A Study of EPEG in Beta Thalassemia Patients

• ClinicalTrials.gov Identifier: NCT02950857
• Recruitment Status: Completed
• First Posted: November 1, 2016
• Last Update Posted: May 25, 2018
• Sponsor: Prolong Pharmaceuticals
• Information provided by (Responsible Party): Prolong Pharmaceuticals

Tracking Information

• First Submitted Date: October 27, 2016
• First Posted Date: November 1, 2016
• Last Update Posted Date: May 25, 2018
• Actual Study Start Date: June 28, 2017
• Actual Primary Completion Date: September 27, 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 31, 2016)

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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: October 31, 2016)

• Change in the Hemoglobin from Baseline to the Final Visit [ Time Frame: 60 Days ]
• A mean increase in Hematocrit (Hct) from Baseline to the Final Visit [ Time Frame: 60 Days ]
• A mean increase in reticulocyte count from Baseline to the Final Visit [ Time Frame: 60 Days ]
• Change in clinical signs and symptoms of β-NTDT from Baseline to the Final Visit [ Time Frame: 60 Days ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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)
• 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.

• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Beta-Thalassemia

Intervention

Drug: EPEG

(Pegylated erythropoeitin)

Study Arms

• Experimental: EPEG (pegylated erythropoietin) - 0.9 µg/kg
  Four weekly subcutaneous injections of 0.9 µg/kg EPEG
  Intervention: Drug: EPEG
• Experimental: EPEG (pegylated erythropoietin) - 1.2 µg/kg
  Four weekly subcutaneous injections of 1.2 µg/kg EPEG
  Intervention: Drug: EPEG
• Experimental: EPEG (pegylated erythropoietin) - 1.5 µg/kg
  Four weekly subcutaneous injections of 1.5 µg/kg EPEG
  Intervention: Drug: EPEG

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: October 31, 2016): 6
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: September 27, 2017
• Actual Primary Completion Date: September 27, 2017 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Thailand

Administrative Information

• NCT Number: NCT02950857
• Other Study ID Numbers: PETH-001
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Prolong Pharmaceuticals
• Original Responsible Party: Same as current
• Current Study Sponsor: Prolong Pharmaceuticals
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Rosa Real, MD; Prolong Pharmaceuticals

• PRS Account: Prolong Pharmaceuticals
• Verification Date: May 2018