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An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry
Following the provision of informed consent, screening visit procedures to be performed will include: a detailed medical history (including concomitant medications), physical exam, vital signs (non-invasive systolic and diastolic arterial blood pressure, heart rate, and respiratory rate, temperature, and oxygen saturation by pulse oximetry,), laboratory testing of blood samples collections for safety (hematology and chemistry), and urinalysis (dipstick and microscopy, if necessary). A serum pregnancy test for all female patients (of child-bearing potential) will be measured during the Screening Visit. Urine pregnancy test for all female patients (of child-bearing potential) will be measured at each dosing visit prior to dosing.
Eligible patients will receive either subcutaneous injection of EPEG (0.9 µg/kg, 1.2 µg/kg, and 1.5 µg/kg,) for four weeks followed by follow up for 5-6 weeks after 4th dose of IP.
Vital signs will be recorded for study documentation at 1 hour after dosing and at discharge of the day (to occur 2 hours after the time of dosing). All patients will receive standard of care as per investigative site standard practice.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| EPEG (pegylated erythropoietin) - 0.9 µg/kg | Experimental | Four weekly subcutaneous injections of 0.9 µg/kg EPEG |
|
| EPEG (pegylated erythropoietin) - 1.2 µg/kg | Experimental | Four weekly subcutaneous injections of 1.2 µg/kg EPEG |
|
| EPEG (pegylated erythropoietin) - 1.5 µg/kg | Experimental | Four weekly subcutaneous injections of 1.5 µg/kg EPEG |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EPEG | Drug | (Pegylated erythropoeitin) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants in each treatment arm with abnormal laboratory values and/or adverse events that are related to treatment. | Safety of treatment as determined by changes in vital signs, chest X-Ray, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events | 60 days |
| Measure | Description | Time Frame |
|---|---|---|
| Change in the Hemoglobin from Baseline to the Final Visit | 60 Days | |
| A mean increase in Hematocrit (Hct) from Baseline to the Final Visit | 60 Days | |
| A mean increase in reticulocyte count from Baseline to the Final Visit |
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Inclusion Criteria:
Exclusion Criteria:
In the judgment of the investigator the patient is not a good candidate for the study
Blood transfusion within the last 30 days
Any of the following medical conditions:
Presence of any other condition, which in the opinion of the investigator, would make the person unsuitable for enrollment or could interfere with compliance in the study, including but not limited to alcohol or drug abuse
Any prior treatment with Erythropoiesis-stimulating Agents (ESA) within 90 days of study treatment;
History of hypersensitivity to erythropoietin or any related drug.
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| Name | Affiliation | Role |
|---|---|---|
| Rosa Real, MD | Prolong Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Faculty of Medicine, Chiang Mai University | Chiang Mai | 50200 | Thailand |
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| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
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| 60 Days |
| Change in clinical signs and symptoms of β-NTDT from Baseline to the Final Visit | 60 Days |
| D006402 |
| Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |