NCT00350662

Brief Summary

Comparison of efficacy and toxicity of the combination treatment of deferiprone and desferrioxamine with the single agent treatment of either drug

Trial Health

85
On Track

Trial Health Score

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

Enrollment
95

participants targeted

Target at P25-P50 for phase_3

Geographic Reach
2 countries

2 active sites

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

January 1, 2002

Completed
4.5 years until next milestone

First Submitted

Initial submission to the registry

July 10, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 11, 2006

Completed
Last Updated

November 19, 2012

Status Verified

November 1, 2012

First QC Date

July 10, 2006

Last Update Submit

November 16, 2012

Conditions

Hemochromatosis

Keywords

DeferiproneL1DesferrioxamineHemochromatosisIron overloadThalassemia

Outcome Measures

Primary Outcomes (1)

  • Clinical efficacy (Iron balance and liver iron concentration)

    At baseline and at 12 months

Secondary Outcomes (4)

  • Safety profile (general, hematologic, and organ toxicity)

    At 3-monthly intervals

  • Liver histology

    At baseline and at 12 months

  • Quality of life (patient's subjective of compliance and tolerance)

    At 3-monthly intervals

  • Actual treatment duration (ATD)

    At 12 months

Study Arms (3)

Deferiprone + Desferrioxamine

EXPERIMENTAL
Drug: Deferiprone (L1)Drug: Desferrioxamine

Deferiprone single agent

EXPERIMENTAL
Drug: Deferiprone (L1)

Desferrioxamine single agent

ACTIVE COMPARATOR
Drug: Desferrioxamine

Interventions

Deferiprone (L1)DRUG

75 mg/kg body weight daily

Deferiprone + DesferrioxamineDeferiprone single agent
DesferrioxamineDRUG

In combination with deferiprone: 40-50 mg/kg body weight 2-times weekly As single agent: 40-50 mg/kg body weight 5- to 7-times weekly

Deferiprone + DesferrioxamineDesferrioxamine single agent

Eligibility Criteria

Age4 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Iron-overloaded patients without prior iron chelation therapy as well as pretreated patients
  • Age: 4 years and older
  • Sex: male and female
  • Written informed consent

You may not qualify if:

  • Children \< 4 years of age
  • Patients non-compliant to DFO or L1
  • Patients with known DFO or L1 toxicity/intolerance
  • Neutropenia (neutrophils \< 1.5 x 10exp9/L)
  • Thrombocytopenia (platelets \< 100 x 10exp9/L)
  • Renal, hepatic (liver enzymes 2.5x of upper normal level and higher) or decompensated heart failure
  • Active viral illness currently treated with interferon-alpha/ribavirin
  • Patients with repeated Yersinia infections
  • HIV-positivity
  • Pregnancy and nursing
  • Female and male of reproductive age planning for family, sexually active but not taking adequate contraceptive precaution

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Pediatric Hospital, Cairo University

Cairo, Egypt

Location

EGE University Medical School

Bornova, Izmir, İzmir, 35100, Turkey (Türkiye)

Location

Related Publications (3)

  • El-Beshlawy A, Manz C, Naja M, Eltagui M, Tarabishi C, Youssry I, Sobh H, Hamdy M, Sharaf I, Mostafa A, Shaker O, Hoffbrand AV, Taher A. Iron chelation in thalassemia: combined or monotherapy? The Egyptian experience. Ann Hematol. 2008 Jul;87(7):545-50. doi: 10.1007/s00277-008-0471-2. Epub 2008 Mar 20.

    PMID: 18351337RESULT

  • Aydinok Y, Ulger Z, Nart D, Terzi A, Cetiner N, Ellis G, Zimmermann A, Manz C. A randomized controlled 1-year study of daily deferiprone plus twice weekly desferrioxamine compared with daily deferiprone monotherapy in patients with thalassemia major. Haematologica. 2007 Dec;92(12):1599-606. doi: 10.3324/haematol.11414.

    PMID: 18055982RESULT

  • Aydinok Y, Evans P, Manz CY, Porter JB. Timed non-transferrin bound iron determinations probe the origin of chelatable iron pools during deferiprone regimens and predict chelation response. Haematologica. 2012 Jun;97(6):835-41. doi: 10.3324/haematol.2011.056317. Epub 2011 Dec 16.

    PMID: 22180427RESULT

MeSH Terms

Conditions

HemochromatosisIron OverloadThalassemia

Interventions

DeferiproneLong Interspersed Nucleotide ElementsDeferoxamine

Condition Hierarchy (Ancestors)

Metal Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesIron Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathies

Intervention Hierarchy (Ancestors)

PyridonesPyridinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsRetroelementsInterspersed Repetitive SequencesRepetitive Sequences, Nucleic AcidBase SequenceMolecular StructureBiochemical PhenomenaChemical PhenomenaGenetic StructuresGenetic PhenomenaGenome ComponentsGenomeHydroxamic AcidsHydroxylaminesAminesOrganic ChemicalsHydroxy AcidsCarboxylic Acids

Study Officials

  • Amal M El-Beshlawy, Prof. Dr.

    Pediatric Hospital, Cairo University, Cairo, Egypt

    PRINCIPAL INVESTIGATOR

  • Yesim Aydinok, Prof. Dr.

    EGE University Medical School Bornova, Izmir, Turkey

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 10, 2006

First Posted

July 11, 2006

Study Start

January 1, 2002

Last Updated

November 19, 2012

Record last verified: 2012-11

Locations

TU(1)EG(1)