- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02198508
Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients
The Shuttle Effect : Combination Therapy With Deferiprone and Deferasirox in Transfusion-dependent Thalassemia Patients.
Background: Three iron chelators now available on the market differ in toxicity and organ specificity; evidence on standardized chelation protocol remains inconclusive, but patients with transfusion-dependent beta-thalassemia treated with DFO infusion show significant differences in the limitations of daily activities, physical activity, and quality of life when treated with oral chelator. With licensing of DFP in America, it is reasonable to combine DFP with DFX. Patients find two oral chelators more acceptable than one oral and one injectable. This pilot study rates use of DFP for improving iron excretion profile of deferasirox.
Methods: The investigators enrolled 13 beta-thalassemia patients in China Medical University Children's Hospital in May 2009-October 2011. Five refused to take part in pharmacokinetics; they only participated in iron excretion study. Seven with irregular bowel function were unable to collect feces in the screening period as baseline data. Subjects were randomly assigned and rotated to undergo all treatments (with informed consent): (A) single oral dose of DFX 30 mg/kg once daily, (B) single oral dose of DFP 40 mg/kg twice a day, (C) oral doses of DFX and DFP administered sequentially (DFX 30 mg/kg/d, deferiprone 40 mg/kg/d and deferiprone 40 mg/kg/d at 7-hour intervals). Three-day drug dosage was followed by four-day washout. Collections of urine and stool proceeded 24 hours per day, each analyzed separately. Through a venous catheter, serial blood samples (1 mL/each sampling) were collected in glass tubes containing heparin as anticoagulant at Time 0 (pre-dosing) and at 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 6, 7, 8, 10, 12 and 24 hours after dose; plasma concentrations of DFP and DFX were measured.
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Not Applicable
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 18 years of age or older
- serum ferritin greater than 2000 ng/mL,
- serum creatinine within normal range for a measuring laboratory
- platelet count exceeding 140000/mm3
- body weight at least 40 Kg
- None had a history of clinical significant of gastrointestinal, hepatic, renal, endocrine, oncologic, infectious, pulmonary or cardiovascular disease
Exclusion Criteria:
- HIV positive, history of immunologic hypersensitivity to any medication
- women pregnant or breast feeding
- drug or alcohol abuse
- patients showed abnormal or irregular bowel function (defined as more than three bowel movements a day or less than one bowel movement every other day)
- receiving warfarin, digoxin, or anti-arrhythmic or antiseizure medication.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Active Comparator: DFX single treatment
a single oral dose of DFX 30 mg/kg once daily, (Exjade®, Novartis Pharmaceuticals Corporation, USA )
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Other Names:
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Active Comparator: DFP single treatment
single oral dose of DFP 40 mg/kg/day twice a day, (Kelfer®, Cipla Ltd., India)
|
Other Names:
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Experimental: combination treatment
sequential oral doses of DFX 30 mg/kg/d, DFP 40 mg/kg/d and DFP 40 mg/kg/d (dosing interval: seven hours).
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Other Names:
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
iron excretion from urine and feces by flame atomic absorption spectroscopy
Time Frame: 25-days
|
Collections of urine and stool (made 24 hours a day) were analyzed separately.
|
25-days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
drug concentration in plasma by pharmacokinetics analysis
Time Frame: 25-day
|
Through a venous catheter, serial blood samples (1 mL/each sampling) were collected into glass tubes containing heparin as an anticoagulant at time 0 (pre-dosing) and at 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 6, 7, 8, 10, 12 and 24 hours after dosing.
Blood samples were centrifuged, with plasma collected and frozen at -20°C until analysis.
|
25-day
|
Collaborators and Investigators
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- DMR-096-IRB-037
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Beta-thalassemia Major
-
CelgeneTerminatedBeta Thalassemia Intermedia | Beta Thalassemia MajorFrance, United Kingdom, Italy, Greece
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M.D. Anderson Cancer CenterWithdrawnSickle Cell Disease | Sickle Beta Thalassemia | Beta Thalassemia Major | Sickle Cell-SS Disease | Sickle Beta 0 Thalassemia | Sickle Beta Plus ThalassemiaUnited States
-
CorrectSequence Therapeutics Co., LtdNot yet recruitingBeta-Thalassemia MajorChina
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First Affiliated Hospital of Guangxi Medical UniversityUnknown
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Editas Medicine, Inc.RecruitingHemoglobinopathies | Thalassemia Major | Thalassemia Intermedia | Transfusion Dependent Beta ThalassemiaUnited States, Canada
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Aga Khan UniversityCompletedBeta Thalassemia Major
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EmeraMedCompletedBeta Thalassemia MajorAlbania
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Tanta UniversityCompletedBeta Thalassemia MajorEgypt
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Nanfang Hospital of Southern Medical UniversityGuangdong Yike Gene Science and Technology CO.,LtdUnknownBeta Thalassemia MajorChina
-
Ain Shams UniversityUnknownBeta Thalassemia MajorEgypt
Clinical Trials on DFX(Deferasirox)
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Novartis PharmaceuticalsCompletedLow and Int 1-risk Myelodysplastic SyndromeGermany, Canada, Korea, Republic of, Sweden, Spain, China, Argentina, Italy, United Kingdom, Algeria
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Consorzio per Valutazioni Biologiche e FarmacologicheEuropean CommissionCompletedChronic Iron OverloadItaly, United Kingdom, Egypt, Greece, Cyprus, Albania, Tunisia
-
Novartis PharmaceuticalsCompletedTransfusion-dependent AnemiaEgypt, Hungary, Turkey, United States, Bulgaria, Italy, Belgium, Russian Federation, Philippines, France, Malaysia, India, Oman, Panama, Lebanon, Thailand, Tunisia
-
Novartis PharmaceuticalsCompletedNon-transfusion-dependent Thalassemia | Transfusion-dependent ThalassemiaEgypt, Turkey, Thailand, Lebanon, Morocco, Saudi Arabia, Vietnam
-
DisperSol Technologies, LLCCompletedThalassemia MajorThailand, United States
-
Novartis PharmaceuticalsWithdrawnThalassemia (Transfusion Delendent)
-
Assistance Publique - Hôpitaux de ParisAssociation pour l'Etude des Fonctions Digestives (AEFD)UnknownPorphyria Cutanea TardaFrance
-
NovartisCompletedBeta-Thalassemia | HemosiderosisEgypt, Lebanon, Oman, Saudi Arabia, Syrian Arab Republic
-
Novartis PharmaceuticalsCompletedNon-transfusion Dependent ThalassemiaThailand, Turkey, Italy, Greece, China, United Kingdom, Lebanon, Tunisia
-
City of Hope Medical CenterTerminatedPrimary Myelofibrosis | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Iron Overload | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell... and other conditionsUnited States