Anemia
Conditions
Brief summary
This single arm study assessed the efficacy and safety of subcutaneous methoxy polyethylene glycol-epoetin beta (Mircera), a continuous erythropoietin receptor activator (C.E.R.A.), for correction and/or maintenance of hemoglobin levels in participants with chronic kidney disease and renal anemia, who were not treated with erythropoiesis-stimulating agents (ESA) or on dialysis. Eligible participants received monthly subcutaneous injections of methoxy polyethylene glycol-epoetin beta at an initial recommended dose of 1.2 micrograms/kilogram (mcg/kg). The anticipated time on study treatment was 3-10 months, and the target sample size was 200 individuals.
Interventions
DRUGmethoxy polyethylene glycol-epoetin beta
1.2 mcg/kg administered subcutaneously (sc) monthly for 36 weeks (initial recommended dose)
Sponsors
Hoffmann-La Roche
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult participants, \>=18 years of age; * Chronic renal anemia; * No ESA therapy during previous 3 months.
Exclusion criteria
* Transfusion of red blood cells during previous 2 months; * Poorly controlled hypertension requiring hospitalization in previous 6 months; * Significant acute or chronic bleeding.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Maintaining Average Hemoglobin Concentration During Efficacy Evaluation Period (EEP) Within Target Range | Weeks 29-36 | The EEP was week 29 through week 36. The target range for average hemoglobin concentration was 10.0 - 12.0 g/dL. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Hemoglobin Concentration to Efficacy Evaluation Period (EEP) | Weeks 0-36 | The mean change Baseline Hemoglobin to the time adjusted average of Hemoglobin during the EEP. |
| Percentage of Participants Maintaining Hemoglobin Concentrations Within Range of 10-12 Grams/Deciliter (g/dL) Throughout Efficacy Evaluation Period (EEP) | Weeks 29-36 | — |
| Mean Time Spent in Hemoglobin Range of 10-12 g/dL During Efficacy Evaluation Period (EEP) | Weeks 29-36 | — |
| Percentage of Participants With Adverse Events | Weeks 1-40 | An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. |
Countries
Belgium
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Methoxy Polyethylene Glycol-Epoetin Beta Participants received methoxy polyethylene glycol-epoetin beta treatment monthly for 36 weeks with an efficacy evaluation period (EEP) during weeks 29-36 and followed by a 4 week follow-up period.
methoxy polyethylene glycol-epoetin beta: 1.2 mcg/kg administered subcutaneously (sc) monthly for 36 weeks (initial recommended dose) | 34 |
| Total | 34 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 2 |
| Overall Study | Blood Transfusion | 2 |
| Overall Study | Death | 2 |
| Overall Study | Dialysis Initiation | 5 |
| Overall Study | Failure to Return | 1 |
| Overall Study | Insufficient Therapeutic Response | 1 |
| Overall Study | Lost to Follow-up | 1 |
Baseline characteristics
| Characteristic | Methoxy Polyethylene Glycol-Epoetin Beta |
|---|---|
| Age, Continuous | 77 years |
| Sex: Female, Male Female | 10 Participants |
| Sex: Female, Male Male | 24 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 29 / 34 |
| serious Total, serious adverse events | 15 / 34 |
Outcome results
Primary
Percentage of Participants Maintaining Average Hemoglobin Concentration During Efficacy Evaluation Period (EEP) Within Target Range
The EEP was week 29 through week 36. The target range for average hemoglobin concentration was 10.0 - 12.0 g/dL.
Time frame: Weeks 29-36
Population: Intent-to-Treat (ITT) population included all participants who entered the titration period and received active drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Methoxy Polyethylene Glycol-Epoetin Beta | Percentage of Participants Maintaining Average Hemoglobin Concentration During Efficacy Evaluation Period (EEP) Within Target Range | 41 percentage of participants |
Secondary
Change From Baseline in Hemoglobin Concentration to Efficacy Evaluation Period (EEP)
The mean change Baseline Hemoglobin to the time adjusted average of Hemoglobin during the EEP.
Time frame: Weeks 0-36
Population: ITT population included all participants who entered the titration period and received active drug.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Methoxy Polyethylene Glycol-Epoetin Beta | Change From Baseline in Hemoglobin Concentration to Efficacy Evaluation Period (EEP) | 0.99 grams/deciliter (g/dL) | Standard Deviation 1.38 |
Secondary
Mean Time Spent in Hemoglobin Range of 10-12 g/dL During Efficacy Evaluation Period (EEP)
Time frame: Weeks 29-36
Population: ITT population included all participants who entered the titration period and received active drug.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Methoxy Polyethylene Glycol-Epoetin Beta | Mean Time Spent in Hemoglobin Range of 10-12 g/dL During Efficacy Evaluation Period (EEP) | 28.6 days | Standard Deviation 21.2 |
Secondary
Percentage of Participants Maintaining Hemoglobin Concentrations Within Range of 10-12 Grams/Deciliter (g/dL) Throughout Efficacy Evaluation Period (EEP)
Time frame: Weeks 29-36
Population: ITT population included all participants who entered the titration period and received active drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Methoxy Polyethylene Glycol-Epoetin Beta | Percentage of Participants Maintaining Hemoglobin Concentrations Within Range of 10-12 Grams/Deciliter (g/dL) Throughout Efficacy Evaluation Period (EEP) | 23.5 percentage of participants |
Secondary
Percentage of Participants With Adverse Events
An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.
Time frame: Weeks 1-40
Population: The safety population included all participants who received at least one dose of active drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Methoxy Polyethylene Glycol-Epoetin Beta | Percentage of Participants With Adverse Events | 97 percentage of participants |