Trial Outcomes & Findings for TARGTEPO Treatment for Anemia in Chronic Kidney Disease (CKD) Patients and End-Stage Renal Disease (ESRD) (NCT NCT02117427)
NCT ID: NCT02117427
Last Updated: 2018-10-30
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
11 participants
Primary outcome timeframe
up to 52 weeks
Results posted on
2018-10-30
Participant Flow
A total of 14 patients signed the Informed Consent Form and underwent the study screening procedures. Of these 14 patients, a total of 11 patients were enrolled into Study MG-EP-RF-02. These 11 patients are included in the ITT cohort. One patient (Group B) had an SAE prior to study procedures and became no longer eligible.
Participant milestones
| Measure |
Group A: 18-25 IU/Kg/Day
|
Group B: 35-45 IU/Kg/Day
|
Group C: 55-65 IU/Kg/Day
|
|---|---|---|---|
|
Overall Study
STARTED
|
6
|
5
|
0
|
|
Overall Study
Run in Phase & Harvest
|
6
|
4
|
0
|
|
Overall Study
Implantation & Efficacy
|
6
|
4
|
0
|
|
Overall Study
Safety Follow-up
|
1
|
0
|
0
|
|
Overall Study
Long Term Safety Follow-up
|
1
|
0
|
0
|
|
Overall Study
COMPLETED
|
1
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
5
|
5
|
0
|
Reasons for withdrawal
| Measure |
Group A: 18-25 IU/Kg/Day
|
Group B: 35-45 IU/Kg/Day
|
Group C: 55-65 IU/Kg/Day
|
|---|---|---|---|
|
Overall Study
Permanent need for exogenous EPO Tx
|
2
|
0
|
0
|
|
Overall Study
Permanent need for exogenous ESA Tx
|
3
|
1
|
0
|
|
Overall Study
Physician Decision
|
0
|
2
|
0
|
|
Overall Study
No longer meets inclusion criteria
|
0
|
1
|
0
|
|
Overall Study
Death
|
0
|
1
|
0
|
Baseline Characteristics
TARGTEPO Treatment for Anemia in Chronic Kidney Disease (CKD) Patients and End-Stage Renal Disease (ESRD)
Baseline characteristics by cohort
| Measure |
Group A
n=6 Participants
18-25 IU/Kg/day
|
Group B
n=5 Participants
35-45 IU/Kg/day
|
Group C
55-65 IU/Kg/day
|
Total
n=11 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: up to 52 weeksPopulation: 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. One subject enrolled was discontinued prior to study procedures and became no longer eligible.
Outcome measures
| Measure |
All Subjects Enrolled
n=10 Participants
|
|---|---|
|
Total EPO Secretion
|
24.1 U/Kg/d
Interval 16.5 to 50.8
|
PRIMARY outcome
Timeframe: 52 weeksPopulation: 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. One subject enrolled was discontinued prior to study procedures and became no longer eligible.
Outcome measures
| Measure |
All Subjects Enrolled
n=10 Participants
|
|---|---|
|
Percent (%) of Hb Measurements After Implantation Within 9-11 g/dL
|
49.2 percent
Interval 1.7 to 88.1
|
PRIMARY outcome
Timeframe: 52 weeksPopulation: 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. One subject enrolled was discontinued prior to study procedures and became no longer eligible.
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.
Outcome measures
| Measure |
All Subjects Enrolled
n=10 Participants
|
|---|---|
|
Percent (%) of Hb Measurements After Implantation Within 9-12 g/dL
|
75.3 percent
Interval 25.0 to 88.1
|
Adverse Events
Group A
Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths
Group B
Serious events: 4 serious events
Other events: 4 other events
Deaths: 1 deaths
Group C
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Group A
n=6 participants at risk
18-25 IU/Kg/day
|
Group B
n=4 participants at risk
35-45 IU/Kg/day
|
Group C
55-65 IU/Kg/day
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
General disorders
Pyrexia
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Appendicitis
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Bronchitis
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Infections and infestations
Pneumonia
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Infections and infestations
Sepsis
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Urosepsis
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Injury, poisoning and procedural complications
Arteriovenous fistula occlusion
|
0.00%
0/6
|
25.0%
1/4 • Number of events 2
|
—
0/0
|
|
Injury, poisoning and procedural complications
Graft thrombosis
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Vascular disorders
Hypertension
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
Other adverse events
| Measure |
Group A
n=6 participants at risk
18-25 IU/Kg/day
|
Group B
n=4 participants at risk
35-45 IU/Kg/day
|
Group C
55-65 IU/Kg/day
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/6
|
50.0%
2/4 • Number of events 2
|
—
0/0
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Ear and labyrinth disorders
Ear pain
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Ear and labyrinth disorders
Vertigo
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Endocrine disorders
Hypoparathyroidism secondary
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Eye disorders
Cataract
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Eye disorders
Retinal microaneurysm NOS
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Eye disorders
Vitreous haemorrhage
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Gastrointestinal disorders
Abdominal pain upper
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/6
|
50.0%
2/4 • Number of events 2
|
—
0/0
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/6
|
25.0%
1/4 • Number of events 3
|
—
0/0
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Gastrointestinal disorders
Nausea
|
16.7%
1/6 • Number of events 1
|
25.0%
1/4 • Number of events 2
|
—
0/0
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
General disorders
Asthenia
|
33.3%
2/6 • Number of events 11
|
75.0%
3/4 • Number of events 8
|
—
0/0
|
|
General disorders
Chest pain
|
33.3%
2/6 • Number of events 2
|
0.00%
0/4
|
—
0/0
|
|
General disorders
Chills
|
0.00%
0/6
|
50.0%
2/4 • Number of events 2
|
—
0/0
|
|
General disorders
Malaise
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
General disorders
Pain
|
0.00%
0/6
|
50.0%
2/4 • Number of events 2
|
—
0/0
|
|
General disorders
Pyrexia
|
16.7%
1/6 • Number of events 1
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Bronchitis
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Infections and infestations
Cellulitis
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Diabetic foot infection
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Herpes zoster
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Ophthalmic herpes simplex
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Respiratory tract infection
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Infections and infestations
Tooth infection
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Infections and infestations
Viral infection
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Injury, poisoning and procedural complications
Arteriovenous fistula thrombosis
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Injury, poisoning and procedural complications
Contusion
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Injury, poisoning and procedural complications
Graft thrombosis
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Injury, poisoning and procedural complications
Post procedural haematoma
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Injury, poisoning and procedural complications
Procedural dizziness
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Injury, poisoning and procedural complications
Procedural pain
|
16.7%
1/6 • Number of events 1
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Injury, poisoning and procedural complications
Thermal burn
|
33.3%
2/6 • Number of events 3
|
0.00%
0/4
|
—
0/0
|
|
Injury, poisoning and procedural complications
Wound
|
33.3%
2/6 • Number of events 2
|
0.00%
0/4
|
—
0/0
|
|
Investigations
Haemoglobin increased
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Metabolism and nutrition disorders
Diabetes mellitus
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Metabolism and nutrition disorders
Hypovolaemia
|
16.7%
1/6 • Number of events 1
|
25.0%
1/4 • Number of events 2
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
33.3%
2/6 • Number of events 3
|
0.00%
0/4
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
66.7%
4/6 • Number of events 15
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Musculoskeletal and connective tissue disorders
Synovial cyst
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • Number of events 1
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Nervous system disorders
Headache
|
0.00%
0/6
|
50.0%
2/4 • Number of events 3
|
—
0/0
|
|
Nervous system disorders
Loss of consciousness
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Nervous system disorders
Paraesthesia
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Psychiatric disorders
Dysthymic disorder
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Renal and urinary disorders
Hematuria
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
50.0%
3/6 • Number of events 10
|
0.00%
0/4
|
—
0/0
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Skin and subcutaneous tissue disorders
Decubitus ulcer
|
0.00%
0/6
|
25.0%
1/4 • Number of events 2
|
—
0/0
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
—
0/0
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
|
Vascular disorders
Hypertension
|
50.0%
3/6 • Number of events 5
|
25.0%
1/4 • Number of events 4
|
—
0/0
|
|
Vascular disorders
Hypotension
|
50.0%
3/6 • Number of events 12
|
25.0%
1/4 • Number of events 3
|
—
0/0
|
|
Vascular disorders
Phlebitis
|
0.00%
0/6
|
25.0%
1/4 • Number of events 1
|
—
0/0
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Should the investigator wish to publish the results of this study, the investigator agrees to provide Medgenics with a manuscript for review 60 days prior to submission for publication. Medgenics retains the right to delete from the manuscript information that is confidential and proprietary and to object to suggested publication and/or its timing (at the Company's sole discretion).
- Publication restrictions are in place
Restriction type: OTHER