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A Study of EPEG in Beta Thalassemia Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02950857
Recruitment Status : Completed
First Posted : November 1, 2016
Last Update Posted : May 25, 2018
Sponsor:
Information provided by (Responsible Party):
Prolong Pharmaceuticals

Study Description
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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

Condition or disease Intervention/treatment Phase
Beta-Thalassemia Drug: EPEG Phase 1

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase Ib, Open-label, Repeat Dose, Study of EPEG in Beta Thalassemia Patients With Non-transfusion Dependent Thalassemia (β NTDT)
Actual Study Start Date : June 28, 2017
Actual Primary Completion Date : September 27, 2017
Actual Study Completion Date : September 27, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: EPEG (pegylated erythropoietin) - 0.9 µg/kg
Four weekly subcutaneous injections of 0.9 µg/kg EPEG
 Drug: EPEG
(Pegylated erythropoeitin)
Experimental: EPEG (pegylated erythropoietin) - 1.2 µg/kg
Four weekly subcutaneous injections of 1.2 µg/kg EPEG
 Drug: EPEG
(Pegylated erythropoeitin)
Experimental: EPEG (pegylated erythropoietin) - 1.5 µg/kg
Four weekly subcutaneous injections of 1.5 µg/kg EPEG
 Drug: EPEG
(Pegylated erythropoeitin)


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants in each treatment arm with abnormal laboratory values and/or adverse events that are related to treatment. [ Time Frame: 60 days ]
    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 :
  1. Change in the Hemoglobin from Baseline to the Final Visit [ Time Frame: 60 Days ]
  2. A mean increase in Hematocrit (Hct) from Baseline to the Final Visit [ Time Frame: 60 Days ]
  3. A mean increase in reticulocyte count from Baseline to the Final Visit [ Time Frame: 60 Days ]
  4. Change in clinical signs and symptoms of β-NTDT from Baseline to the Final Visit [ Time Frame: 60 Days ]

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female
  2. Age 18 - 65 years of age
  3. Confirmed diagnosis of Non-Transfusion Dependent β-thalassemia (β-NTDT)
  4. Hemoglobin 6.0-10.0 g/dL
  5. Signed and dated informed written consent by the subject
  6. Able to receive subcutaneous injections of study drug
  7. Female patients must be non-lactating
  8. Female patients of reproductive potential must have a negative serum pregnancy (β-HCG) test at screening.

Exclusion Criteria:

  1. In the judgment of the investigator the patient is not a good candidate for the study
  2. Blood transfusion within the last 30 days

  3. Any of the following medical conditions:

    1. Severe kidney insufficiency, defined as use of hemodialysis or serum creatinine at levels greater than 2.5 mg/dL at the time of screening
    2. Cardiac disease with adjustment of cardiac medications in the 60 days before study entry
    3. 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
    4. Stroke, defined as a new focal neurological deficit lasting more than 24 hours in the 45 days before study entry
    5. New diagnosis of pulmonary embolism by ventilation-perfusion scan, angiography, or any other technique in the 90 days before study entry
    6. History of retinal detachment or retinal hemorrhage in the 180 days before study entry
    7. Use of nitrate-based vasodilators, prostacyclin (inhaled, subcutaneous, or intravenous)
    8. Acute asthma exacerbation requiring use of prednisone in the 60 days before study entry
    9. Initiation or dosage increase of calcium channel blockers in the 30 days before study entry
    10. Initiation of any other cardiac or pulmonary medication in the 90 days before study entry
  4. 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
  5. Any prior treatment with Erythropoiesis-stimulating Agents (ESA) within 90 days of study treatment;
  6. History of hypersensitivity to erythropoietin or any related drug.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02950857


Locations
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Thailand
Faculty of Medicine, Chiang Mai University
Chiang Mai, Thailand, 50200
Sponsors and Collaborators
Prolong Pharmaceuticals
Investigators
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Study Director: Rosa Real, MD Prolong Pharmaceuticals
More Information
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Responsible Party: Prolong Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02950857    
Other Study ID Numbers: PETH-001
First Posted: November 1, 2016    Key Record Dates
Last Update Posted: May 25, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn