Myelodysplastic Syndromes
Conditions
Keywords
Myelodysplastic Syndromes, MDS
Brief summary
At the conclusion of study AZA PH GL 2003 CL 001 (NCT00071799), eligible participants could be enrolled in an optional extension phase in order to continue treatment with azacitidine until it became commercially available; the continued treatment was for ethical and safety reasons only and not to provide additional efficacy data.
Detailed description
At the conclusion of study AZA PH GL 2003 CL 001 (NCT00071799), eligible participants could be enrolled in an optional extension phase in order to continue treatment with azacitidine until it became commercially available; the continued treatment was for ethical and safety reasons only and not to provide additional efficacy data. During the extension phase, participants were treated based on 28-day cycles and monitored for hematologic, nonhematologic, and renal toxicities. Recommended monitoring procedures included complete blood count with differential and platelets at least once each cycle prior to dosing and as needed, bone marrow biopsy and aspirate as clinically indicated, and additional tests or more frequent monitoring at the investigator's discretion based on the patient's clinical status. The azacitidine dose could be modified for toxicities. Laboratory data were not collected during the extension phase.
Interventions
DRUGAzacitidine
Azacitidine was injected subcutaneously (SC) for 7 days. The 7-day dosing was repeated every 28 days with dose adjustments allowed. The initial dose during the primary study was 75mg/m\^2/day.
Sponsors
Celgene
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
* Participants were considered eligible if they had been randomized to azacitidine treatment in the primary study and were receiving azacitidine at the time of study closure, had completed 12 months of treatment and observation in the primary study, and had signed the informed consent document for the extension phase of the study. * See study: AZA PH GL 2003 CL 001 for a list of inclusion criteria for the primary study.
Exclusion criteria
* None specific to the extension phase of the study * See study: AZA PH GL 2003 CL 001 for a list of
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period | 43- 68 months | Participant counts for a variety of subsets of treatment emergent adverse events (TEAEs)during the extension study period (43-68 months). Subsets include participants counts for serious TEAEs, serious TEAEs that the investigator evaluated as releated to treatment, TEAEs leading to discontinuation of therapy, or a dose reduction, or a dose interruption. |
Countries
Australia, Bulgaria, France, Germany, Greece, Hungary, Italy, Netherlands, Poland, Spain, United Kingdom
Participant flow
Pre-assignment details
Participants were eligible for the extension study of AZA PH GL 2003 CL 001 if they had been randomized to azacitidine treatment in the primary study and receiving azacitidine at the time of study closure, completed 12 months of treatment and observation in the primary study, and signed the informed consent document for the extension study.
Participants by arm
| Arm | Count |
|---|---|
| Azacitidine Azacitidine (study drug) plus best supportive care. Azacitidine was injected subcutaneously (SC) for 7 days. The 7-day dosing was repeated every 28 days with dose adjustments allowed. The initial dose during the primary study was 75mg/m\^2/day. | 40 |
| Total | 40 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Bone marrow transplant | 1 |
| Overall Study | Death | 7 |
| Overall Study | Myocardial infarction | 1 |
| Overall Study | NCI CTC Grade 3 or 4 toxicity | 1 |
| Overall Study | No longer receiving clinical benefit | 17 |
| Overall Study | Program closed | 5 |
| Overall Study | Sponsor decision | 4 |
| Overall Study | Started additional treatment | 1 |
| Overall Study | Transformation to AML | 1 |
| Overall Study | Withdrawal by Subject | 2 |
Baseline characteristics
| Characteristic | Azacitidine |
|---|---|
| Age, Continuous | 68.6 years STANDARD_DEVIATION 8.23 |
| French-American-British (FAB) Classification Acute myeloid leukemia | 1 participants |
| French-American-British (FAB) Classification Modified chronic myelomonocytic leukemia | 1 participants |
| French-American-British (FAB) Classification Myeloproliferative disease | 1 participants |
| French-American-British (FAB) Classification RAEB in transformation | 10 participants |
| French-American-British (FAB) Classification Refractory anemia with excess blasts (RAEB) | 27 participants |
| International Prognostic Scoring System (IPSS) High risk (2.5-3.5) | 14 participants |
| International Prognostic Scoring System (IPSS) Indeterminate | 2 participants |
| International Prognostic Scoring System (IPSS) Intermediate risk level 1 (0.5-1.0) | 1 participants |
| International Prognostic Scoring System (IPSS) Intermediate risk level 2 (1.5-2.0) | 21 participants |
| International Prognostic Scoring System (IPSS) Not applicable | 2 participants |
| Race/Ethnicity, Customized Caucasian | 40 participants |
| Sex: Female, Male Female | 8 Participants |
| Sex: Female, Male Male | 32 Participants |
| World Health Organization (WHO) Classification Acute myeloid leukemia | 11 participants |
| World Health Organization (WHO) Classification Chronic myelomonocytic leukemia - 1 (CMMoL-1) | 0 participants |
| World Health Organization (WHO) Classification Chronic myelomonocytic leukemia - 2 (CMMoL-2) | 2 participants |
| World Health Organization (WHO) Classification Refractory anemia with excess blasts - 1 | 5 participants |
| World Health Organization (WHO) Classification Refractory anemia with excess blasts - 2 | 22 participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 36 / 40 |
| serious Total, serious adverse events | 20 / 40 |
Outcome results
Primary
Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period
Participant counts for a variety of subsets of treatment emergent adverse events (TEAEs)during the extension study period (43-68 months). Subsets include participants counts for serious TEAEs, serious TEAEs that the investigator evaluated as releated to treatment, TEAEs leading to discontinuation of therapy, or a dose reduction, or a dose interruption.
Time frame: 43- 68 months
Population: Safety population includes all 40 participants in the extension study.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Azacitidine | Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period | Participants with >=1 treatment emergent AE (TEAE) | 39 participants |
| Azacitidine | Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period | Participants with >=1 treatment related TEAE | 27 participants |
| Azacitidine | Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period | Participants with >=1 serious TEAE | 20 participants |
| Azacitidine | Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period | Participants with >=1 serious trtment related TEAE | 5 participants |
| Azacitidine | Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period | Participants w TEAE leading to discontinued treat | 15 participants |
| Azacitidine | Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period | Participants w TEAE leading to dose reduction | 4 participants |
| Azacitidine | Number of Participants in Different Categories of Treatment Emergent Adverse Events for the Extension Period | Participants w TEAE leading to dose interruption | 15 participants |