Anemia of End Stage Renal Disease
Conditions
Keywords
Peritoneal Dialysis, End Stage Renal Disease, Anemia
Brief summary
The objectives of this study are to assess safety and to evaluate the biologic activity of TARGTEPO treatment in Peritoneal Dialysis patients
Detailed description
This is a Phase II, open-label study. Each patient will be administered with a targeted dose of EPO delivered via TARGTEPO. The targeted doses will be determined according to 2 cohorts as follows: Group A (18-25 IU/Kg/day), Group B (35-45 IU/Kg/day). The objective is to evaluate safety and biologic activity of TARGTEPO treatment when maintaining Hb levels within the target range of 9-12 g/dl. Biological activity assessments will include duration of TARGTEPO secretion as measured by serum EPO levels above baseline.
Interventions
BIOLOGICALMDGN201 TARGTEPO
MDGN201 TARGTEPO secreting EPO (18-25 IU/Kg/day)
Sponsors
Medgenics Medical Israel Ltd.
Aevi Genomic Medicine, LLC, a Cerecor company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Adult male or female subjects between 18 to 80 years of age at the time of screening visit. 2. Subject diagnosed with anemia due to Chronic Kidney Disease (CKD) Stage 5 on peritoneal dialysis treatment for at least 6 months. Average Hgb during last month between 9 to 12g/dL. Stable dose of ESAs over the past month prior to enrollment. 3. Hypertensive subjects are stable on anti HTN treatment for the past one month prior to enrollment and has Systolic blood pressure (BP) below 150 and Diastolic BP below 90 upon enrollment. 4. Kt/V \> 1. 5. INR ≤ 1.2. 6. Serum albumin \> 3.2. 7. Subjects with adequate iron stores (transferrin saturation \> 20.0% and/or ferritin \>100 ng/ml). 8. Capable of providing signed written informed consent to participate in the study.
Exclusion criteria
1. Uncontrolled hypertension (defined as diastolic blood pressure \> 110 mmHg or systolic blood pressure \> 180 mmHg during screening). 2. Subjects who receive oral anti-coagulation treatment (e.g. warfarin). 3. Subjects who receive acetylsalicylic acid (ASA) above 325 mg/day or patients who receive ASA treatment between 100mg/d and 325 mg/d who cannot discontinue it for 1 week prior to each Harvest or Implantation Procedure. 4. Congestive heart failure (New York Heart Association functional class III or IV). 5. Grand mal seizures within 2 years of the Screening Visit. 6. Clinical evidence of severe hyperparathyroidism as defined by parathyroid hormone (PTH) levels of \> 10 times the upper normal limits. 7. Major surgery within 12 weeks of the Screening Visit. 8. Systemic hematologic diseases (e.g., sickle cell anemia, thalassemia (excluding thalassemia minor), myelodysplastic syndromes, hematologic malignancy, myeloma, hemolytic anemia). 9. Current systemic infection, active inflammatory disease, or malignancy under active treatment. 10. Subjects known to have tested positive at any time in the past for antibodies to erythropoietic proteins. 11. Subject has history of malignancy within the past 2 years prior to the Screening Visit, with the exception of basal cell carcinoma. 12. Subjects with other concurrent severe and/or uncontrolled medical condition that could compromise participation in the study (i.e. active infection, uncontrolled diabetes, uncontrolled hypertension, congestive heart failure, unstable angina, ventricular arrhythmias, active ischemic heart disease, myocardial infarction within six months, uncompensated cirrhosis, active upper gastrointestinal (GI) tract ulceration). 13. Subject is currently enrolled in, or has not yet completed a period of at least 30 days or five half-lives of the investigational drug whichever is longer, since ending other investigational device or drug trial(s) prior to Screening Phase. 14. Psychiatric, addictive, or any other disorder that compromises ability to provide informed consent for participation in this study. 15. Female subjects of child-bearing potential and males that do not agree to use acceptable methods of contraception during the study. 16. Pregnant or lactating female subjects. 17. Chronic alcoholic or drug abuse subjects. 18. Steroid or other immunosuppressive treatment (other than topical or inhaled steroids). 19. Subjects unwilling or unable to comply with the study procedures. 20. EPO-naïve subjects. 21. Known sensitivity to gentamicin and amphotericin. 22. History of chronic or active hepatitis B and/or C infection or positive serology at screening, and known positive Human Immunodeficiency Virus (HIV) or positive serology at screening. 23. Subject had a blood transfusion within 84 days prior to Screening Visit. 24. Subject has a date for renal transplantation. 25. Refer to the United States Product Insert (USPI) - Depo-Medrol (DM) - Methylprednisolone Acetate - Injectable (Appendix A) for any concomitant drug taken by the patient, which its interactions with DM will warrant exclusion from this protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Patients Who Achieved Biological Activity of MDGN201 TARGTEPO Secretion as Measured by Serum EPO Levels Above Baseline. | 52 weeks |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Group A TARGTEPO 18-25 IU/kg/day | 3 |
| Group B TARGTEPO 35-45 IU/kg/day | 0 |
| Total | 3 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Early Study Termination by Sponsor | 1 | 0 |
| Overall Study | Poor in-vitro secreting TARGTs | 1 | 0 |
| Overall Study | Screen Failure | 1 | 0 |
Baseline characteristics
| Characteristic | Group A | Total | Group B |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants | 3 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 3 Participants | 3 Participants | 0 Participants |
| Sex: Female, Male Female | 1 Participants | 1 Participants | 0 Participants |
| Sex: Female, Male Male | 2 Participants | 2 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 2 / 2 | 0 / 0 |
| serious Total, serious adverse events | 1 / 2 | 0 / 0 |
Outcome results
Primary
Patients Who Achieved Biological Activity of MDGN201 TARGTEPO Secretion as Measured by Serum EPO Levels Above Baseline.
Time frame: 52 weeks
Population: No statistical analysis was performed as only one subject was treated with MDGN201 TARGTEPO due to the Sponsor's decision to discontinue study.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Group A | Patients Who Achieved Biological Activity of MDGN201 TARGTEPO Secretion as Measured by Serum EPO Levels Above Baseline. | 1 Participants |
| Group B | Patients Who Achieved Biological Activity of MDGN201 TARGTEPO Secretion as Measured by Serum EPO Levels Above Baseline. | 0 Participants |