Myelodysplastic Syndrome (MDS)
Conditions
Keywords
Myelodysplastic Syndrome (MDS), Darbepoetin alfa, low risk MDS, intermediate-1 risk MDS, International Prognostic Scoring System (IPSS)
Brief summary
The primary objective of the study was to provide required access of investigational product (darbepoetin alfa) beyond the end of the active treatment period (EOATP) of the darbepoetin alfa MDS 20090160 (NCT01362140) study for patients who had continued demonstration of benefit from darbepoetin alfa treatment and to describe the safety of longer-term use in this patient population.
Detailed description
This is a phase 3b, multi-centre, open-label, single-arm companion study to the MDS 20090160 study (NCT01362140) for the treatment of anaemic patients with MDS. Participants who completed the active-treatment period of the darbepoetin alfa MDS 20090160 study and met the eligibility criteria could be enrolled into this study to continue treatment of darbepoetin alfa for up to 73 weeks or until progression to acute myelogenous leukemia (AML), whichever occurs first.
Interventions
DRUGDarbepoetin Alfa
The first dose of darbepoetin alfa was the same as that administered at the last dosing visit of the active treatment period in Study 20090160. Doses could be increased up to a maximum of 500 μg every two weeks (Q2W).
Sponsors
Amgen
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
* Subject or subject's legally acceptable representative has provided informed consent prior to any study-specific activities/ procedures being initiated; * Subject must continue long term follow up within parent study (20090160); * Subject must have an ongoing clinically relevant erythroid response as assessed by the Investigator using current response criteria (ie, International Working Group (IWG) response criteria);
Exclusion criteria
* Transfusion dependence defined as receiving a total of ≥ 4 units of red blood cell (RBC) transfusion in the previous 8-week period prior to enrolment; * Known diagnosis of acute myelogenous leukemia (AML) or marrow collagen fibrosis; * Known refractory anaemia with excess blast-2 (RAEB-2); * Known diagnosis of intermediate-2 or high risk MDS per International Prognostic Scoring System (IPSS); * Subjects received thrombopoiesis-stimulating factors (eg, eltrombopag, romiplostim) in the MDS 20090160 study or planning to receive such agents during the study; * Other protocol defined inclusion and
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events | From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks. | Adverse events (AEs) were graded for severity according to the Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, where Grade 1 indicates a mild AE, Grade 2 indicates a moderate AE, Grade 3 indicates severe or medically significant but not immediately life-threatening and Grade 4 indicates life-threatening consequences; urgent intervention indicated. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: * fatal * life threatening * required in-patient hospitalization or prolongation of existing hospitalization * resulted in persistent or significant disability/incapacity * congenital anomaly/birth defect * other medically important serious event The investigator assessed whether each adverse events was related to darbepoetin alfa. |
Countries
Belgium
Participant flow
Recruitment details
This study was conducted at 5 centers in Belgium from 12 June 2014 (first participant enrolled) to 20 March 2017 (last participant completed study).
Pre-assignment details
This study enrolled participants who completed the active treatment period of the phase 3 Study 20090160 (NCT01362140).
Participants by arm
| Arm | Count |
|---|---|
| Darbepoetin Alfa Participants received darbepoetin alfa for up to 73 weeks or until progression to acute myeloid leukemia (AML), whichever occurred first. | 9 |
| Total | 9 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Protocol-specified Criteria | 1 |
Baseline characteristics
| Characteristic | Darbepoetin Alfa |
|---|---|
| Age, Continuous | 71.3 years STANDARD_DEVIATION 7.9 |
| Age, Customized 18 - 64 years | 1 Participants |
| Age, Customized 65 - 74 years | 6 Participants |
| Age, Customized 75 - 84 years | 2 Participants |
| Age, Customized ≥ 85 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 8 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race/Ethnicity, Customized White | 9 Participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 9 |
| other Total, other adverse events | 9 / 9 |
| serious Total, serious adverse events | 3 / 9 |
Outcome results
Primary
Number of Participants With Treatment-emergent Adverse Events
Adverse events (AEs) were graded for severity according to the Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, where Grade 1 indicates a mild AE, Grade 2 indicates a moderate AE, Grade 3 indicates severe or medically significant but not immediately life-threatening and Grade 4 indicates life-threatening consequences; urgent intervention indicated. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: * fatal * life threatening * required in-patient hospitalization or prolongation of existing hospitalization * resulted in persistent or significant disability/incapacity * congenital anomaly/birth defect * other medically important serious event The investigator assessed whether each adverse events was related to darbepoetin alfa.
Time frame: From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Population: All enrolled participants
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Darbepoetin Alfa | Number of Participants With Treatment-emergent Adverse Events | All adverse events | 9 Participants |
| Darbepoetin Alfa | Number of Participants With Treatment-emergent Adverse Events | Adverse events ≥ grade 2 | 8 Participants |
| Darbepoetin Alfa | Number of Participants With Treatment-emergent Adverse Events | Adverse events ≥ grade 3 | 2 Participants |
| Darbepoetin Alfa | Number of Participants With Treatment-emergent Adverse Events | Adverse events ≥ grade 4 | 0 Participants |
| Darbepoetin Alfa | Number of Participants With Treatment-emergent Adverse Events | Serious adverse events | 3 Participants |
| Darbepoetin Alfa | Number of Participants With Treatment-emergent Adverse Events | AEs leading to discontinuation of darbepoetin alfa | 1 Participants |
| Darbepoetin Alfa | Number of Participants With Treatment-emergent Adverse Events | Fatal adverse events | 0 Participants |
| Darbepoetin Alfa | Number of Participants With Treatment-emergent Adverse Events | Treatment-related adverse events | 0 Participants |