Iron OverloadPhase 4NCT02443545
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
A Phase 4 study testing Deferiprone in people with Iron Overload and Sickle Cell Disease, run by ApoPharma at 13 sites in 5 countries.
Trial summary
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Iron Overload, Sickle Cell Disease, Other Anemias
- 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
- 134 participants
- Where
- 13 sitesin 5 countries
- Study dates
- 21 May 2015to 21 Aug 2019
- Phase
- Phase 4
- 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completed study LA38-0411
- Females of childbearing potential must have a negative pregnancy test result at Visit 1. In addition, if applicable, they must:
- Use an effective method of contraception according to local requirements, during the study and within 30 days following their last dose of study medication, OR
- Have had a tubal ligation (supporting evidence required), OR
- Have had a hysterectomy (supporting evidence required), OR
- Participate in a non-heterosexual lifestyle, OR
Show all 9Show fewer
- Have a male sexual partner who has been sterilized (supporting evidence required)
- Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication
- All patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and assent will be obtained from patients who are considered to be minors. Patients must be able to adhere to study restrictions, appointments, and evaluation schedules.
Who cannot join
- Plan to participate in another clinical trial at any time from the day of enrollment until 30 days post-treatment in the current study
- For only those patients who were treated with deferoxamine in study LA38-0411 (Group 2): Presence of any medical condition (including clinically significant laboratory abnormalities, such as ALT (alanine aminotransferase) ≥ 5 x ULN or creatinine ≥ 2 x ULN), psychological condition, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise.
- Pregnant, breastfeeding, or planning to become pregnant during the study period.
- Treatment failure after 1 year on deferiprone which in the investigator's judgment indicates the need for the patient to be started on a different iron chelator
Trial locations and contacts
13 sites in 5 countries.
13 sites in 5 countries
United States5 sites
University of Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, 48109
Medical University of South Carolina
Charleston, South Carolina, 29425
Children's Hospital of Michigan
Detroit, Michigan, 48201
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
The Children's Hospital of Philadephia
Philadelphia, Pennsylvania, 19104-4399
Egypt4 sites
Zagazig University
Alexandria
Ain Shams University
Cairo
Cairo University
Cairo
Pediatric Hospital of Cairo University
Cairo
United Kingdom2 sites
Barts and The London
London
Evelina Children's Hospital
London
About this study
Official title: Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.
Read the detailed description
Deferiprone (brand name Ferriprox®) is an iron chelator that is approved in the United States and over 60 other countries for the treatment of iron overload in patients with thalassemia, when other treatments are inadequate. This study has been designed to evaluate the long-term efficacy, safety, and tolerability of deferiprone to treat iron overload in patients who have sickle cell disease or other anemias.
Only patients who have completed an earlier study, LA38-0411, are eligible to enroll in this one.
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Being tested
- Deferiprone
- Keywords
- Iron overload, Sickle cell disease, Deferiprone, Ferriprox, Iron chelation
- Medical subject headings
- Iron Overload, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferiproneDrug
Also called Ferriprox tablets, Ferriprox oral solution
| Study group | Role | What they receive |
|---|---|---|
| Group 1: Deferiprone 3 years | Experimental | Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411, and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study). Receives: Drug: Deferiprone |
| Group 2: Deferiprone 2 years | Experimental | Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411, and hence will receive deferiprone for 2 years (both of them in the extension study). Receives: Drug: Deferiprone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With Adverse Events
Number of patients with at least one adverse event (AE) of any type; number of patients with at least one serious adverse event, and number of patients who withdrew from the study due to an AE
Measured: From the first day of the study until the last study visit (Week 104 or early termination)
Secondary outcomes (3)
Change From Baseline in Liver Iron Concentration (LIC)
LIC was measured by MRI, in units of mg of iron per gram of liver (dry weight). The change from baseline in LIC was determined for three different periods of exposure to deferiprone: one year, two years, and three years.
Measured: One year, two years, and three years after the start of deferiprone therapy
Change From Baseline in Cardiac MRI T2*
The change from baseline in cardiac MRI T2* was determined for three different periods of exposure to deferiprone: one year, two years, and three years
Measured: One year, two years, and three years after the start of deferiprone therapy
Change From Baseline in Serum Ferritin
The change from baseline in serum ferritin (SF) was determined for three different periods of exposure to deferiprone: one year, two years, and three years.
Measured: One year, two years, and three years after the start of deferiprone therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2015 (estimated)
- Study start21 May 2015
- Primary completion30 Apr 2019
- Study completion21 Aug 2019
Why it stopped: Recommendation by DSMB, which felt that sufficient data had been obtained
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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 134 participants (actual)
Sponsor and investigators
- Lead sponsor
- ApoPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Janet Kwiatkowski, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02443545
- Sponsor's protocol ID
- LA38-EXT
- Status last verified
- Dec 2023
- First submitted
- 5 May 2015
- Last updated
- 10 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Elalfy MS, Hamdy M, El-Beshlawy A, Ebeid FSE, Badr M, Kanter J, Inusa B, Adly AAM, Williams S, Kilinc Y, Lee D, Fradette C, Rozova A, Temin NT, Tricta F, Kwiatkowski JL. Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years. Blood Adv. 2023 Feb 28;7(4):611-619. doi: 10.1182/bloodadvances.2021006778.
PubMed 36018224
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias
- ClinicalTrials.gov ID
- NCT02443545
- Acronym / sponsor ID
- LA38-EXT
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Keywords
- Iron overload, Sickle cell disease, Deferiprone, Ferriprox, Iron chelation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Recommendation by DSMB, which felt that sufficient data had been obtained
- Enrolment
- 134 participants (actual)
- Lead sponsor
- ApoPharmaIndustry (pharma, biotech, devices)
- Study start
- 21 May 2015 (actual)
- Primary completion
- 30 Apr 2019 (actual)
- Study completion
- 21 Aug 2019 (actual)
- First posted
- 14 May 2015 (estimated)
- Results
- Posted 10 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.
Detailed description
Deferiprone (brand name Ferriprox®) is an iron chelator that is approved in the United States and over 60 other countries for the treatment of iron overload in patients with thalassemia, when other treatments are inadequate. This study has been designed to evaluate the long-term efficacy, safety, and tolerability of deferiprone to treat iron overload in patients who have sickle cell disease or other anemias.
Only patients who have completed an earlier study, LA38-0411, are eligible to enroll in this one.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1: Deferiprone 3 years | Experimental | Drug: DeferipronePatients in this group are those who were randomized to the deferiprone arm in study LA38-0411, and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study). |
| Group 2: Deferiprone 2 years | Experimental | Drug: DeferipronePatients in this group are those who were randomized to the deferoxamine arm in study LA38-0411, and hence will receive deferiprone for 2 years (both of them in the extension study). |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Completed study LA38-0411
- Females of childbearing potential must have a negative pregnancy test result at Visit 1. In addition, if applicable, they must:
- Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication
- All patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and assent will be obtained from patients who are considered to be minors. Patients must be able to adhere to study restrictions, appointments, and evaluation schedules.
Key exclusion criteria (4 listed)
- Plan to participate in another clinical trial at any time from the day of enrollment until 30 days post-treatment in the current study
- For only those patients who were treated with deferoxamine in study LA38-0411 (Group 2): Presence of any medical condition (including clinically significant laboratory abnormalities, such as ALT (alanine aminotransferase) ≥ 5 x ULN or creatinine ≥ 2 x ULN), psychological condition, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise.
- Pregnant, breastfeeding, or planning to become pregnant during the study period.
- Treatment failure after 1 year on deferiprone which in the investigator's judgment indicates the need for the patient to be started on a different iron chelator
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 tablets, Ferriprox oral solution | Drug | Group 1: Deferiprone 3 years, Group 2: Deferiprone 2 years |
Primary outcome
Number of Patients With Adverse Events
Time frame: From the first day of the study until the last study visit (Week 104 or early termination)
Number of patients with at least one adverse event (AE) of any type; number of patients with at least one serious adverse event, and number of patients who withdrew from the study due to an AE
3 secondary outcomes
Change From Baseline in Liver Iron Concentration (LIC)
Time frame: One year, two years, and three years after the start of deferiprone therapy
Change From Baseline in Cardiac MRI T2*
Time frame: One year, two years, and three years after the start of deferiprone therapy
Change From Baseline in Serum Ferritin
Time frame: One year, two years, and three years after the start of deferiprone therapy
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36018224Elalfy MS, Hamdy M, El-Beshlawy A, Ebeid FSE, Badr M, Kanter J, Inusa B, Adly AAM, Williams S, Kilinc Y, Lee D, Fradette C, Rozova A, Temin NT, Tricta F, Kwiatkowski JL. Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years. Blood Adv. 2023 Feb 28;7(4):611-619. doi: 10.1182/bloodadvances.2021006778.
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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.
Janet Kwiatkowski, MD principal investigator
Children's Hospital of Philadelphia
13 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Deferiprone in people with Iron Overload and Sickle Cell Disease, run by ApoPharma at 13 sites in 5 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 9 inclusion and 4 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: stopped early, as of the sponsor's last update on 10 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Egypt, United Kingdom, Canada, Saudi Arabia. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 10 Jan 2024). 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?
Number of Patients With Adverse Events (time frame: From the first day of the study until the last study visit (Week 104 or early termination)). 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 NCT02443545, 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 10 Jan 2024.