Phase 3
N=256
Fludarabine-IV Busulfan ± Clofarabine and Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)
Disorder Related to Bone Marrow Transplantation · Leukemia · Transplantation Infection
Bottom Line
View on ClinicalTrials.gov: NCT01471444 ↗Enrolled (actual)
256
Serious AEs
25.6%
Results posted
Dec 2021
Primary outcome: Primary: Progression-Free Survival (PFS) — 69; 61 Number of events
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Fludarabine (Drug); Clofarabine (Drug); Busulfan (Drug); Thymoglobulin (Drug); Stem Cell Infusion (Procedure); Tacrolimus (Drug); Methotrexate (Drug)
- Age
- Pediatric, Adult, Older Adult · 3+ yrs
- Sex
- All
- Sponsor
- M.D. Anderson Cancer Center
- Primary completion
- Dec 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Progression-Free Survival (PFS) |
69; 61 | — |
| SECONDARY Overall Survival (OS) Post Transplant at 1, 3 and 5 Years |
83; 82; 69; 69; 64; 63 | — |
| SECONDARY Number of Participants in the Study Who Are With no Grade 3 or 4 Acute Graft-versus-host Disease at Any Time During the First 100 Days Post Transplant. |
125; 115 | — |
| SECONDARY Number of Participants With Non Relapse Mortality at 100 Day Post Transplant |
3; 6 | — |
Summary
The goal of this clinical research study is to learn if combining busulfan with clofarabine and fludarabine can help control the disease better than the previous standard method (using busulfan and fludarabine alone) in patients with AML or MDS. The safety of this combination therapy will also be studied.
Eligibility Criteria
Inclusion Criteria
- Patients must have one of the following hematologic malignancies: a) Acute myeloid leukemia (AML) any stage and cytogenetic risk-group with the only exception being that patients with AML and favorable cytogenetics (t(8;21, inv 16, or t(15;17) who achieve complete remission with one course of induction chemotherapy are not eligible . Patients with treatment related AML are eligible. b) Myelodysplastic syndromes (MDS) with intermediate or high risk International Prognostic Scoring System score (IPSS scores) or treatment related MDS. Patients with low risk MDS are eligible if they fail to respond to hypomethylating agent therapy such as azacitidine or decitabine.
- Age 3-70 years old. Eligibility for pediatric patients will be determined in conjunction with an MDACC pediatrician.
- Performance score of >/= 60 by Karnofsky or PS 0 to 2 (ECOG) (age > 12 years), or Lansky Play-Performance Scale >/= 60 or greater (age /=92% on room air by pulse oximetry.
- Creatinine 50 ml/min).
- Bilirubin /=12 mg/kg given by mouth or >/=10 mg/kg given IV; or a total-body irradiation (> 4 Gy).
- Active or prior CNS leukemia, unless in complete remission for at least 3 months.
- Previous therapeutic XRT to the liver as part of involved-field radiation.
- History of serious chronic mental disorder or drug-abuse accompanied by documented problems of compliance with therapeutic programs.
- Lack of care-giver for the early (100-day) post-transplant period.
Data sourced from ClinicalTrials.gov (NCT01471444). 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.