A Phase 4 interventional study of Deferiprone in Iron Overload, Sickle Cell Disease and Other Anemias, sponsored by ApoPharma. Terminated at 13 sites in 5 countries. Open to participants aged 3 Years and older. Per ClinicalTrials.gov, last updated 2024-01-10.
Sponsored by ApoPharma (part of Chiesi Farmaceutici S.p.A.) · Phase 4, Interventional, and Treatment
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.
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.
156 studies on the registry are indexed under Iron Overload; 6 are open to participants now.
This study's enrollment of 134 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.
Females of must have a negative pregnancy test result at Visit 1. In addition, if applicable, they must:
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.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
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.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
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.
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).
Drug: Deferiprone
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).
Drug: Deferiprone
Also known as: Ferriprox tablets, Ferriprox oral solution
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
Time frame: From the first day of the study until the last study visit (Week 104 or early termination)
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.
Time frame: 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
Time frame: 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.
Time frame: One year, two years, and three years after the start of deferiprone therapy
| Milestone | Group 1: Deferiprone 3 Years | Group 2: Deferiprone 2 Years |
|---|---|---|
| Started | 89 | 45 |
| Completed | 50 | 26 |
| Not completed | 39 | 19 |
| Withdrew: Adverse event | 1 | 1 |
| Withdrew: Lost to follow-up | 0 | 2 |
| Withdrew: Physician decision | 3 | 0 |
| Withdrew: Protocol violation | 8 | 1 |
| Withdrew: Pregnancy | 1 | 1 |
| Withdrew: Withdrawal by subject | 6 | 3 |
| Withdrew: Termination of study | 20 | 11 |
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
| Participants | Deferiprone |
|---|---|
| Number of patients with any type of adverse event | 104 |
| Number of patients with a serious adverse event | 35 |
| Number of patients who discontinued due to an AE | 2 |
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.
| mg of iron per gram of liver dry weight | Deferiprone |
|---|---|
| Change from baseline in LIC after 1 year | -2.64 ± 4.64 |
| Change from baseline in LIC after 2 years | -3.91 ± 6.38 |
| Change from baseline in LIC after 3 years | -6.64 ± 7.72 |
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
| milliseconds | Deferiprone |
|---|---|
| Change from baseline in cardiac iron after 1 year | 1.02 ± 19.65 |
| Change from baseline in cardiac iron after 2 years | 1.00 ± 21.18 |
| Change from baseline in cardiac iron after 3 years | 0.98 ± 24.53 |
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.
| micrograms per liter | Deferiprone |
|---|---|
| Change from baseline in SF after 1 year | -1 ± 1986 |
| Change from baseline in SF after 2 years | -771 ± 2171 |
| Change from baseline in SF after 3 years | -1016 ± 3617 |
Collected over Baseline to end of study (up to 2 years). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Deferiprone | 1/134 (0.7%) | 35/134 (26.1%) | 99/134 (73.9%) |
| Event | Deferiprone |
|---|---|
| Sickle cell anaemia with crisisBlood and lymphatic system disorders | 19/134 |
| NeutropeniaBlood and lymphatic system disorders | 12/134 |
| PyrexiaGeneral disorders | 5/134 |
| CholecystectomySurgical and medical procedures | 3/134 |
| AgranulocytosisBlood and lymphatic system disorders | 2/134 |
| PneumoniaInfections and infestations | 2/134 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 2/134 |
| SplenectomySurgical and medical procedures | 2/134 |
| HypotensionVascular disorders | 2/134 |
| ThrombocytopeniaBlood and lymphatic system disorders | 1/134 |
| Event | Deferiprone |
|---|---|
| Bone painMusculoskeletal and connective tissue disorders | 35/134 |
| PyrexiaGeneral disorders | 33/134 |
| Abdominal painGastrointestinal disorders | 26/134 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 18/134 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 18/134 |
| Sickle cell anaemia with crisisBlood and lymphatic system disorders | 17/134 |
| NasopharyngitisInfections and infestations | 17/134 |
| Back painMusculoskeletal and connective tissue disorders | 17/134 |
| Neutrophil count decreasedInvestigations | 16/134 |
| HeadacheNervous system disorders | 12/134 |
| Age, Continuous(years) | Deferiprone |
|---|---|
| Mean | 16.2 ± 8.6 |
| Sex: Female, Male(Participants) | Deferiprone |
|---|---|
| Female | 53 |
| Male | 81 |
| Ethnicity (NIH/OMB)(Participants) | Deferiprone |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 133 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Deferiprone |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 20 |
| White | 114 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Deferiprone |
|---|---|
| Saudi Arabia | 5 |
| Canada | 2 |
| United States | 14 |
| Egypt | 109 |
| United Kingdom | 4 |
| Liver iron concentration at baseline(mg iron per gram of liver dry weight) | Deferiprone |
|---|---|
| Mean | 14.93 ± 7.61 |
| Cardiac iron at baseline(milliseconds) | Deferiprone |
|---|---|
| Mean | 32.69 ± 17.66 |
| Serum ferritin level at baseline(micrograms per liter) | Deferiprone |
|---|---|
| Mean | 3894 ± 2591 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is terminated, as verified in Dec 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Deferiprone