- ICH GCP
- Amerikanska kliniska prövningsregistret
- Klinisk prövning NCT01700673
Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS
A Phase II Study of 5-Azacitidine (5AC) in Combination With Sargramostim (GM-CSF) as Maintenance Treatment, After Definitive Therapy With Either Stem Cell Transplant (SCT) or Cytarabine-based Chemotherapy, in Patients With Poor-risk Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
Studieöversikt
Status
Betingelser
Intervention / Behandling
Detaljerad beskrivning
We propose a phase II study to determine the impact of maintenance therapy with 5-azacytidine and GM-CSF in patients with poor-risk AML or MDS, who are in remission after definitive treatment with either stem cell transplant or cytarabine-based consolidation chemotherapy.
In order to precede relapse and to avoid lead time bias, treatment would need to commence within 185 days of definitive therapy. Furthermore, approximately 50% of relapses occur within the first year and up to 80% within two years after SCT, therefore we would limit the duration of maintenance therapy to one year, followed by two years of follow-up.
Studietyp
Inskrivning (Faktisk)
Fas
- Fas 2
Kontakter och platser
Studieorter
-
-
Maryland
-
Baltimore, Maryland, Förenta staterna, 21287
- The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
-
-
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
Tar emot friska volontärer
Kön som är behöriga för studier
Beskrivning
Inclusion Criteria:
- Age > 6 months
- Initial diagnosis of poor -risk AML or MDS (defined in section 3.2), treated with either stem cell transplant or cytarabine-based consolidation chemotherapy, within the past 60-185 days
- ECOG performance status 0-2
- No morphologic evidence of leukemia or active MDS as determined by JHH Hematopathologist independent review of a bone marrow aspirate and biopsy done following the completion of therapy and within 14 days prior to enrollment
- Peripheral blood count recovery: Neutrophil count ≥ 1000 /µL, platelet count ≥ 50x 109 /µL without platelet transfusions, and adequate hematocrit independent of red cell transfusions .
- No evidence of extramedullary leukemia, such as CNS or soft tissue involvement
- Adequate end organ function as measured by the following: AST and ALT < 4 x normal, total serum bilirubin < 2 x upper limit normal (unless due to hemolysis, Gilbert's syndrome, or ineffective erythropoiesis), creatinine < 2 x upper limit of normal
- Ability to give informed consent
- In agreement to use an effective barrier method of birth control to avoid pregnancy during the study and for a minimum of 30 days after study treatment, for all male and female patients who are fertile
Exclusion Criteria:
- Patients with untreated or uncontrolled infections
- Patients with untreated or uncontrolled grade 3 or 4 GVHD
- Pregnancy and lactation
- Concurrent use of any other investigational agents.
- Known HIV-positive patients.
- Known hypersensitivity to 5AC or GM-CSF
Studieplan
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Icke-randomiserad
- Interventionsmodell: Parallellt uppdrag
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Experimentell: Myeloablative BMT
Azacitidine and sargramostim after myeloablative stem cell transplant
|
Azacitidine will be administered days 1-5 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
Andra namn:
Sargramostim will be administered days 1-10 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
Andra namn:
|
|
Experimentell: Non-myeloablative BMT
Azacitidine and sargramostim after non-myeloablative stem cell transplant
|
Azacitidine will be administered days 1-5 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
Andra namn:
Sargramostim will be administered days 1-10 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
Andra namn:
|
|
Experimentell: Standard consolidation
Azacitidine and sargramostim after standard consolidation
|
Azacitidine will be administered days 1-5 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
Andra namn:
Sargramostim will be administered days 1-10 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
Andra namn:
|
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Two-year Relapse Free Survival of Patients
Tidsram: 2 year
|
To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.
|
2 year
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Hematologic Toxicity as Determined by Anemia
Tidsram: 1 year
|
Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim
|
1 year
|
|
One-year RFS
Tidsram: 1 year
|
We will report the number of participants with one year RFS
|
1 year
|
|
Overall Survival
Tidsram: 2 years
|
Percentage of participants with overall survival at 2 years.
|
2 years
|
Samarbetspartners och utredare
Samarbetspartners
Utredare
- Studiestol: Margaret Showel, MD, JHU
Studieavstämningsdatum
Studera stora datum
Studiestart (Faktisk)
Primärt slutförande (Faktisk)
Avslutad studie (Faktisk)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Uppskatta)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
- Neoplasmer efter histologisk typ
- Neoplasmer
- Benmärgssjukdomar
- Hematologiska sjukdomar
- Precancerösa tillstånd
- Myelodysplastiska syndrom
- Leukemi
- Leukemi, myeloid
- Leukemi, Myeloid, Akut
- Preleukemi
- Läkemedels fysiologiska effekter
- Molekylära mekanismer för farmakologisk verkan
- Enzyminhibitorer
- Antimetaboliter, antineoplastiska
- Antimetaboliter
- Antineoplastiska medel
- Immunologiska faktorer
- Azacitidin
- Sargramostim
Andra studie-ID-nummer
- J1240
- P01CA015396 (U.S.S. NIH-anslag/kontrakt)
- NA_00072223 (Annan identifierare: JHMIRB)
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
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