical company listed on the stock exchange under the ticker NVS. The drug is being studied for the treatment of chronic iron overload due to transfusion-dependent anemias and is currently in Phase 2 clinical trials.

What phase is Deferasirox dispersible in?

Deferasirox dispersible is in Phase 2 clinical development. It is an investigational drug, not yet approved by regulatory authorities. A Phase 2 study has been completed to evaluate the benefits of an improved film-coated tablet formulation of deferasirox in patients with chronic iron overload.

What clinical trials is Deferasirox dispersible in?

Deferasirox dispersible has one completed clinical trial, NCT02125877, a Phase 2 study investigating the benefits of an improved film-coated tablet formulation. The trial enrolled 173 participants with chronic iron overload due to transfusion-dependent anemias across multiple countries, including the United States, Argentina, and France.

Is Deferasirox dispersible the same as deferasirox film-coated tablet?

Deferasirox dispersible refers to the dispersible tablet formulation of deferasirox, while the clinical trial NCT02125877 investigated an improved film-coated tablet formulation. Both contain the same active ingredient, deferasirox, but differ in formulation. The study aimed to compare the benefits of the film-coated tablet, which may offer improved convenience.