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Safety and Efficacy of Sustained Erythropoietin Therapy

Primary Purpose

Anemia

Status

Completed

Phase

Phase 1

Locations

Israel

Study Type

Interventional

Intervention

EPODURE (dermal Biopump for erythropoietin)

About this trial

This is an interventional treatment trial for Anemia

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Adult male or female subjects between 18 to 75 years of age at the time of screening visit.
Subject with Anemia of Chronic Renal Failure CKD stage 3-4. estimated GFR of 15-60 ml/min with Female: Hb < 10 g%, Male: Hb < 11 g%.
Chronic renal failure subjects who are EPO naïve or have been off EPO or similar Erythropoietic drugs for more than four (4) weeks.
Subjects who are clinically stable.
Adequate iron stores (transferrin saturation ≥ 20.0% and ferritin ≥100 ng/ml).
Subjects who receive anti-coagulation treatment may be enrolled provided anti-coagulation treatment can be discontinued two weeks prior to the harvesting visit. INR level must be within normal range (0.9 - 1.5).

Signed written informed consent to participate in the study by subject

Exclusion Criteria:

Uncontrolled hypertension (defined as diastolic blood pressure > 110 mmHg or systolic blood pressure > 180 mmHg during screening).
Congestive heart failure (New York Heart Association functional class III or IV).
Grand mal seizures within 2 years of the screening visit.
Clinical evidence of severe hyperparathyroidism as defined by PTH levels of > 10 times the upper normal limits.
Major surgery within 12 weeks of the screening visit.
Systemic hematologic diseases (e.g., sickle cell anemia, myelodysplastic syndromes, hematologic malignancy, myeloma, hemolytic anemia).
Current systemic infection, active inflammatory disease, or malignancy under treatment.
Known positivity for HIV antibody.
Subjects known to have tested positive at any time in the past for antibodies to erythropoietic proteins.
Subject has history of malignancy within the past 2 years prior to the screening visit, with the exception of basal cell carcinoma.
Subjects with other concurrent severe and/or uncontrolled medical condition which could compromise participation in the study (i.e. active infection, uncontrolled diabetes, uncontrolled hypertension, congestive cardiac failure, unstable angina, ventricular arrhythmias, active ischemic heart disease, myocardial infarction within six months, uncompensated cirrhosis, active upper GI tract ulceration).
Subject is currently enrolled in, or has not yet completed a period of at least 30 days since ending other investigational device or drug trial(s).
Psychiatric, addictive, or any other disorder that compromises ability to give truly informed consent for participation in this study.
Female subjects of child-bearing potential and not having undergone permanent sterilization procedures.
Pregnant and lactating female subjects.
Chronic alcoholic or drug abuse subjects.
Steroid or other immunosuppressive treatment.
Subjects unwilling or unable to comply with the study procedures.

Sites / Locations

Hadassah Medical Organization
Tel Aviv Sourasky Medical Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Arm Description

Cohort 1 (Low Dose group) 18-25 IU/kg/day

Cohort 2 (Intermediate Dose group): 35-45 IU/kg/day

Cohort 3 (High Dose group): 55-65 IU/kg/day

Outcomes

Primary Outcome Measures

Safety Endpoints: adverse events; safety laboratory values; immune response by determination of anti-EPO antibodies; dermal safety outcomes

Secondary Outcome Measures

Elevation of serum EPO levels at least 10 mU/ml above baseline for duration of at least six (6) weeks following implantation

Full Information

NCT ID

NCT00542568

First Posted

October 10, 2007

Last Updated

April 16, 2014

Sponsor

Medgenics Medical Israel Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT00542568

Brief Title

Safety and Efficacy of Sustained Erythropoietin Therapy

Official Title

Safety and Efficacy of Sustained Erythropoietin Therapy of Anemia in Chronic Kidney Disease Patients Using EPODURE Biopump

Study Type

Interventional

2. Study Status

Record Verification Date

April 2014

Overall Recruitment Status

Completed

Study Start Date

August 2008 (undefined)

Primary Completion Date

July 2012 (Actual)

Study Completion Date

January 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Medgenics Medical Israel Ltd.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The purposes of this study are to assess safety, efficacy, and subject satisfaction of EPODURE Biopump (an autologous dermal biopump capable of sustained secretion of therapeutic EPO in the body, using a small tissue explant from the patient's own skin) treatment in Chronic Kidney Disease (CKD) patients over a period of up to six (6) months.

Detailed Description

Anemia, is a common complication of chronic kidney disease (CKD) resulting from insufficient production of the hormone erythropoietin by the damaged kidney leading to a decrease in red blood cells production by the bone marrow. Replacement therapy with recombinant human erythropoietin can effectively correct anemia in patients. However, despite the availability of recombinant human erythropoietin for more than a decade for use in CKD patients, two thirds of patients initiating dialysis have a hematocrit less than 30%, and three fourths have a hemoglobin (Hb) less than 11 g/dL the level recommended by the National Kidney Foundation Kidney Disease Outcome Quality Initiative. Treatment with recombinant human erythropoietin typically involves subcutaneous (SC) administration at regular intervals followed by frequent laboratory tests to monitor hemoglobin concentration. There is a need to provide an significantly improved care in this area using a sustained therapy approach. EPODURE, is an autologous dermal biopump capable of sustained secretion of therapeutic EPO in the body, using a small tissue explant from the patient's own skin. The EPODURE biopump is harvested directly from the patient's dermis under local anesthetic. EPODURE Biopumps, produced by ex vivo transduction of MOs with Helper Dependent Adenoviral EPO vectors (HDAd-EPO), expresses and secretes EPO. The EPODURE Biopump is subsequently implanted subcutaneously back to the patient in order to provide continuous delivery of a known amount of EPO.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Anemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

19 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

Cohort 1 (Low Dose group) 18-25 IU/kg/day

Arm Title

Arm Type

Experimental

Arm Description

Cohort 2 (Intermediate Dose group): 35-45 IU/kg/day

Arm Title

Arm Type

Experimental

Arm Description

Cohort 3 (High Dose group): 55-65 IU/kg/day

Intervention Type

Biological

Intervention Name(s)

EPODURE (dermal Biopump for erythropoietin)

Intervention Description

Subjects will undergo similar study procedures and evaluations; however each dosage group will receive a different targeted dose of EPO delivered via EPODURE Biopump

Primary Outcome Measure Information:

Title

Safety Endpoints: adverse events; safety laboratory values; immune response by determination of anti-EPO antibodies; dermal safety outcomes

Time Frame

6 weeks and 6 months

Secondary Outcome Measure Information:

Title

Elevation of serum EPO levels at least 10 mU/ml above baseline for duration of at least six (6) weeks following implantation

Time Frame

6 weeks and 4 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Adult male or female subjects between 18 to 75 years of age at the time of screening visit. Subject with Anemia of Chronic Renal Failure CKD stage 3-4. estimated GFR of 15-60 ml/min with Female: Hb < 10 g%, Male: Hb < 11 g%. Chronic renal failure subjects who are EPO naïve or have been off EPO or similar Erythropoietic drugs for more than four (4) weeks. Subjects who are clinically stable. Adequate iron stores (transferrin saturation ≥ 20.0% and ferritin ≥100 ng/ml). Subjects who receive anti-coagulation treatment may be enrolled provided anti-coagulation treatment can be discontinued two weeks prior to the harvesting visit. INR level must be within normal range (0.9 - 1.5). Signed written informed consent to participate in the study by subject Exclusion Criteria: Uncontrolled hypertension (defined as diastolic blood pressure > 110 mmHg or systolic blood pressure > 180 mmHg during screening). Congestive heart failure (New York Heart Association functional class III or IV). Grand mal seizures within 2 years of the screening visit. Clinical evidence of severe hyperparathyroidism as defined by PTH levels of > 10 times the upper normal limits. Major surgery within 12 weeks of the screening visit. Systemic hematologic diseases (e.g., sickle cell anemia, myelodysplastic syndromes, hematologic malignancy, myeloma, hemolytic anemia). Current systemic infection, active inflammatory disease, or malignancy under treatment. Known positivity for HIV antibody. Subjects known to have tested positive at any time in the past for antibodies to erythropoietic proteins. Subject has history of malignancy within the past 2 years prior to the screening visit, with the exception of basal cell carcinoma. Subjects with other concurrent severe and/or uncontrolled medical condition which could compromise participation in the study (i.e. active infection, uncontrolled diabetes, uncontrolled hypertension, congestive cardiac failure, unstable angina, ventricular arrhythmias, active ischemic heart disease, myocardial infarction within six months, uncompensated cirrhosis, active upper GI tract ulceration). Subject is currently enrolled in, or has not yet completed a period of at least 30 days since ending other investigational device or drug trial(s). Psychiatric, addictive, or any other disorder that compromises ability to give truly informed consent for participation in this study. Female subjects of child-bearing potential and not having undergone permanent sterilization procedures. Pregnant and lactating female subjects. Chronic alcoholic or drug abuse subjects. Steroid or other immunosuppressive treatment. Subjects unwilling or unable to comply with the study procedures.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Prof. Eithan Galun, M.D

Organizational Affiliation

Hadassah Medical Organization

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Doron Schwartz, MD

Organizational Affiliation

Tel-Aviv Sourasky Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Hadassah Medical Organization

City

Jerusalem

Country

Israel

Facility Name

Tel Aviv Sourasky Medical Center

City

Tel Aviv

Country

Israel

12. IPD Sharing Statement

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Safety and Efficacy of Sustained Erythropoietin Therapy

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