Iron OverloadPhase 3NCT00529152
Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients
A Phase 3 study testing Deferiprone in people with Iron Overload, run by ApoPharma at 4 sites in 3 countries.
Trial summary
The primary objective is to assess the safety of Ferriprox oral solution for the treatment of iron overload in pediatric patients with transfusion-dependent anemia. The secondary objective is to assess the efficacy of Ferriprox oral solution in reducing iron overload in pediatric patients with transfusion-dependent… Read more about this study
- Who is it for?
- Ages Up to 10 years
- Conditions the study is for
- Iron Overload
- What is being tested?
- Deferiprone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 4 sitesin 3 countries
- Study dates
- Aug 2007to Jul 2008
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Up to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients who are ≤ 10 years of age.
- Patients who have a confirmed diagnosis of transfusion-dependent anemia, other than Blackfan-Diamond anemia, and have chronic iron overload requiring chelation therapy.
- Patients who are in a chronic transfusion program, and who have received at least eight (8) red blood cell transfusions per year for a minimum of one year.
- Patients who are iron overloaded as assessed by serum ferritin concentration greater than 1000 µg/L.
Who cannot join
- Patients who have a diagnosis of Blackfan-Diamond anemia.
- Patients who have experienced neutropenia/agranulocytosis (absolute neutrophil count (ANC) < 1.5 x 109/L) or thrombocytopenia (platelet count < 50.0 x 109/L).
- Patients who have had previous treatment with Ferriprox and presented serious adverse reaction or intolerance requiring withdrawal of Ferriprox.
- Patients with evidence of abnormal liver function (ALT level > 3 times the upper limit of normal; entry may be delayed until values return to normal).
- Patients with evidence of renal failure, characterized by serum creatinine level > 2 times the upper limit of normal; entry may be delayed until values return to normal.
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
Egypt2 sites
Abo El Reish Hospital, Cairo University
Cairo
Children Hospital, Ain Shams University
Cairo
About this study
Official title: A 24-Week, Open Label, Uncontrolled Study of the Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients With Transfusion-Dependent Anemia
- The primary objective is to assess the safety of Ferriprox oral solution for the treatment of iron overload in pediatric patients with transfusion-dependent anemia.
- The secondary objective is to assess the efficacy of Ferriprox oral solution in reducing iron overload in pediatric patients with transfusion-dependent anemia.
Read the detailed description
This will be a multi-centre, open label, single treatment, uncontrolled study. A total of 100 iron-overloaded pediatric patients with transfusion-dependent anemia will be enrolled in the study.Eligible patients will receive Ferriprox (deferiprone) oral solution, 100 mg/mL, at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided in three (3) doses, for 24 weeks.
- Conditions
- Iron Overload
- Being tested
- Deferiprone
- Keywords
- Iron Overload
- Medical subject headings
- Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferiproneDrug
Also called Ferriprox Oral Solution
Ferriprox (deferiprone) oral solution will be given orally at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided into 3 doses, for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| A | Other | Ferriprox Oral Solution single treatment Receives: Drug: Deferiprone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of Adverse Events
Number of Adverse Events over 24 weeks
Measured: 24 Weeks
Secondary outcomes (1)
Change in Serum Ferritin Concentration From Baseline.
The change in serum ferritin concentration from baseline to week 24 was measured and analyzed for all participants in the study
Measured: Baseline and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2007 (estimated)
- Study startAug 2007
- Primary completionJul 2008
- Study completionJul 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Iron OverloadStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- ApoPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Prof. Mohsen S El Alfy, MD, Principal investigator, Children Hospital, Ain Shams University, Cairo, Egypt
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00529152
- Sponsor's protocol ID
- LA30-0307
- Status last verified
- Aug 2009
- First submitted
- 12 Sep 2007
- Last updated
- 2 Sep 2009
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
ElAlfy MS, Sari TT, Lee CL, Tricta F, El-Beshlawy A. The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload. J Pediatr Hematol Oncol. 2010 Nov;32(8):601-5. doi: 10.1097/MPH.0b013e3181ec0f13.
PubMed 20921906
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 24-Week, Open Label, Uncontrolled Study of the Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients With Transfusion-Dependent Anemia
- ClinicalTrials.gov ID
- NCT00529152
- Acronym / sponsor ID
- LA30-0307
- Conditions
- Iron Overload
- Keywords
- Iron Overload
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- ApoPharmaIndustry (pharma, biotech, devices)
- Study start
- Aug 2007
- Primary completion
- Jul 2008 (actual)
- Study completion
- Jul 2008 (actual)
- First posted
- 14 Sep 2007 (estimated)
- Results
- Posted 12 Aug 2009
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
- The primary objective is to assess the safety of Ferriprox oral solution for the treatment of iron overload in pediatric patients with transfusion-dependent anemia.
- The secondary objective is to assess the efficacy of Ferriprox oral solution in reducing iron overload in pediatric patients with transfusion-dependent anemia.
Detailed description
This will be a multi-centre, open label, single treatment, uncontrolled study. A total of 100 iron-overloaded pediatric patients with transfusion-dependent anemia will be enrolled in the study.Eligible patients will receive Ferriprox (deferiprone) oral solution, 100 mg/mL, at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided in three (3) doses, for 24 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | Other | Drug: DeferiproneFerriprox Oral Solution single treatment |
Who may qualify
- Ages
- Up to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Patients who are ≤ 10 years of age.
- Patients who have a confirmed diagnosis of transfusion-dependent anemia, other than Blackfan-Diamond anemia, and have chronic iron overload requiring chelation therapy.
- Patients who are in a chronic transfusion program, and who have received at least eight (8) red blood cell transfusions per year for a minimum of one year.
- Patients who are iron overloaded as assessed by serum ferritin concentration greater than 1000 µg/L.
Key exclusion criteria (5 listed)
- Patients who have a diagnosis of Blackfan-Diamond anemia.
- Patients who have experienced neutropenia/agranulocytosis (absolute neutrophil count (ANC) < 1.5 x 109/L) or thrombocytopenia (platelet count < 50.0 x 109/L).
- Patients who have had previous treatment with Ferriprox and presented serious adverse reaction or intolerance requiring withdrawal of Ferriprox.
- Patients with evidence of abnormal liver function (ALT level > 3 times the upper limit of normal; entry may be delayed until values return to normal).
- Patients with evidence of renal failure, characterized by serum creatinine level > 2 times the upper limit of normal; entry may be delayed until values return to normal.
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 |
|---|---|---|
| DeferiproneAlso: Ferriprox Oral SolutionFerriprox (deferiprone) oral solution will be given orally at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided into 3 doses, for 24 weeks. | Drug | A |
Primary outcome
Occurrence of Adverse Events
Time frame: 24 Weeks
Number of Adverse Events over 24 weeks
1 secondary outcome
Change in Serum Ferritin Concentration From Baseline.
Time frame: Baseline and 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2009.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 20921906ElAlfy MS, Sari TT, Lee CL, Tricta F, El-Beshlawy A. The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload. J Pediatr Hematol Oncol. 2010 Nov;32(8):601-5. doi: 10.1097/MPH.0b013e3181ec0f13.
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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.
Prof. Mohsen S El Alfy, MD principal investigator
Children Hospital, Ain Shams University, Cairo, Egypt
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Deferiprone in people with Iron Overload, run by ApoPharma at 4 sites in 3 countries.
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 5 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: finished, as of the sponsor's last update on 2 Sep 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Egypt, Indonesia, Malaysia. 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 phase mean?
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?
Yes. Results are posted in the registry (first posted 12 Aug 2009). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurrence of Adverse Events (time frame: 24 Weeks). 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 NCT00529152, 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 2 Sep 2009.