Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2012 by Ain Shams University.
Recruitment status was  Not yet recruiting
Sponsor:
Information provided by (Responsible Party):
Mohsen Saleh Elalfy, Ain Shams University
ClinicalTrials.gov Identifier:
NCT01511848
First received: January 6, 2012
Last updated: February 3, 2012
Last verified: February 2012
  Purpose

Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload

Primary Outcome Measures:

• The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator.

Secondary Outcome Measures:

• The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.


Condition Intervention Phase
Beta-thalassemia Major
Sickle Cell Disease
Iron Hemosiderosis
Drug: DFP (ferriprox) and deferasirox (ICL 670)
Drug: DFP, DFO
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Prospective Randomized Comparative Study of Efficacy and Safety of Combined Deferiprone (DFP) and Deferasirox Versus DFP and Desferrioxamine (DFO) Therapy in Diseases With Severe Iron Overload

Resource links provided by NLM:


Further study details as provided by Ain Shams University:

Primary Outcome Measures:
  • to assess efficacy of combining DFP and deferasirox compared to combined DFP and DFO in decreasing the serum ferritin level in conditions with severe chronic iron overload. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    • The primary outcome measure is to measure the change in serum ferritin level from baseline in the 2 combination therapy.


Secondary Outcome Measures:
  • to determine the safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP. [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    • The secondary outcome measure is to determine the number of patients who will develop adverse reactions upon administering the drugs in combination.


Estimated Enrollment: 60
Study Start Date: February 2012
Estimated Study Completion Date: February 2013
Estimated Primary Completion Date: February 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: arm 1
30 Patients will be treated with combined DFP and deferasirox.
Drug: DFP (ferriprox) and deferasirox (ICL 670)

Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg 3 times/d (better tolerated if started and then built up over 4 weeks).

Dose may be increased up to 100mg/kg/day guided by serum ferritin. Agranulocytosis risk 1-2%. Monitor TLC and granulocytic count / 2weeks. Patients need to be continually educated about this risk, know that they must stop DFP if they have a fever or infection.

Drug: Deferasirox will be orally administered at a dose of 20 mg/kg once daily at evening for 5 days.

Other Name: ICL670

Active Comparator: arm 2
Patients will be treated for 6 days with a combination of deferoxamine and DFP
Drug: DFP, DFO

Drug: Deferoxamine It will be administered subcutaneously over 8 hours for 5 days at a dose of 40 mg/kg.

Other Name: Desferal, DFO Drug: DFP DFP: will be orally administered at a dose of 75mg/kg orally in 3 divided doses for 7 days


  Show Detailed Description

  Eligibility

Ages Eligible for Study:   6 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects with transfusional iron overload secondary to thalassemia major , sickle cell disease showing up-trend in SF aged 6 Years or older, may participate after Approval of Ethical committee giving written informed consent.
  • Subjects must have a serum ferritin greater than >2500 ng/mL, a platelet count greater than 100,000/mm3, and a serum creatinine within the normal range.
  • A woman of childbearing potential must have a negative serum pregnancy test at screening. She must use a medically acceptable form of birth control during the study and for 1 month afterward.
  • The subjects must also have a level of understanding and willingness to cooperate with the confinement and procedures described in the consent form and scheduled by the study site. In addition, he/she must be able to provide voluntary written informed consent.

Exclusion Criteria:

  • Subjects with a past history of agranulocytosis, history of clinically significant gastrointestinal, renal, hepatic ALT > 10 times high normal, OR > 50% increase of serum creatinine from basal value, pulmonary or cardiovascular disease. Patients with a history of tuberculosis, epilepsy, psychosis, glaucoma or any other condition, which in the opinion of the investigators, would jeopardize the safety of the subject or impact the validity of the study results.
  • Subjects with HIV positive or have active HCV.
  • A history of serious immunologic hypersensitivity to any medication, such as anaphylaxis or angioedema.
  • Participation in a previous investigational drug study within the 30 days preceding screening..
  • Women who are pregnant, or breast-feeding.
  • Current alcohol or drug abuse.
  • An inability to adhere to the designated procedures and restrictions of this protocol.
  • Subjects receiving warfarin, digoxin, or anti-arrhythmic or anti-seizure medications.
  • Subjects with a known allergy to Exjade or DFP that prevents chronic administration.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01511848

Contacts
Contact: Amira A M Adly, Asst. prof. 0105245837 amiradiabetes@yahoo.com

Locations
Egypt
Pediatric Hematology clinic, Ain Shams University Not yet recruiting
Cairo, Egypt
Sponsors and Collaborators
Ain Shams University
Investigators
Principal Investigator: Mohsen S. Elalfy, professour Ain Shams University
  More Information

No publications provided

Responsible Party: Mohsen Saleh Elalfy, professour of pediatrics, Ain Shams University
ClinicalTrials.gov Identifier: NCT01511848     History of Changes
Other Study ID Numbers: AinShamsU
Study First Received: January 6, 2012
Last Updated: February 3, 2012
Health Authority: Egypt: Institutional Review Board

Keywords provided by Ain Shams University:
1- Beta-thalassemia major patients;
Iron chelation
Iron balance
Secondary iron overload
deferoxamine
deferasirox
Deferiprone
Patients with high iron stores
Serum ferritin consistently > 2500 mcg/l and or increasing trend over previous 12 months
Liver iron >14 mg/g dry weight- by R2 MRI
2- Sickle cell disease
3- Other causes of transfusional iron hemosiderosis

Additional relevant MeSH terms:
Beta-Thalassemia
Anemia, Sickle Cell
Hemosiderosis
Thalassemia
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Deferoxamine
Deferiprone
Deferasirox
Isoflurophate
Siderophores
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Cholinesterase Inhibitors
Enzyme Inhibitors
Cholinergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Protease Inhibitors

ClinicalTrials.gov processed this record on July 31, 2014