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

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 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: April 18, 2016
Last verified: April 2016

December 16, 2013
April 18, 2016
December 2013
December 2016   (final data collection date for primary outcome measure)
Overall survival probabilities [ Time Frame: 3 years ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT02016781 on ClinicalTrials.gov Archive Site
  • Leukemia-free survival (LFS) [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • QOL measures [ Time Frame: 3 years ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Allo vs Hypomethylating/Best Supportive Care in MDS (BMT CTN 1102)
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)
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.

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.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
MDS
  • Procedure: Allogeneic Hematopoietic Cell Transplant
    Bone marrow or peripheral blood stem cell transplant. The specific transplant treatment regimen will be at the discretion of the treating physician.
    Other Name: RIC alloHCT
  • Procedure: Hypomethylating Therapy / Best Supportive Care
    The specific non-transplant treatment regimen will be at the discretion of the treating physician.
    Other Name: Non-transplant
  • Active Comparator: Transplant
    Reduced intensity conditioning allogeneic hematopoietic cell transplantation (RIC-alloHCT)
    Intervention: Procedure: Allogeneic Hematopoietic Cell Transplant
  • Active Comparator: Non-Transplant
    Hypomethylating Therapy / Best Supportive Care
    Intervention: Procedure: Hypomethylating Therapy / Best Supportive Care
Saber W, Le Rademacher J, Sekeres M, Logan B, Lewis M, Mendizabal A, Leifer E, Appelbaum FR, Horowitz MM, Nakamura R, Cutler CS. Multicenter biologic assignment trial comparing reduced-intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50 to 75 with intermediate-2 and high-risk myelodysplastic syndrome: Blood and Marrow Transplant Clinical Trials Network #1102 study rationale, design, and methods. Biol Blood Marrow Transplant. 2014 Oct;20(10):1566-72. doi: 10.1016/j.bbmt.2014.06.010. Epub 2014 Jun 24.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
400
December 2019
December 2016   (final data collection date for primary outcome measure)

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 an acceptable MDS subtype:

      • Refractory cytopenia with unilineage dysplasia (RCUD) (includes refractory anemia (RA))
      • Refractory anemia with ringed sideroblasts (RARS)
      • Refractory anemia with excess blasts (RAEB-1)
      • Refractory anemia with excess blasts (RAEB-2)
      • Refractory cytopenia with multilineage dysplasia (RCMD)
      • Myelodysplastic syndrome with isolated del(5q) (5q-syndrome)
      • Myelodysplastic syndrome (MDS), unclassifiable
    3. Patients must have fewer than 20% marrow blasts within 60 days of consent.
    4. 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 enrollment.
    5. Age 50.0-75.0 years.
    6. Karnofsky performance status > 70 or Eastern Cooperative Oncology Group (ECOG) ≤ 1.
    7. Patients are eligible if no formal unrelated donor search has been activated prior to date of consent. A formal unrelated donor search begins at the time at which samples are requested from potential National Marrow Donor Program (NMDP) donors. Patients who have started a sibling donor search or who have found a matched sibling donor are eligible.
    8. 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.
    9. 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.
    10. Signed informed consent

Exclusion Criteria:

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

    1. Therapy-related MDS (defined as the occurrence of MDS due to prior exposure to systemic chemotherapy and/or radiation for malignancy)
    2. Current or prior diagnosis of AML
    3. Chronic myelomonocytic leukemia or myelodysplastic/myeloproliferative neoplasm (unacceptable MDS subtypes); uncontrolled bacterial, viral or fungal infection (currently taking medication and with progression or no clinical improvement) at time of enrollment.
    4. Patients with prior malignancies, except treated non-melanoma skin cancer or treated cervical carcinoma in situ. Cancer treated with curative surgery without chemotherapy/radiation therapy > 5 years previously will be allowed. Cancer treated with curative surgery < 5 years previously will not be allowed unless approved by the Protocol Officer or one of the Protocol Chairs.
    5. Prior autologous or allogeneic HCT
    6. Human Immunodeficiency Virus (HIV) infection
    7. Patients of childbearing potential unwilling to use contraceptive techniques
    8. Patients with psychosocial conditions that would prevent study compliance
Both
50 Years to 75 Years   (Adult, Senior)
No
Contact: Heather Wittsack hwittsack@emmes.com
Contact: Adam Mendizabal, PhD amendizabal@emmes.com
United States
 
NCT02016781
BMTCTN1102, 2U10HL069294-11
Yes
Yes
Findings will be published in a manuscript.
Medical College of Wisconsin
Medical College of Wisconsin
  • National Heart, Lung, and Blood Institute (NHLBI)
  • National Cancer Institute (NCI)
  • Blood and Marrow Transplant Clinical Trials Network
Study Director: Mary Horowitz, MD, MS Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin
Medical College of Wisconsin
April 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP