Beta-thalassemia MajorNCT02198508
Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients
A study testing DFX(Deferasirox) and DFP(Deferiprone) in people with Beta-thalassemia Major, run by China Medical University Hospital.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta-thalassemia Major
- What is being tested?
- DFX(Deferasirox) and DFP(Deferiprone)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- No sites listed yet
- Study dates
- Jul 2007to Jul 2008
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
About this study
Official title: 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.
- Conditions
- Beta-thalassemia Major
- Being tested
- DFX(Deferasirox), DFP(Deferiprone)
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DFX(Deferasirox)Drug
Also called Exjade®, Novartis Pharmaceuticals Corporation, USA
DFP(Deferiprone)Drug
Also called Kelfer®, Cipla Ltd., India
| Study group | Role | What they receive |
|---|---|---|
| DFX single treatment | Active comparator | a single oral dose of DFX 30 mg/kg once daily, (Exjade®, Novartis Pharmaceuticals Corporation, USA ) Receives: Drug: DFX(Deferasirox) |
| DFP single treatment | Active comparator | single oral dose of DFP 40 mg/kg/day twice a day, (Kelfer®, Cipla Ltd., India) Receives: Drug: DFP(Deferiprone) |
| combination treatment | Experimental | sequential oral doses of DFX 30 mg/kg/d, DFP 40 mg/kg/d and DFP 40 mg/kg/d (dosing interval: seven hours). Receives: Drug: DFX(Deferasirox); Drug: DFP(Deferiprone) |
What is being measured
The primary outcome is the main question the study is designed to answer.
iron excretion from urine and feces by flame atomic absorption spectroscopy
Collections of urine and stool (made 24 hours a day) were analyzed separately.
Measured: 25-days
Secondary outcomes (1)
drug concentration in plasma by pharmacokinetics analysis
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.
Measured: 25-day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2014 (estimated)
- Study startJul 2007
- Primary completionJul 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-thalassemia MajorStudies of the same condition, open and under way.
- More trials run by China Medical University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- China Medical University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ching-Tien Peng, China Medical University, Taiwan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02198508
- Sponsor's protocol ID
- DMR-096-IRB-037
- Status last verified
- Jul 2014
- First submitted
- 19 Jul 2014
- Last updated
- 23 Jul 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Shuttle Effect : Combination Therapy With Deferiprone and Deferasirox in Transfusion-dependent Thalassemia Patients.
- ClinicalTrials.gov ID
- NCT02198508
- Acronym / sponsor ID
- DMR-096-IRB-037
- Conditions
- Beta-thalassemia Major
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- China Medical University HospitalUniversity, hospital or other
- Study start
- Jul 2007
- Primary completion
- Jul 2008 (actual)
- First posted
- 23 Jul 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| DFX single treatment | Active comparator | Drug: DFX(Deferasirox)a single oral dose of DFX 30 mg/kg once daily, (Exjade®, Novartis Pharmaceuticals Corporation, USA ) |
| DFP single treatment | Active comparator | Drug: DFP(Deferiprone)single oral dose of DFP 40 mg/kg/day twice a day, (Kelfer®, Cipla Ltd., India) |
| combination treatment | Experimental | Drug: DFX(Deferasirox), Drug: DFP(Deferiprone)sequential oral doses of DFX 30 mg/kg/d, DFP 40 mg/kg/d and DFP 40 mg/kg/d (dosing interval: seven hours). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DFX(Deferasirox)Also: Exjade®, Novartis Pharmaceuticals Corporation, USA | Drug | DFX single treatment, combination treatment |
| DFP(Deferiprone)Also: Kelfer®, Cipla Ltd., India | Drug | DFP single treatment, combination treatment |
Primary outcome
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.
1 secondary outcome
drug concentration in plasma by pharmacokinetics analysis
Time frame: 25-day
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2014.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing DFX(Deferasirox) and DFP(Deferiprone) in people with Beta-thalassemia Major, run by China Medical University Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
iron excretion from urine and feces by flame atomic absorption spectroscopy (time frame: 25-days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jul 2014.