NCT02117427

Brief Summary

The objectives of this study are to assess safety and to evaluate the biologic activity of TARGTEPO treatment.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2014

Typical duration for phase_1

Geographic Reach
1 country

5 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 16, 2014

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 21, 2014

Completed
1 month until next milestone

Study Start

First participant enrolled

May 27, 2014

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2016

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 20, 2017

Completed
1.6 years until next milestone

Results Posted

Study results publicly available

October 30, 2018

Completed
Last Updated

October 30, 2018

Status Verified

February 1, 2018

Enrollment Period

2.1 years

First QC Date

April 16, 2014

Results QC Date

July 20, 2017

Last Update Submit

February 15, 2018

Conditions

Outcome Measures

Primary Outcomes (3)

  • Total EPO Secretion

    up to 52 weeks

  • Percent (%) of Hb Measurements After Implantation Within 9-11 g/dL

    52 weeks

  • Percent (%) of Hb Measurements After Implantation Within 9-12 g/dL

    Due to the small sample size and the dispersion of total EPO secretion, the efficacy analyses were performed on all of the patients as a single cohort of intended dose between 25 and 45 U/Kg/d.

    52 weeks

Study Arms (3)

Group A

EXPERIMENTAL

MDGN201 TARGTEPO secreting EPO (18-25 IU/Kg/day)

Biological: MDGN201 TARGTEPO

Group B

EXPERIMENTAL

MDGN201 TARGTEPO secreting EPO (35-45 IU/Kg/day)

Biological: MDGN201 TARGTEPO

Group C

EXPERIMENTAL

MDGN201 TARGTEPO secreting EPO (55-65 IU/Kg/day)

Biological: MDGN201 TARGTEPO

Interventions

Erythropoietin secreted by TARGTEPO transduced with MDGN201

Group AGroup BGroup C

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • For ESRD patients: Subject diagnosed with Anemia due to Chronic Renal Failure CKD stage 5 on hemodialysis treatment for at least 6 months. Average Hb during last month between 9 to 12g/dL.
  • Kt/V \>1
  • INR not higher than 1.2
  • Serum albumin \>3.5
  • Subjects with adequate iron stores (transferrin saturation \> 20.0% and/or ferritin \>100 ng/ml).

You may not qualify if:

  • Uncontrolled hypertension (defined as diastolic blood pressure \> 110 mmHg or systolic blood pressure \> 180 mmHg during screening).
  • Subjects who receive oral anti-coagulation treatment (e.g. warfarin)
  • Subjects who receive Acetyl Salicylic 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 EPODURE procedure
  • 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, thalassemia, myelodysplastic syndromes, hematologic malignancy, myeloma, hemolytic anemia).
  • Current systemic infection, active inflammatory disease, or malignancy under active treatment.
  • 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 heart 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 or five half-lives of the investigational drug whichever is longer, since ending other investigational device or drug trial(s) prior to Screening phase.
  • Psychiatric, addictive, or any other disorder that compromises ability to provide informed consent for participation in this study.
  • Female subjects of child-bearing potential and males that do not agree to use acceptable methods of contraception during the study.
  • +10 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Barzili Medical Center

Ashkelon, Israel

Location

Meir Medical Center

Kfar Saba, Israel

Location

Medical Center of the Galilee

Nahariya, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, Israel

Location

Assaf Harofeh Medical Center

Zrifin, Israel

Location

Related Links

MeSH Terms

Conditions

Renal Insufficiency, ChronicKidney Failure, Chronic

Condition Hierarchy (Ancestors)

Renal InsufficiencyKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Results Point of Contact

Title
Garry A Neil
Organization
Aevi Genomic Medicine

Study Officials

  • Shany Blum, MD PhD

    Aevi Genomic Medicine, LLC, a Cerecor company

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

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

April 16, 2014

First Posted

April 21, 2014

Study Start

May 27, 2014

Primary Completion

June 30, 2016

Study Completion

March 20, 2017

Last Updated

October 30, 2018

Results First Posted

October 30, 2018

Record last verified: 2018-02

Locations