A Study of EPEG in Beta Thalassemia Patients

NCT02950857 | Completed Phase 1

• 1 other identifier: PETH-001
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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

Conditions

Beta-Thalassemia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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

Study Arms (3)

EPEG (pegylated erythropoietin) - 0.9 µg/kg

EXPERIMENTAL

Four weekly subcutaneous injections of 0.9 µg/kg EPEG

Drug: EPEG

EPEG (pegylated erythropoietin) - 1.2 µg/kg

EXPERIMENTAL

Four weekly subcutaneous injections of 1.2 µg/kg EPEG

Drug: EPEG

EPEG (pegylated erythropoietin) - 1.5 µg/kg

EXPERIMENTAL

Four weekly subcutaneous injections of 1.5 µg/kg EPEG

Drug: EPEG

Interventions

EPEG DRUG

(Pegylated erythropoeitin)

EPEG (pegylated erythropoietin) - 0.9 µg/kg; EPEG (pegylated erythropoietin) - 1.2 µg/kg; EPEG (pegylated erythropoietin) - 1.5 µg/kg

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Prolong Pharmaceuticals: lead

Study Sites (1)

Faculty of Medicine, Chiang Mai University

Chiang Mai, 50200, Thailand

Location

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Rosa Real, MD

  Prolong Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 27, 2016
• First Posted: November 1, 2016
• Study Start: June 28, 2017
• Primary Completion: September 27, 2017
• Study Completion: September 27, 2017
• Last Updated: May 25, 2018

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share

Locations

TH (1)