Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload
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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 |
- 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.
- 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 |
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| Contact: Amira A M Adly, Asst. prof. | 0105245837 | amiradiabetes@yahoo.com |
| Egypt | |
| Pediatric Hematology clinic, Ain Shams University | Not yet recruiting |
| Cairo, Egypt | |
| 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:
|
Serum ferritin consistently > 2500 mcg/l and or increasing trend over previous 12 months Iron chelation Iron balance Secondary iron overload deferoxamine deferasirox |
Deferiprone 1- Beta-thalassemia major patients; Patients with high iron stores 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 Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Protease Inhibitors Enzyme Inhibitors Cholinesterase Inhibitors Cholinergic Agents Neurotransmitter Agents Physiological Effects of Drugs |
ClinicalTrials.gov processed this record on June 17, 2013