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

Study point of contact

Amira A M Adly, Asst. prof.
0105245837
[email protected]

Locations

1 Egypt site

Age

6 to 18 Years

Phase

Phase 2/Phase 3

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

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.

participation requirements

– 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.

participation restrictions

– 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.

Locations

  • Cairo, Egypt, Pediatric Hematology clinic, Ain Shams University