A Phase 2 interventional study of Deferiprone DR tablets 1000 mg (Low dosage) and Deferiprone DR tablets 1000 mg (High dosage) in Iron Overload Due to Repeated Red Blood Cell Transfusions, sponsored by ApoPharma. Completed at 5 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-07-16.
Sponsored by ApoPharma (part of Chiesi Farmaceutici S.p.A.) · Phase 2, Interventional, and Treatment
Safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload.
This study is looking at the safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload who are currently taking deferiprone immediate-release tablets (Ferriprox) three times a day. Ferriprox doses range from 75 milligrams per kilogram of body weight (mg/kg) per day to 100 mg/kg per day. Half the patients in the study will be on a dosage that is closer to the low end of the range, and half will be on a dosage that is closer to the high end. Both groups will be switched for one month to deferiprone DR tablets at approximately the same total daily dosage that they have been taking for Ferriprox.
156 studies on the registry are indexed under Iron Overload; 6 are open to participants now.
This study's enrollment of 30 is below 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 number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
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.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
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.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 75 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.
Drug: Deferiprone DR tablets 1000 mg (Low dosage)
Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 100 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.
Drug: Deferiprone DR tablets 1000 mg (High dosage)
Deferiprone DR tablets 1000 mg
Deferiprone DR tablets 1000 mg
The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.
Levels of the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern. The criteria for being considered a safety concern are meeting one of the following: * For a patient whose level was within the normal range at baseline, the criterion is reaching a value of 5 times the upper limit of normal (ULN) * For a patient whose level was above the ULN at baseline, the criterion is reaching either 5 times the baseline value or 10 x ULN
Time frame: Day 28
The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress.
Patients will be asked to report any events of GI distress during the study, such as nausea, vomiting, diarrhea, abdominal pain, and dyspepsia.
Time frame: Day 28
The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation.
At the end of the study, patients will complete a questionnaire to indicate which formulation they prefer.
Time frame: Day 28
| Milestone | Low Dosage | High Dosage |
|---|---|---|
| Started | 15 | 15 |
| Completed | 15 | 13 |
| Not completed | 0 | 2 |
| Withdrew: Adverse event | 0 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 |
Levels of the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern. The criteria for being considered a safety concern are meeting one of the following: * For a patient whose level was within the normal range at baseline, the criterion is reaching a value of 5 times the upper limit of normal (ULN) * For a patient whose level was above the ULN at baseline, the criterion is reaching either 5 times the baseline value or 10 x ULN
| Participants | Low Dosage | High Dosage |
|---|---|---|
| Patients with elevated ALT of clinical concern | 0 | 0 |
| Patients with elevated AST of clinical concern | 0 | 0 |
Patients will be asked to report any events of GI distress during the study, such as nausea, vomiting, diarrhea, abdominal pain, and dyspepsia.
| Participants | Low Dosage | High Dosage |
|---|---|---|
| The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress. | 3 | 3 |
At the end of the study, patients will complete a questionnaire to indicate which formulation they prefer.
| Participants | Low Dosage | High Dosage |
|---|---|---|
| The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation. | 13 | 13 |
Collected over Baseline to Day 28. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Low Dosage | 0/15 (0%) | 0/15 (0%) | 10/15 (66.7%) |
| High Dosage | 0/14 (0%) | 0/14 (0%) | 9/14 (64.3%) |
| Event | Low Dosage | High Dosage |
|---|---|---|
| DiarrhoeaGastrointestinal disorders | 0/15 | 3/14 |
| HeadacheNervous system disorders | 3/15 | 3/14 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 3/15 | 1/14 |
| BlepharitisEye disorders | 0/15 | 2/14 |
| Joint injuryInjury, poisoning and procedural complications | 2/15 | 0/14 |
| Ear painEar and labyrinth disorders | 1/15 | 1/14 |
| PyrexiaGeneral disorders | 1/15 | 1/14 |
| ConjunctivitisInfections and infestations | 0/15 | 1/14 |
| PharyngitisInfections and infestations | 0/15 | 1/14 |
| Alanine aminotransferase increasedInvestigations | 0/15 | 1/14 |
One patient in the high-dosage group withdrew before receiving any study product
| Age, Continuous(years) | Low Dosage | High Dosage | Total |
|---|---|---|---|
| Mean | 41.9 ± 9.9 | 40.4 ± 6.8 | 41.1 ± 8.4 |
| Sex: Female, Male(Participants) | Low Dosage | High Dosage | Total |
|---|---|---|---|
| Female | 6 | 7 | 13 |
| Male | 9 | 7 | 16 |
| Ethnicity (NIH/OMB)(Participants) | Low Dosage | High Dosage | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 1 | 1 |
| Not Hispanic or Latino | 15 | 13 | 28 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Low Dosage | High Dosage | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 2 | 0 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 13 | 13 | 26 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 1 | 1 |
| Region of Enrollment(participants) | Low Dosage | High Dosage | Total |
|---|---|---|---|
| Greece | 3 | 5 | 8 |
| Canada | 3 | 0 | 3 |
| United States | 1 | 2 | 3 |
| Italy | 8 | 7 | 16 |
| Level of liver enzymes at baseline for low-dosage group(units per liter) | Low Dosage | High Dosage | Total |
|---|---|---|---|
| Baseline ALT | 29.60 ± 19.88 | — | 29.60 ± 19.88 |
| Baseline AST | 27.53 ± 10.00 | — | 27.53 ± 10.00 |
| Level of liver enzymes at baseline for high-dosage group(units per liter) | Low Dosage | High Dosage | Total |
|---|---|---|---|
| Baseline ALT | — | 38.29 ± 22.43 | 38.29 ± 22.43 |
| Baseline AST | — | 28.50 ± 11.39 | 28.50 ± 11.39 |
PDFs hosted by the registry, ClinicalTrials.gov.
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