Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
NCT ID: NCT02125877
Last Updated: 2017-07-25
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
173 participants
INTERVENTIONAL
2014-07-08
2016-02-24
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Deferasirox dispersible tablet (DFX-DT)
Iron 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 dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
Deferasirox film-coated tablet (DFX-FCT)
Participants 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
Defearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Interventions
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Deferasirox dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
Defearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision.
* History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
* Serum ferritin \> 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
Exclusion Criteria
* Serum creatinine \> 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
* ALT (SGPT) \> 5xULN, unless LIC confirmed as \>10 mg Fe/dw within 6 months prior to screening visit 1.
* Significant proteinuria as indicated by a urinary protein/creatinine ratio \> 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
* Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
* Liver disease with severity of Child-Pugh Class B or C
10 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Novartis Pharmaceuticals
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
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Children's Hospital of Orange County Onc Dept
Orange, California, United States
Lurie Children's Hospital of Chicago Onc Dept
Chicago, Illinois, United States
Children's Hospital Boston Department of Hematology
Boston, Massachusetts, United States
Weill Cornell Medical College-Cornell University Onc Dept
New York, New York, United States
Children's Hospital of Philadelphia Onc. Dept
Philadelphia, Pennsylvania, United States
Novartis Investigative Site
Buenos Aires, , Argentina
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Linz, , Austria
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Vienna, , Austria
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Lille, , France
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Paris, , France
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Mannheim, Baden-Wurttemberg, Germany
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Berlin, , Germany
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Dresden, , Germany
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Goslar, , Germany
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Hanover, , Germany
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Leipzig, , Germany
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Potsdam, , Germany
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Athens, GR, Greece
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Patra - RIO, GR, Greece
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Thessaloniki, GR, Greece
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Brindisi, BR, Italy
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Catania, CT, Italy
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Cona, FE, Italy
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Genova, GE, Italy
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Genova, GE, Italy
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Cagliari, ITA, Italy
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Lecce, LE, Italy
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Milan, MI, Italy
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Milan, MI, Italy
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Palermo, PA, Italy
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Palermo, PA, Italy
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Reggio Calabria, RC, Italy
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Verona, VR, Italy
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Napoli, , Italy
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Hazmiyeh, Beyrouth, Lebanon
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Kuala Lumpur, , Malaysia
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Pulau Pinang, , Malaysia
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Mexico City, Mexico City, Mexico
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Moscow, Russia, Russia
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Dammam, , Saudi Arabia
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Dammam, , Saudi Arabia
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Jeddah, , Saudi Arabia
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Riyadh, , Saudi Arabia
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Barcelona, , Spain
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Madrid, , Spain
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Bangkok, , Thailand
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Bangkok, , Thailand
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Al Ain - Abu Dhabi, United Arab Emirates, United Arab Emirates
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Dubai, , United Arab Emirates
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London, , United Kingdom
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London, , United Kingdom
Countries
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References
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Taher AT, Origa R, Perrotta S, Kouraklis A, Ruffo GB, Kattamis A, Goh AS, Huang V, Zia A, Herranz RM, Porter JB. Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias. Health Qual Life Outcomes. 2018 Nov 19;16(1):216. doi: 10.1186/s12955-018-1041-5.
Other Identifiers
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CICL670F2201
Identifier Type: -
Identifier Source: org_study_id
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