Allo vs Hypomethylating/Best Supportive Care in MDS (BMT CTN 1102)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2016 by Medical College of Wisconsin
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
National Cancer Institute (NCI)
Blood and Marrow Transplant Clinical Trials Network
Information provided by (Responsible Party):
Medical College of Wisconsin
ClinicalTrials.gov Identifier:
NCT02016781
First received: December 16, 2013
Last updated: February 5, 2016
Last verified: February 2016
  Purpose
This study is designed as a multicenter trial, with biological assignment to one of two study arms; Arm 1: Reduced intensity conditioning allogeneic hematopoietic cell transplantation (RIC-alloHCT), Arm 2: Non-Transplant Therapy/Best Supportive Care.

Condition Intervention
MDS
Procedure: Allogeneic Hematopoietic Cell Transplant
Procedure: Hypomethylating Therapy or Best Supportive Care

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Multi-Center Biologic Assignment Trial Comparing Allogeneic Hematopoietic Cell Transplant to Hypomethylating Therapy or Best Supportive Care in Patients w/Intermediate-2 & High Risk Myelodysplastic Syndrome (BMT CTN #1102)

Resource links provided by NLM:


Further study details as provided by Medical College of Wisconsin:

Primary Outcome Measures:
  • Overall survival probabilities [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Leukemia-free survival (LFS) [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • QOL measures [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA
Patient peripheral blood and buccal swab samples will be collected at enrollment. Peripheral blood (and bone marrow samples if available) will also be collected from patients assigned to the HCT arm who experience relapse at the time of relapse and stored to support future research studies. If available, bone marrow will be collected pre-transplant for the patients assigned to the HCT arm.

Estimated Enrollment: 400
Study Start Date: December 2013
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Myelodysplastic Syndrome
Allogeneic Hematopoietic Cell Transplant with Hypomethylating Therapy or Best Supportive Care
Procedure: Allogeneic Hematopoietic Cell Transplant
Bone marrow or peripheral blood stem cell transplant
Procedure: Hypomethylating Therapy or Best Supportive Care
All supportive care will be given in keeping with local institutional guidelines

Detailed Description:

Background: 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.

  Eligibility

Ages Eligible for Study:   50 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
De novo MDS patient between the ages of 50 and 75 years with no prior history of allo HCT.
Criteria

Inclusion Criteria:

  • Patients fulfilling the following criteria will be eligible for entry into this study:

    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) ≤ 1 (see comparison scale in Appendix D).
    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:

      1. No intent to proceed with alloHCT using donor sources not specified in this protocol, including Human leukocyte antigen (HLA)-mismatched related or unrelated donors < 6/6 HLA related matched or < 8/8 HLA unrelated matched) or umbilical cord blood unit(s).
      2. No intent to use myeloablative conditioning regimens.
      3. 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.
    8. 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.
    9. Signed informed consent

Exclusion Criteria:

  • Patients with the following will be ineligible for registration onto this study:

    1. Therapy-related MDS
    2. Current or prior diagnosis of AML
    3. Uncontrolled bacterial, viral or fungal infection
    4. Concurrent malignancy other than superficial squamous cell or basal cell carcinoma of the skin
    5. Prior autologous or allogeneic HCT
    6. Human Immunodeficiency Virus (HIV) infection
    7. Fertile patients unwilling to use contraceptive techniques
    8. Patients with psychosocial conditions that would prevent study compliance
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02016781

Contacts
Contact: Heather Wittsack hwittsack@emmes.com
Contact: Adam Mendizabal, PhD amendizabal@emmes.com

  Show 36 Study Locations
Sponsors and Collaborators
Medical College of Wisconsin
National Heart, Lung, and Blood Institute (NHLBI)
National Cancer Institute (NCI)
Blood and Marrow Transplant Clinical Trials Network
Investigators
Study Director: Mary Horowitz, MD, MS Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin
  More Information

Additional Information:
No publications provided

Responsible Party: Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT02016781     History of Changes
Other Study ID Numbers: BMTCTN1102  2U10HL069294-11 
Study First Received: December 16, 2013
Last Updated: February 5, 2016
Health Authority: United States: Federal Government
United States: Food and Drug Administration

Keywords provided by Medical College of Wisconsin:
Myelodysplastic Syndrome

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Bone Marrow Diseases
Hematologic Diseases
Neoplasms
Precancerous Conditions

ClinicalTrials.gov processed this record on February 08, 2016