Trial Outcomes & Findings for Darbepoetin Alfa MDS Companion Protocol (NCT NCT02175277)
NCT ID: NCT02175277
Last Updated: 2018-11-14
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
9 participants
Primary outcome timeframe
From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Results posted on
2018-11-14
Participant Flow
This study was conducted at 5 centers in Belgium from 12 June 2014 (first participant enrolled) to 20 March 2017 (last participant completed study).
This study enrolled participants who completed the active treatment period of the phase 3 Study 20090160 (NCT01362140).
Participant milestones
| Measure |
Darbepoetin Alfa
Participants received darbepoetin alfa for up to 73 weeks or until progression to acute myeloid leukemia (AML), whichever occurred first.
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Darbepoetin Alfa
Participants received darbepoetin alfa for up to 73 weeks or until progression to acute myeloid leukemia (AML), whichever occurred first.
|
|---|---|
|
Overall Study
Protocol-specified Criteria
|
1
|
Baseline Characteristics
Darbepoetin Alfa MDS Companion Protocol
Baseline characteristics by cohort
| Measure |
Darbepoetin Alfa
n=9 Participants
Participants received darbepoetin alfa for up to 73 weeks or until progression to acute myeloid leukemia (AML), whichever occurred first.
|
|---|---|
|
Age, Continuous
|
71.3 years
STANDARD_DEVIATION 7.9 • n=5 Participants
|
|
Age, Customized
18 - 64 years
|
1 Participants
n=5 Participants
|
|
Age, Customized
65 - 74 years
|
6 Participants
n=5 Participants
|
|
Age, Customized
75 - 84 years
|
2 Participants
n=5 Participants
|
|
Age, Customized
≥ 85 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
9 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.Population: All enrolled participants
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.
Outcome measures
| Measure |
Darbepoetin Alfa
n=9 Participants
Participants received darbepoetin alfa for up to 73 weeks or until progression to acute myeloid leukemia (AML), whichever occurred first.
|
|---|---|
|
Number of Participants With Treatment-emergent Adverse Events
All adverse events
|
9 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events
Adverse events ≥ grade 2
|
8 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events
Adverse events ≥ grade 3
|
2 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events
Adverse events ≥ grade 4
|
0 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events
Serious adverse events
|
3 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events
AEs leading to discontinuation of darbepoetin alfa
|
1 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events
Fatal adverse events
|
0 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events
Treatment-related adverse events
|
0 Participants
|
Adverse Events
Darbepoetin Alfa
Serious events: 3 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Darbepoetin Alfa
n=9 participants at risk
Participants received darbepoetin alfa for up to 73 weeks or until progression to acute myeloid leukemia (AML), whichever occurred first.
|
|---|---|
|
Eye disorders
Macular fibrosis
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
General disorders
Pyrexia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Nervous system disorders
Ischaemic stroke
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
Other adverse events
| Measure |
Darbepoetin Alfa
n=9 participants at risk
Participants received darbepoetin alfa for up to 73 weeks or until progression to acute myeloid leukemia (AML), whichever occurred first.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
22.2%
2/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Blood and lymphatic system disorders
Neutropenia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Cardiac disorders
Palpitations
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Eye disorders
Chalazion
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Eye disorders
Visual acuity reduced
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
33.3%
3/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Abdominal pain
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Aphthous ulcer
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Diarrhoea
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Dry mouth
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Haematochezia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Inguinal hernia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Nausea
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
General disorders
Asthenia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
General disorders
Fatigue
|
44.4%
4/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
General disorders
Injection site haematoma
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
General disorders
Malaise
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
General disorders
Pyrexia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Bronchitis
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Gingivitis
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Herpes simplex
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Influenza
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Nasopharyngitis
|
22.2%
2/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Oral fungal infection
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Pharyngitis
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Sinusitis
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Upper respiratory tract infection
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Urinary tract infection
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Injury, poisoning and procedural complications
Contusion
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Injury, poisoning and procedural complications
Fall
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Investigations
Blood creatinine increased
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
22.2%
2/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Musculoskeletal and connective tissue disorders
Hypercreatinaemia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Nervous system disorders
Dizziness
|
22.2%
2/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Nervous system disorders
Headache
|
22.2%
2/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Nervous system disorders
Restless legs syndrome
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Psychiatric disorders
Depression
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Renal and urinary disorders
Nocturia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
3/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
44.4%
4/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
33.3%
3/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Rash
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Skin discomfort
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Vascular disorders
Haematoma
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Vascular disorders
Hypotension
|
11.1%
1/9 • From first dose of darbepoetin alfa to 30 days after last dose; the maximum treatment duration was 73 weeks.
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER