NCT02198508

Brief 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 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.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for not_applicable

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2007

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2008

Completed
6.1 years until next milestone

First Submitted

Initial submission to the registry

July 19, 2014

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 23, 2014

Completed
Last Updated

July 23, 2014

Status Verified

July 1, 2014

Enrollment Period

1 year

First QC Date

July 19, 2014

Last Update Submit

July 22, 2014

Conditions

Beta-thalassemia Major

Outcome Measures

Primary Outcomes (1)

  • iron excretion from urine and feces by flame atomic absorption spectroscopy

    Collections of urine and stool (made 24 hours a day) were analyzed separately.

    25-days

Secondary Outcomes (1)

  • drug concentration in plasma by pharmacokinetics analysis

    25-day

Study Arms (3)

DFX single treatment

ACTIVE COMPARATOR

a single oral dose of DFX 30 mg/kg once daily, (Exjade®, Novartis Pharmaceuticals Corporation, USA )

Drug: DFX(Deferasirox)

DFP single treatment

ACTIVE COMPARATOR

single oral dose of DFP 40 mg/kg/day twice a day, (Kelfer®, Cipla Ltd., India)

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).

Drug: DFX(Deferasirox)Drug: DFP(Deferiprone)

Interventions

DFX(Deferasirox)DRUG
Also known as: Exjade®, Novartis Pharmaceuticals Corporation, USA
DFX single treatmentcombination treatment
DFP(Deferiprone)DRUG
Also known as: Kelfer®, Cipla Ltd., India
DFP single treatmentcombination treatment

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

ThalassemiaAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

BenzoatesAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsTriazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
superintendent

Study Record Dates

First Submitted

July 19, 2014

First Posted

July 23, 2014

Study Start

July 1, 2007

Primary Completion

July 1, 2008

Last Updated

July 23, 2014

Record last verified: 2014-07