A Study of EPEG in Beta Thalassemia Patients

A Phase Ib, Open-label, Repeat Dose, Study of EPEG in Beta Thalassemia Patients With Non-transfusion Dependent Thalassemia (β NTDT)

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

Study Overview

• Status: Completed
• Conditions: Beta-Thalassemia
• Intervention / Treatment: Drug: EPEG

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 Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 1

Contacts and Locations

Study Locations

• Thailand
  • Chiang Mai, Thailand, 50200
    • Faculty of Medicine, Chiang Mai University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

3

Arms and Interventions

Participant Group / 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	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	60 Days
Change in clinical signs and symptoms of β-NTDT from Baseline to the Final Visit	60 Days

Collaborators and Investigators

• Sponsor: Prolong Pharmaceuticals
• Investigators: Study Director: Rosa Real, MD, Prolong Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): June 28, 2017
• Primary Completion (Actual): September 27, 2017
• Study Completion (Actual): September 27, 2017

Study Registration Dates

• First Submitted: October 27, 2016
• First Submitted That Met QC Criteria: October 31, 2016
• First Posted (Estimate): November 1, 2016

Study Record Updates

• Last Update Posted (Actual): May 25, 2018
• Last Update Submitted That Met QC Criteria: May 23, 2018
• Last Verified: May 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: PETH-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No