Back to resultsA Study of EPEG in Beta Thalassemia Patients
Primary Purpose
Beta-Thalassemia
Status
Completed
Phase
Phase 1
Locations
Thailand
Study Type
Interventional
Intervention
EPEG
Sponsored by
About this trial
This is an interventional treatment trial for Beta-Thalassemia
Eligibility Criteria
Inclusion Criteria:
- Male or female
- Age 18 - 65 years of age
- Confirmed diagnosis of Non-Transfusion Dependent β-thalassemia (β-NTDT)
- Hemoglobin 6.0-10.0 g/dL
- Signed and dated informed written consent by the subject
- Able to receive subcutaneous injections of study drug
- Female patients must be non-lactating
- Female patients of reproductive potential must have a negative serum pregnancy (β-HCG) test at screening.
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:
- Severe kidney insufficiency, defined as use of hemodialysis or serum creatinine at levels greater than 2.5 mg/dL at the time of screening
- Cardiac disease with adjustment of cardiac medications in the 60 days before study entry
- Symptomatic coronary artery disease, as indicated by a history of chest pain, angina, claudication, or surgery to treat coronary artery disease in the 1 year before study entry
- Stroke, defined as a new focal neurological deficit lasting more than 24 hours in the 45 days before study entry
- New diagnosis of pulmonary embolism by ventilation-perfusion scan, angiography, or any other technique in the 90 days before study entry
- History of retinal detachment or retinal hemorrhage in the 180 days before study entry
- Use of nitrate-based vasodilators, prostacyclin (inhaled, subcutaneous, or intravenous)
- Acute asthma exacerbation requiring use of prednisone in the 60 days before study entry
- Initiation or dosage increase of calcium channel blockers in the 30 days before study entry
- Initiation of any other cardiac or pulmonary medication in the 90 days before study entry
- 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.
Sites / Locations
- Faculty of Medicine, Chiang Mai University
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
EPEG (pegylated erythropoietin) - 0.9 µg/kg
EPEG (pegylated erythropoietin) - 1.2 µg/kg
EPEG (pegylated erythropoietin) - 1.5 µg/kg
Arm Description
Four weekly subcutaneous injections of 0.9 µg/kg EPEG
Four weekly subcutaneous injections of 1.2 µg/kg EPEG
Four weekly subcutaneous injections of 1.5 µg/kg EPEG
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Change in the Hemoglobin from Baseline to the Final Visit
A mean increase in Hematocrit (Hct) from Baseline to the Final Visit
A mean increase in reticulocyte count from Baseline to the Final Visit
Change in clinical signs and symptoms of β-NTDT from Baseline to the Final Visit
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02950857
Brief Title
A Study of EPEG in Beta Thalassemia Patients
Official Title
A Phase Ib, Open-label, Repeat Dose, Study of EPEG in Beta Thalassemia Patients With Non-transfusion Dependent Thalassemia (β NTDT)
Study Type
Interventional
2. Study Status
Record Verification Date
May 2018
Overall Recruitment Status
Completed
Study Start Date
June 28, 2017 (Actual)
Primary Completion Date
September 27, 2017 (Actual)
Study Completion Date
September 27, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Prolong Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta-Thalassemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
EPEG (pegylated erythropoietin) - 0.9 µg/kg
Arm Type
Experimental
Arm Description
Four weekly subcutaneous injections of 0.9 µg/kg EPEG
Arm Title
EPEG (pegylated erythropoietin) - 1.2 µg/kg
Arm Type
Experimental
Arm Description
Four weekly subcutaneous injections of 1.2 µg/kg EPEG
Arm Title
EPEG (pegylated erythropoietin) - 1.5 µg/kg
Arm Type
Experimental
Arm Description
Four weekly subcutaneous injections of 1.5 µg/kg EPEG
Intervention Type
Drug
Intervention Name(s)
EPEG
Intervention Description
(Pegylated erythropoeitin)
Primary Outcome Measure Information:
Title
Number of participants in each treatment arm with abnormal laboratory values and/or adverse events that are related to treatment.
Description
Safety of treatment as determined by changes in vital signs, chest X-Ray, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events
Time Frame
60 days
Secondary Outcome Measure Information:
Title
Change in the Hemoglobin from Baseline to the Final Visit
Time Frame
60 Days
Title
A mean increase in Hematocrit (Hct) from Baseline to the Final Visit
Time Frame
60 Days
Title
A mean increase in reticulocyte count from Baseline to the Final Visit
Time Frame
60 Days
Title
Change in clinical signs and symptoms of β-NTDT from Baseline to the Final Visit
Time Frame
60 Days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female
Age 18 - 65 years of age
Confirmed diagnosis of Non-Transfusion Dependent β-thalassemia (β-NTDT)
Hemoglobin 6.0-10.0 g/dL
Signed and dated informed written consent by the subject
Able to receive subcutaneous injections of study drug
Female patients must be non-lactating
Female patients of reproductive potential must have a negative serum pregnancy (β-HCG) test at screening.
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:
Severe kidney insufficiency, defined as use of hemodialysis or serum creatinine at levels greater than 2.5 mg/dL at the time of screening
Cardiac disease with adjustment of cardiac medications in the 60 days before study entry
Symptomatic coronary artery disease, as indicated by a history of chest pain, angina, claudication, or surgery to treat coronary artery disease in the 1 year before study entry
Stroke, defined as a new focal neurological deficit lasting more than 24 hours in the 45 days before study entry
New diagnosis of pulmonary embolism by ventilation-perfusion scan, angiography, or any other technique in the 90 days before study entry
History of retinal detachment or retinal hemorrhage in the 180 days before study entry
Use of nitrate-based vasodilators, prostacyclin (inhaled, subcutaneous, or intravenous)
Acute asthma exacerbation requiring use of prednisone in the 60 days before study entry
Initiation or dosage increase of calcium channel blockers in the 30 days before study entry
Initiation of any other cardiac or pulmonary medication in the 90 days before study entry
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rosa Real, MD
Organizational Affiliation
Prolong Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Faculty of Medicine, Chiang Mai University
City
Chiang Mai
ZIP/Postal Code
50200
Country
Thailand
12. IPD Sharing Statement
Plan to Share IPD
No
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A Study of EPEG in Beta Thalassemia Patients
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