Beta-thalassemia MajorPhase 2/3NCT01511848
Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload
A Phase 2/3 study testing DFP (ferriprox) and deferasirox (ICL 670) and DFP, DFO in people with Beta-thalassemia Major and Sickle Cell Disease, run by Ain Shams University at 1 site in Egypt.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Beta-thalassemia Major, Sickle Cell Disease, Iron Hemosiderosis
- What is being tested?
- DFP (ferriprox) and deferasirox (ICL 670) and DFP, DFO
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteEgypt
- Study dates
- Feb 2012to Feb 2013
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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:
Read more
- 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.
Read the detailed description
Study population:
- Beta-thalassemia major patients; 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
- Other causes of transfusional hemosiderosis
Estimated Enrollment: 30 patients in each arm Study Start Date: January 1st ; 2011 enrollment period 8 weeks Estimated Study Completion Date: End of February 1212
Arms Assigned Interventions
Arm 1: Patients will be treated with combined DFP and deferasirox. Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg in 3 divided doses (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 Arm 2: Patients will be treated for 6 days with a combination of deferoxamine and DFP.
Interventions:
- Drug: Deferoxamine
- Drug: DFP Drug: Deferoxamine Deferoxamine 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
Eligibility Ages Eligible for Study : 6 to 18Years Genders Eligible for Study: Both
Inclusion Criteria:
- Subjects with transfusional iron overload 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 prophylaxis 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.
- Being tested
- DFP (ferriprox) and deferasirox (ICL 670), DFP, DFO
- Keywords
- Iron chelation, Iron balance, Secondary iron overload, deferoxamine, deferasirox, Deferiprone, 1- Beta-thalassemia major patients;, 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
- Medical subject headings
- beta-Thalassemia, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DFP (ferriprox) and deferasirox (ICL 670)Drug
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
DFP, DFODrug
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
| Study group | Role | What they receive |
|---|---|---|
| arm 1 | Active comparator | 30 Patients will be treated with combined DFP and deferasirox. Receives: Drug: DFP (ferriprox) and deferasirox (ICL 670) |
| arm 2 | Active comparator | Patients will be treated for 6 days with a combination of deferoxamine and DFP Receives: Drug: DFP, DFO |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
- The primary outcome measure is to measure the change in serum ferritin level from baseline in the 2 combination therapy.
Measured: 12 months
Secondary outcomes (1)
to determine the safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.
- The secondary outcome measure is to determine the number of patients who will develop adverse reactions upon administering the drugs in combination.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2012 (estimated)
- Study startFeb 2012
- Primary completionFeb 2013 (estimated)
- Study completionFeb 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Beta-thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Ain Shams UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ain Shams University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mohsen Saleh Elalfy, Ain Shams University
- Investigators
- Mohsen S. Elalfy, professour, Principal investigator, Ain Shams University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01511848
- Sponsor's protocol ID
- AinShamsU
- Status last verified
- Feb 2012
- First submitted
- 6 Jan 2012
- Last updated
- 6 Feb 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Elalfy MS, Adly AM, Wali Y, Tony S, Samir A, Elhenawy YI. Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients. Eur J Haematol. 2015 Nov;95(5):411-20. doi: 10.1111/ejh.12507. Epub 2015 Mar 27.
PubMed 25600572
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01511848
- Acronym / sponsor ID
- AinShamsU
- Keywords
- Iron chelation, Iron balance, Secondary iron overload, deferoxamine, deferasirox, Deferiprone, 1- Beta-thalassemia major patients;, Patients with high iron stores
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Ain Shams UniversityUniversity, hospital or other
- Study start
- Feb 2012
- Primary completion
- Feb 2013 (estimated)
- Study completion
- Feb 2013 (estimated)
- First posted
- 19 Jan 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study population:
- Beta-thalassemia major patients; 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
- Other causes of transfusional hemosiderosis
Estimated Enrollment: 30 patients in each arm Study Start Date: January 1st ; 2011 enrollment period 8 weeks Estimated Study Completion Date: End of February 1212
Arms Assigned Interventions
Arm 1: Patients will be treated with combined DFP and deferasirox. Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg in 3 divided doses (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 Arm 2: Patients will be treated for 6 days with a combination of deferoxamine and DFP.
Interventions:
- Drug: Deferoxamine
- Drug: DFP Drug: Deferoxamine Deferoxamine 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
Eligibility Ages Eligible for Study : 6 to 18Years Genders Eligible for Study: Both
Inclusion Criteria:
- Subjects with transfusional iron overload 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 prophylaxis 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| arm 1 | Active comparator | Drug: DFP (ferriprox) and deferasirox (ICL 670)30 Patients will be treated with combined DFP and deferasirox. |
| arm 2 | Active comparator | Drug: DFP, DFOPatients will be treated for 6 days with a combination of deferoxamine and DFP |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Drug | arm 1 |
| DFP, DFODrug: 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 | Drug | arm 2 |
Primary outcome
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
• The primary outcome measure is to measure the change in serum ferritin level from baseline in the 2 combination therapy.
1 secondary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2012.
- Derived from this study: PubMed 25600572Elalfy MS, Adly AM, Wali Y, Tony S, Samir A, Elhenawy YI. Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients. Eur J Haematol. 2015 Nov;95(5):411-20. doi: 10.1111/ejh.12507. Epub 2015 Mar 27.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mohsen S. Elalfy, professour principal investigator
Ain Shams University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing DFP (ferriprox) and deferasirox (ICL 670) and DFP, DFO in people with Beta-thalassemia Major and Sickle Cell Disease, run by Ain Shams University at 1 site in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 6 Feb 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Amira A M Adly, Asst. prof. (0105245837, amiradiabetes@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01511848, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2012.