Phase 2
N=11
Allogeneic Hematopoietic Stem Cell Transplantation (AlloSCT) Initial Salvage Therapy for Induction Failure Acute Myeloid Leukemia (AML)
Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT02441803 ↗Enrolled (actual)
11
Serious AEs
45.5%
Results posted
Jan 2024
Primary outcome: Primary: Overall Response (OR) Post Transplant — 1; 6; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Busulfan (Drug); Fludarabine (Drug); Clofarabine (Drug); Total Body Irradiation (TBI) (Radiation); Thymoglobulin (Drug); Stem Cell Infusion (Biological); Cyclophosphamide (Drug); Tacrolimus (Drug); Mycophenolate mofetil (Drug); Decitabine (Drug); Cytarabine (Drug); Idarubicin (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- M.D. Anderson Cancer Center
- Primary completion
- Sep 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Response (OR) Post Transplant |
1; 6; 0 | — |
| PRIMARY Treatment-Related Mortality (TRM) |
0; 1; 0 | — |
| SECONDARY Overall Survival (OS) |
0; 1; 0 | — |
Summary
Objectives:
Primary Objectives:
1. To determine the safety and feasibility of allogeneic hematopoietic stem cell transplantation (AHSCT) as initial salvage treatment for patients with primary induction failure (PIF) acute myeloid leukemia (AML).
2. To determine efficacy of AHSCT following decitabine, clofarabine, idarubicin, and cytarabine (DCIA) salvage chemotherapy evaluated by overall response rate (RR), defined as complete response (CR) or CR without platelet recovery (CRp) or CR with insufficient hematological recovery (CRi).
Secondary Objectives:
1. To determine the percentage of patients with PIF AML eligible for AHSCT after up to 2 courses of induction chemotherapy.
2. To determine the early treatment-related mortality (TRM) (within first 4 weeks of first salvage chemotherapy regimen with DCIA and day 100 TRM after AHSCT.
3. To determine the efficacy DCIA regimen as salvage chemotherapy for patients with PIF AML (% of patients who achieve </=5% bone marrow blasts prior to AHSCT.
4. To determine the TRM at 1 year, relapse rate (RR), overall survival (OS) and event-free survival (EFS) for patients with PIF AML treated with DCIA followed by early AHSCT.
Eligibility Criteria
Inclusion Criteria
- Patients age 18-60 years.
- Patients with diagnosis of AML, judged primary refractory after up to 2 courses of AML induction therapy (> 5% blasts on day 21 (+/-7 days) bone marrow aspirate and/or biopsy from the beginning of induction chemotherapy, up to 42 days).
- Eastern Cooperative Oncology Group (ECOG) Performance Status /= 40% (by either ECHO or MUGA).
- Willingness to have an allogeneic transplant.
- Patient or patient's legal representative able to provide written informed consent.
- Patients are required to meet the following criteria to proceed to AHSCT:
- Donor criteria: Availability of a donor either an HLA matched sibling donor (MSD) or a haploidentical (5-9/10 HLA matched); alternatively a 8/8 HLA matched unrelated donor (MUD) by high resolution typing is immediately available;
- Disease criteria: Day 21 (+/-7 days) bone marrow aspiration or biopsy from the beginning of salvage DCIA: a. In complete morphologic remission with 1,000/µL) and /= 50 ml/min (calculated by Cockcroft-Gault formula); b. Total bilirubin /= 40% on ECHO or MUGA; e. DLCO >/= 50% predicted after correction for hemoglobin (must be performed in patients with history of smoking or lung disease;); DLCO may be omitted in patients without history of pulmonary disease if approved by the Study Chair.
- No active infection: Patients should be afebrile. If present, pulmonary infiltrates or other sites of infection must be improving on antibiotics. Patients should not require oxygen. Study Chair will be the arbiter of this criterion.
Exclusion Criteria
- HIV positive; active hepatitis B or C.
- Uncontrolled active infections (viral, bacterial, and fungal); the Study Chair will be the final arbiter of this criterion.
- Patients with active secondary malignancy unless approved by the Study Chair.
- Liver cirrhosis.
- Active CNS involvement within the previous 2 months.
- Prior induction therapy with DAC + CIA.
- Positive pregnancy test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.
- Breast feeding women.
- Men must agree not to father a child and agree to use a condom if his partner is of child bearing potential.
- Inability to comply with medical therapy or follow-up.
Data sourced from ClinicalTrials.gov (NCT02441803). 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.