Phase 2
N=40
Azacytidine Prior to in Vivo T-cell Depleted Allo Stem Cell Transplant for Patients With Myeloid Malignancies in CR
Leukemia, Erythroblastic, Acute · Myelodysplastic Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT02497404 ↗Enrolled (actual)
40
Serious AEs
70.0%
Results posted
Sep 2021
Primary outcome: Primary: Disease Free Survival at 1 Year Post-transplant — 18 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- 5-Azacytidine (Drug); Fludarabine (Drug); Melphalan (Drug); Alemtuzumab (Drug); Total Body Irradiation (Radiation)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Weill Medical College of Cornell University
- Primary completion
- Jun 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Disease Free Survival at 1 Year Post-transplant |
18 | — |
| SECONDARY Disease Free Survival at 6 Months Post-transplant |
25 | — |
| SECONDARY Disease Free Survival at 2 Years Post-transplant |
13 | — |
| SECONDARY Overall Survival at 6 Months Post-transplant |
35 | — |
| SECONDARY Overall Survival at 1 Year Post-Transplant |
25 | — |
| SECONDARY Overall Survival at 2 Years Post-Transplant |
15 | — |
| SECONDARY Graft Failure |
1 | — |
| SECONDARY Acute Graft-versus-Host Disease (GVHD) |
13 | — |
| SECONDARY High-Risk Extensive Chronic Graft-versus-Host-Disease |
2 | — |
Summary
The purpose of this study is to determine whether 5-Azacytidine priming before the conditioning regimen for subjects receiving a hematopoietic stem cell transplant is an effective treatment for high risk myeloid malignancies in complete remission (CR).
Eligibility Criteria
Inclusion Criteria
- Patients must have histologically or cytologically confirmed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) as specified below:
- Acute myeloid leukemia with poor risk cytogenetics in complete morphologic remission. These include: del (5q)/-5, del (7q)/-7, abn 3q, 9q, 11q, 20q, 21q, 17p, t(6;9), t(9;22) or complex karyotypes (≥ 3 unrelated abnormalities); or
- Acute myeloid leukemia with either Flt-3, TET-2, p53, DNMT3A, or ASXL1 mutation, mutations of genes involved in the chromatin/spliceosome category (EZH2, SRSF2, U2AF1, ZRSR2), BCOR, and RUNX1, as well as MLL rearrangement, EVI1 overexpression in complete morphologic remission; or
- Acute myeloid leukemia with a white blood cell count of greater than or equal to 50,000/mcL at presentation in first complete morphologic remission; or
- Acute myeloid leukemia in first complete morphologic remission, having required more than one course of induction chemotherapy to attain remission status; or
- Acute myeloid leukemia, all types, excluding M3 (Promyelocytic leukemia) in second or higher complete morphologic remission; or
- Myelodysplastic syndromes (intermediate-2, high risk and chronic myelomonocytic leukemia (CMML) with bone marrow blasts 60 mL/min (eGFR as estimated by the modified Modification of Diet in Renal Disease Study (MDRD) equation)
- Ability to understand and the willingness to sign a written informed consent document.
Exclusion Criteria
- Evidence of chronic active hepatitis or cirrhosis
- HIV infection
- Uncontrolled illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant and lactating women are excluded from the study because the risks to an unborn fetus or potential risks in nursing infants are unknown.
- There are no prior therapies or concomitant medications that would render the patients ineligible
Data sourced from ClinicalTrials.gov (NCT02497404). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.