Protocol Summary

Protocol No.: OCR13125

Sponsor Protocol No.: 1102

Study Title
A Multi-Center Biologic Assignment Trial Comparing Reduced Intensity Allogeneic Hematopoietic Cell Transplant to Hypomethylating Therapy or Best Supportive Care in Patients Aged 50-75 with Intermediate-2 and High Risk Myelodysplastic Syndrome (BMT CTN #1102)

Principal Investigator(s)
Wingard, John

Objective
This study is being conducted to compare the benefits of reduced-intensity allogeneic stem cell transplant with the benefits of non-transplant therapies (chemotherapy alone, blood transfusion support, etc.).

Description
MDS is a clonal disorder of hematopoietic precursors and stem cells, which may evolve to a terminal phase resembling acute leukemia. A subject of clinical urgency for researchers, clinicians, patients, and health care underwriters such as Medicare, is the role of allogeneic hematopoietic cell transplantation (alloHCT) in the treatment of older patients with higher risk myelodysplastic syndromes (MDS). The use of reduced intensity conditioning (RIC) regimens has extended HCT to the care of older patients with acute myelogenous leukemia (AML) and lymphoma and a number of retrospective and phase II trials for patients with MDS now show the curative potential of RIC alloHCT in selected patients.

This protocol is designed to evaluate the relative benefits of RIC alloHCT compared to non-transplant therapies focusing on overall survival. This will be done by having patients biologically assigned to the alloHCT arm or the hypomethylating therapy/best supportive care arm and following them for survival at 3 years.

Phase: NA

Age Group: Adult

Scope: National

Treatment
Arm 1: Reduced intensity conditioning allogeneic hematopoietic cell transplantation (RIC-alloHCT)

Arm 2: Non-Transplant Therapy/Best Supportive Care.

Detailed Eligibility
Inclusion Criteria:
1. Patients with de novo MDS who have, or have previously had, Intermediate-2 or High risk disease as determined by the International Prognostic Scoring System (IPSS). (Current Intermediate-2 or High risk disease is NOT a requirement.)
2. Patients must have fewer than 20% marrow blasts at the time (within 30 days) of registration and consent.
3. Patients may have received prior therapy for the treatment of MDS, including but not limited to: growth factor, transfusion support, immunomodulatory (IMID) therapy, DNA hypomethylating therapy or cytotoxic chemotherapy prior to trial registration.
4. Age 50.0-75.0 years.
5. Karnofsky performance status > 70 or Eastern Cooperative Oncology Group (ECOG) =6. Patients are eligible if no formal unrelated donor search has been activated prior to enrollment. Patients who have started a sibling donor search or who have found a matched sibling donor are eligible.
7. Patients and physicians must be willing to comply with treatment assignment:
8. No intent to proceed with alloHCT using donor sources not specified in this protocol, including Human leukocyte antigen (HLA)-mismatched related or unrelated donors 9. No intent to use myeloablative conditioning regimens.
10. Intent to proceed with RIC alloHCT if a matched sibling or matched unrelated donor is identified. There is no requirement as to the timing of the transplantation.
11. Patients must be considered to be suitable RIC alloHCT candidates at the time of enrollment based on medical history, physical examination and available laboratory tests. (Specific testing for organ function is not required for eligibility but, if available, these tests should be used to judge eligibility.)
12. Signed informed consent

Exclusion Criteria:
1. Patients with the following will be ineligible for registration onto this study:
-Therapy-related MDS
-Current or prior diagnosis of AML
-Uncontrolled bacterial, viral or fungal infection
-Concurrent malignancy other than superficial squamous cell or basal cell carcinoma of the skin
-Prior autologous or allogeneic HCT
-Human Immunodeficiency Virus (HIV) infection
-Fertile patients unwilling to use contraceptive techniques
-Patients with psychosocial conditions that would prevent study compliance

Applicable Disease Sites
Myelodysplastic Syndrome (MDS)

Therapies Involved
Stem Cell Transplant

Drugs Involved
5-azacytidine (Azacitidine)
Azacitidine
Vidaza (Azacitidine)

Participating Institutions
UF Gainesville : Meghan Vazquez

Contact
Meghan Vazquez, RN
Phone: +1 352-273-6843

Email: meghanvazquez@ufl.edu

More Information:
View study listing on ClinicialTrials.gov
http://www.clinicaltrials.gov/ct2/show/
NCT02016781