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The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease.
This is a single-arm, single-dose study of Ferriprox in patients with sickle cell disease. Patients found to be eligible will visit the clinic the day before receiving the drug, in order to reconfirm eligibility and to undergo baseline assessments, and will receive a single dose of 1500 mg Ferriprox under fasting conditions. Blood and urine samples for pharmacokinetic assessment will be collected over a 10-hour period. Standard safety assessments will be performed throughout the study, and patients will return for a safety follow-up.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ferriprox | No Intervention | single 1500 mg dose of Ferriprox |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| single 1500 mg dose of Ferriprox | Drug | A single dose of 1500mg of Ferriprox (three 500mg tablets) administered under fasting conditions |
|
| Measure | Description | Time Frame |
|---|---|---|
| Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide | Cmax (maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. | 10-hour interval |
| Tmax for Deferiprone and Deferiprone 3-O-glucuronide | Tmax (time to the maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. The results of the Tmax parameter are reported as the median and range (other parameters are reported as mean and standard deviation). | 10-hour interval |
| AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide | AUC0-∞ (area under the curve, zero to infinity) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. | 10-hour interval |
| T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide | T1/2 (apparent terminal elimination half-life) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. | 10-hour interval |
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of Adverse Events | From Day 1 (Dosing) to Day 7 plus/minus 3 days (Follow-up) | |
| Frequency of Serious Adverse Events | From Day 1 (Dosing) to Day 30 post-dose |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Denis Soulieres, MD | CHUM - Hôpital Notre-Dame | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHUM-Hôpital Notre-Dame | Montreal | Quebec | H2L 4M1 | Canada |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34981144 | Derived | Soulieres D, Mercier-Ross J, Fradette C, Rozova A, Tsang YC, Tricta F. The pharmacokinetic and safety profile of single-dose deferiprone in subjects with sickle cell disease. Ann Hematol. 2022 Mar;101(3):533-539. doi: 10.1007/s00277-021-04728-0. Epub 2022 Jan 4. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Ferriprox | A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Ferriprox | A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide | Cmax (maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. | Posted | Mean | Standard Deviation | μg/mL | 10-hour interval |
|
|
From the time of dosing to 7 ±3 days post-dose
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ferriprox | A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal Pain | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Caroline Fradette, PhD | ApoPharma Inc. | 416-401-7543 | cfradett@apopharma.com |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D019190 | Iron Overload |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D012847 | Single Person |
| D000077543 | Deferiprone |
| ID | Term |
|---|---|
| D017533 | Marital Status |
| D005191 | Family Characteristics |
| D003710 | Demography |
| D011154 | Population Characteristics |
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| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
| Secondary | Frequency of Adverse Events | Posted | Number | participants | From Day 1 (Dosing) to Day 7 plus/minus 3 days (Follow-up) |
|
|
|
| Secondary | Frequency of Serious Adverse Events | Posted | Number | participants | From Day 1 (Dosing) to Day 30 post-dose |
|
|
|
| Primary | Tmax for Deferiprone and Deferiprone 3-O-glucuronide | Tmax (time to the maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. The results of the Tmax parameter are reported as the median and range (other parameters are reported as mean and standard deviation). | Posted | Median | Full Range | hr | 10-hour interval |
|
|
|
| Primary | AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide | AUC0-∞ (area under the curve, zero to infinity) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. | Posted | Mean | Standard Deviation | µg*hr/mL | 10-hour interval |
|
|
|
| Primary | T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide | T1/2 (apparent terminal elimination half-life) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. | Posted | Mean | Standard Deviation | hr | 10-hour interval |
|
|
|
| 0 |
| 8 |
| 2 |
| 8 |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Fever | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
All unpublished information given to the PI by ApoPharma shall not be published or disclosed to a third party without the prior written consent of ApoPharma.
The data generated by this study are considered confidential information and the property of ApoPharma. This confidential information may be published only in collaboration with participating personnel from ApoPharma or upon ApoPharma written consent to publish the article.
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D012959 |
| Socioeconomic Factors |
| D011728 | Pyridones |
| D011725 | Pyridines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |