A Phase 4 interventional study of Deferiprone and Deferoxamine in Iron Overload, Sickle Cell Disease and Other Anemias, sponsored by ApoPharma. Terminated at 33 sites in 8 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2021-08-10.
Sponsored by ApoPharma (part of Chiesi Farmaceutici S.p.A.) · Phase 4, Interventional, and Treatment
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body. We will be comparing deferiprone with deferoxamine, another drug that removes iron from the body.
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 efficacy, safety, and tolerability of deferiprone vs. deferoxamine in patients who have SCD or other anemias, and who require chelation because of the extra iron they are taking in through blood transfusions.
About 300 people from North America, South America, Europe, and the Middle East will take part in this study. Participants will be randomized in a 2:1 ratio to receive therapy for 52 weeks with either deferiprone or deferoxamine, another type of iron chelator. Patients who are randomized to the deferiprone group can choose to get the drug as either tablets or liquid, and must take it three times daily. Patients who are randomized to the deferoxamine group will receive it as a subcutaneous infusion that lasts from 8 to 12 hours and is given 5 to 7 days per week. For both drugs, the starting dosage is based on how much extra iron they have taken in through transfusions in the last 3 months and on the severity of iron load, as measured by serum ferritin levels in the blood and by the amount of iron in the liver and the heart. For deferiprone, the starting dosage will be increased each week over the first 3 weeks; and for both drugs, the dosage may be adjusted up or down during the study based on the level of iron overload and on safety considerations.
Patients will need to have their blood count checked every week for the first 26 weeks, then every other week for the remaining 26 weeks; they will also have to give a blood sample for more detailed safety testing every month; and to give a blood sample for the measurement of serum ferritin every 3 months. Every six months, they will undergo an ECG and an MRI scan, and will be asked to complete a quality of life survey.
At the end of the 52 weeks, participants will be invited to enter a 2-year study in which all patients will receive deferiprone, including those who were randomized to receive deferoxamine in the first year.
156 studies on the registry are indexed under Iron Overload; 6 are open to participants now.
This study's enrollment of 230 is above the median of 60 across 97 interventional studies indexed under Iron Overload.
Browse Iron Overload studies →ApoPharma is the lead sponsor of 23 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients randomized to the deferiprone arm will be prescribed either tablets or liquid medication. Deferiprone is taken orally, at a dosage that is calculated in terms of milligrams per kilogram of body weight (mg/kg) and is divided into 3 equal doses taken approximately 8 hours apart. The daily dosage is 75 mg/kg (25 mg/kg per dose) for patients with less severe iron load, and 99 mg/kg (33 mg/kg per dose) for those with more severe iron load.
Drug: Deferiprone
Patients randomized to the deferoxamine arm will be prescribed the drug as per the approved US prescribing information. Deferoxamine is administered as a subcutaneous infusion over 8-12 hours, 5 to 7 days a week. The dosage is 20 mg/kg (children) or 40 mg/kg (adults) in patients with less severe iron load, and up to 40 mg/kg (children) or 50 mg/kg (adults) in those with more severe iron load.
Drug: Deferoxamine
Also known as: Ferriprox tablets, Deferiprone oral solution
Change From Baseline in Liver Iron Concentration (LIC)
LIC was measured by MRI. A score \>7 mg/g dw is indicative of iron overload.
Time frame: Change from baseline to Week 52
Change From Baseline in Cardiac Iron
Cardiac iron is measured by MRI in milliseconds (ms). A score of less than 20 ms is indicative of cardiac iron overload.
Time frame: Change from baseline to Week 52
Change From Baseline in Serum Ferritin
Serum ferritin provides a measure of iron level in the blood. Normal levels of serum ferritin are under 300 µg/L for females and 400 µg/L for males.
Time frame: Change from baseline to Week 52
Change in Patient-reported Quality of Life, as Measured by the Short Form Health Survey (SF-36) or the Child Health Questionnaire (CHQ-PF50).
Adult patients completed the SF-36 questionnaire and minors completed the CHQ-PF50. These questionnaires yield a profile of functional health and well-being, based on 8 scales of physical and mental health measures: Physical Functioning, Role Limitations due to Physical Health, Bodily Pain, General Health Perceptions, Vitality, Social Functioning, Role Limitations due to Emotional Problems, and Mental Health (MH), and summary scores are produced for physical well-being and mental well-being. The summaries are scored from 0-100, with higher scores reflecting better outcomes.
Time frame: Change from baseline to Week 52
| Milestone | Deferiprone | Deferoxamine |
|---|---|---|
| Started | 152 | 78 |
| Completed | 106 | 58 |
| Not completed | 46 | 20 |
| Withdrew: Adverse event | 9 | 3 |
| Withdrew: Lost to follow-up | 4 | 1 |
| Withdrew: Physician decision | 1 | 1 |
| Withdrew: Withdrawal by subject | 13 | 7 |
| Withdrew: Protocol violation | 11 | 4 |
| Withdrew: Termination of the study | 7 | 3 |
| Withdrew: Required bone marrow transplant | 0 | 1 |
| Withdrew: Patient stopped dosing on unknown date | 1 | 0 |
LIC was measured by MRI. A score \>7 mg/g dw is indicative of iron overload.
| mg of iron per gram of liver dry weight | Deferiprone | Deferoxamine |
|---|---|---|
| Change From Baseline in Liver Iron Concentration (LIC) | -4.13 ± 0.50 | -4.38 ± 0.59 |
Cardiac iron is measured by MRI in milliseconds (ms). A score of less than 20 ms is indicative of cardiac iron overload.
| milliseconds | Deferiprone | Deferoxamine |
|---|---|---|
| Change From Baseline in Cardiac Iron | -0.022068 ± 0.021133 | -0.021773 ± 0.025261 |
Serum ferritin provides a measure of iron level in the blood. Normal levels of serum ferritin are under 300 µg/L for females and 400 µg/L for males.
| micrograms per liter | Deferiprone | Deferoxamine |
|---|---|---|
| Change From Baseline in Serum Ferritin | -385.83 ± 234.05 | -760.89 ± 289.40 |
Adult patients completed the SF-36 questionnaire and minors completed the CHQ-PF50. These questionnaires yield a profile of functional health and well-being, based on 8 scales of physical and mental health measures: Physical Functioning, Role Limitations due to Physical Health, Bodily Pain, General Health Perceptions, Vitality, Social Functioning, Role Limitations due to Emotional Problems, and Mental Health (MH), and summary scores are produced for physical well-being and mental well-being. The summaries are scored from 0-100, with higher scores reflecting better outcomes.
| score on a scale | Deferiprone | Deferoxamine |
|---|---|---|
| SF-36 Physical Summary | 43.1 ± 1.8 | 43.0 ± 2.0 |
| SF-36 Mental Summary | 44.7 ± 2.7 | 40.9 ± 2.9 |
| CHQ-PF50 Physical Summary | 29.3 ± 1.8 | 30.5 ± 2.4 |
| CHQ-PF50 Psychosocial Summary | 42.5 ± 1.5 | 41.3 ± 2.1 |
Collected over 1 year. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Deferiprone | 1/152 (0.7%) | 40/152 (26.3%) | 134/152 (88.2%) |
| Deferoxamine | 1/76 (1.3%) | 14/76 (18.4%) | 67/76 (88.2%) |
| Event | Deferiprone | Deferoxamine |
|---|---|---|
| Sickle cell anaemia with crisisBlood and lymphatic system disorders | 16/152 | 4/76 |
| PyrexiaGeneral disorders | 5/152 | 3/76 |
| NeutropeniaBlood and lymphatic system disorders | 4/152 | 1/76 |
| VomitingGastrointestinal disorders | 1/152 | 2/76 |
| PneumoniaInfections and infestations | 2/152 | 2/76 |
| Back painMusculoskeletal and connective tissue disorders | 0/152 | 2/76 |
| Abdominal pain/upperGastrointestinal disorders | 3/152 | 1/76 |
| Acute chest syndromeRespiratory, thoracic and mediastinal disorders | 3/152 | 0/76 |
| VertigoEar and labyrinth disorders | 0/152 | 1/76 |
| DiarrhoeaGastrointestinal disorders | 1/152 | 1/76 |
| Event | Deferiprone | Deferoxamine |
|---|---|---|
| Bone painMusculoskeletal and connective tissue disorders | 38/152 | 26/76 |
| PyrexiaGeneral disorders | 40/152 | 22/76 |
| Abdominal pain/upperGastrointestinal disorders | 37/152 | 10/76 |
| HeadacheNervous system disorders | 30/152 | 10/76 |
| VomitingGastrointestinal disorders | 28/152 | 7/76 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 27/152 | 11/76 |
| Back painMusculoskeletal and connective tissue disorders | 20/152 | 13/76 |
| CoughRespiratory, thoracic and mediastinal disorders | 12/152 | 11/76 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 15/152 | 11/76 |
| Sickle cell anaemia with crisisBlood and lymphatic system disorders | 21/152 | 9/76 |
Number of patients who received study product
| Age, Continuous(years) | Deferiprone | Deferoxamine | Total |
|---|---|---|---|
| Mean | 16.9 ± 10.2 | 16.9 ± 8.5 | 16.9 ± 9.6 |
| Sex: Female, Male(Participants) | Deferiprone | Deferoxamine | Total |
|---|---|---|---|
| Female | 69 | 38 | 107 |
| Male | 83 | 38 | 121 |
| Ethnicity (NIH/OMB)(Participants) | Deferiprone | Deferoxamine | Total |
|---|---|---|---|
| Hispanic or Latino | 10 | 6 | 16 |
| Not Hispanic or Latino | 142 | 70 | 212 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Deferiprone | Deferoxamine | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 23 | 14 | 37 |
| White | 120 | 56 | 176 |
| More than one race | 9 | 6 | 15 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(Participants) | Deferiprone | Deferoxamine | Total |
|---|---|---|---|
| Saudi Arabia | 8 | 3 | 11 |
| Canada | 1 | 1 | 2 |
| Turkey | 1 | 1 | 2 |
| United States | 16 | 7 | 23 |
| Egypt | 105 | 51 | 156 |
| Brazil | 9 | 7 | 16 |
| United Kingdom | 6 | 5 | 11 |
| Tunisia | 6 | 1 | 7 |
| Liver iron concentration at baseline(mg iron per gram of liver dry weight) | Deferiprone | Deferoxamine | Total |
|---|---|---|---|
| Mean | 16.54 ± 7.49 | 15.97 ± 7.20 | 16.26 ± 7.30 |
| Cardiac iron at baseline(milliseconds) | Deferiprone | Deferoxamine | Total |
|---|---|---|---|
| Mean | 33.52 ± 6.26 | 33.87 ± 6.17 | 33.64 ± 6.22 |
| Serum ferritin level at baseline(micrograms per liter) | Deferiprone | Deferoxamine | Total |
|---|---|---|---|
| Mean | 4125.4 ± 2407.4 | 4055.3 ± 2708.5 | 4101.89 ± 2505.66 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is terminated, as verified in Jul 2021. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Deferiprone