Allo vs Hypomethylating/Best Supportive Care in MDS (BMTCTN1102)

NCT02016781 | Completed Phase 2 Results DMC

• 3 other identifiers: BMTCTN11022U10HL069294-115U24CA076518
• Study Type: interventional
• Target: 384
• Locations: 1 country
• Sites: 37

Brief Summary

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.

Conditions

MDS

Keywords

Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Overall Survival (OS)

  The primary endpoint for this study is overall survival (OS) at three years post-consent. Death from any cause will be considered an event for this endpoint. Surviving participants are censored at the time of last follow-up. Three year OS estimates are adjusted for age, race/ethnicity, performance status, IPSS score, duration of disease, and response to prior hypomethylating therapy. The results posted are from the February 2020 interim analysis per protocol study design. Two interim analyses for efficacy were performed previously in January and November 2019 and presented to the Data and Safety Monitoring Board (DSMB). Results at the second analysis was crossing the efficacy boundary. Subsequently, the DSMB approved early release of study data as of February 2020.

  3 years

Secondary Outcomes (13)

• Percentage of Participants With Leukemia-free Survival (LFS)

  3 years

• Quality of Life (QOL) - Functional Assessment of Cancer Therapy-General (FACT-G)

  3 years

• Quality of Life (QOL) - Medical Outcomes Study Short Form (MOS SF-36)

  3 years

• Quality of Life (QOL) - EQ-5D

  3 years

• Percentage of Participants With Overall Survival (OS) in As-treated Population

  3 years

Study Arms (2)

Transplant

ACTIVE COMPARATOR

Reduced intensity conditioning allogeneic hematopoietic cell transplantation (RIC-alloHCT)

Procedure: Allogeneic Hematopoietic Cell Transplant

Hypomethylating Therapy / Best Supportive Care

ACTIVE COMPARATOR

The specific non-transplant treatment regimen will be at the discretion of the treating physician.

Procedure: Hypomethylating Therapy / Best Supportive Care

Interventions

Allogeneic Hematopoietic Cell Transplant PROCEDURE

Bone marrow or peripheral blood stem cell transplant.from a fully matched related (6/6) or unrelated (8/8) donor. The specific transplant treatment regimen will be at the discretion of the treating physician but is required to be reduced-intensity.

Also known as: RIC alloHCT

Transplant

Hypomethylating Therapy / Best Supportive Care PROCEDURE

The specific non-transplant treatment regimen will be at the discretion of the treating physician.

Also known as: Non-transplant

Hypomethylating Therapy / Best Supportive Care

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients fulfilling the following criteria will be eligible for entry into this study:
• 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.
• 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
• Patients must have fewer than 20% marrow blasts within 60 days of consent.
• 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.
• Age 50.0-75.0 years.
• Karnofsky performance status \> 70 or Eastern Cooperative Oncology Group (ECOG) ≤ 1.
• 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.

You may not qualify if:

• Patients with the following will be ineligible for registration onto this study:
• Therapy-related MDS (defined as the occurrence of MDS due to prior exposure to systemic chemotherapy and/or radiation for malignancy)
• Current or prior diagnosis of AML
• 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.
• 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.
• Prior autologous or allogeneic HCT
• Human Immunodeficiency Virus (HIV) infection
• Patients of childbearing potential unwilling to use contraceptive techniques
• Patients with psychosocial conditions that would prevent study compliance

Sponsors & Collaborators

• Medical College of Wisconsin: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator
• National Cancer Institute (NCI): collaborator
• Blood and Marrow Transplant Clinical Trials Network: collaborator
• National Marrow Donor Program: collaborator

Study Sites (37)

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

Stanford Hospital and Clinics

Stanford, California, 94305, United States

Location

University of Florida College of Medicine

Gainesville, Florida, 32610-0277, United States

Location

H. Lee Moffitt Cancer Center

Tampa, Florida, 33624, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

University of Chicago

Chicago, Illinois, 60637-1470, United States

Location

University of Kansas Hospital

Kansas City, Kansas, 66160, United States

Location

University of Kentucky

Lexington, Kentucky, 40536, United States

Location

University of Maryland Medical Systems - Greenebaum Cancer Center

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins

Baltimore, Maryland, 21231, United States

Location

Dana Farber Cancer Institute/Brigham & Women's

Boston, Massachusetts, 02115, United States

Location

Dana Farber Cancer Institute/Massachusetts General Hospital

Boston, Massachusetts, 02115, United States

Location

Karmanos Cancer Institute/BMT

Detroit, Michigan, 48201, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

Washington University/Barnes Jewish Hospital

St Louis, Missouri, 63110, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

Mount Sinai Hospital

New York, New York, 10029, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

University of North Carolina Hospital at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

Jewish Hospital BMT Program

Cincinnati, Ohio, 45236, United States

Location

University Hospitals of Cleveland/ Case Western

Cleveland, Ohio, 44106, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Ohio State/Arthur G. James Cancer Hospital

Columbus, Ohio, 43210, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239-3098, United States

Location

University of Pennsylvania Cancer Center

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-8210, United States

Location

Baylor College of Medicine/The Methodist Hospital

Houston, Texas, 77030, United States

Location

University of Texas/MD Anderson CRC

Houston, Texas, 77030, United States

Location

University of Utah Med School

Salt Lake City, Utah, 84132, United States

Location

Virginia Commonwealth University MCV Hospitals

Richmond, Virginia, 23298, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

West Virginia University Hospital

Morgantown, West Virginia, 26506, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53211, United States

Location

Related Publications (5)

• 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.

  PMID: 24972249 BACKGROUND

• Saber W, Bansal A, Li L, Scott BL, Sangaralingham LR, Thao V, Roth JA, Wright W, Steuten LMG, Pidala JA, Mishra A, Maziarz RT, Westervelt P, McGuirk JP, Cutler C, Nakamura R, Ramsey SD. Cost-Effectiveness of Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation for Older Patients With High-Risk Myelodysplastic Syndrome: Analysis of BMT CTN 1102. JCO Oncol Pract. 2024 Apr;20(4):572-580. doi: 10.1200/OP.23.00413. Epub 2024 Jan 23.

  PMID: 38261970 DERIVED

• Versluis J, Saber W, Tsai HK, Gibson CJ, Dillon LW, Mishra A, McGuirk J, Maziarz RT, Westervelt P, Hegde P, Mukherjee D, Martens MJ, Logan B, Horowitz M, Hourigan CS, Nakamura R, Cutler C, Lindsley RC; Blood and Marrow Transplant Clinical Trials Network. Allogeneic Hematopoietic Cell Transplantation Improves Outcome in Myelodysplastic Syndrome Across High-Risk Genetic Subgroups: Genetic Analysis of the Blood and Marrow Transplant Clinical Trials Network 1102 Study. J Clin Oncol. 2023 Oct 1;41(28):4497-4510. doi: 10.1200/JCO.23.00866. Epub 2023 Aug 22.

  PMID: 37607457 DERIVED

• Cusatis R, Martens MJ, Nakamura R, Cutler CS, Saber W, Lee SJ, Logan BR, Shaw BE, Gregory A, D'Souza A, Hamilton BK, Horowitz MM, Flynn KE. Health-related quality of life in reduced-intensity hematopoietic cell transplantation based on donor availability in patients aged 50-75 with advanced myelodysplastic syndrome: BMT CTN 1102. Am J Hematol. 2023 Feb;98(2):229-250. doi: 10.1002/ajh.26768. Epub 2022 Nov 21.

  PMID: 36251401 DERIVED

• Nakamura R, Saber W, Martens MJ, Ramirez A, Scott B, Oran B, Leifer E, Tamari R, Mishra A, Maziarz RT, McGuirk J, Westervelt P, Vasu S, Patnaik M, Kamble R, Forman SJ, Sekeres MA, Appelbaum F, Mendizabal A, Logan B, Horowitz M, Cutler C. Biologic Assignment Trial of Reduced-Intensity Hematopoietic Cell Transplantation Based on Donor Availability in Patients 50-75 Years of Age With Advanced Myelodysplastic Syndrome. J Clin Oncol. 2021 Oct 20;39(30):3328-3339. doi: 10.1200/JCO.20.03380. Epub 2021 Jun 9.

  PMID: 34106753 DERIVED

Related Links

• National Marrow Donor Program

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Adam Mendizabal, PhD
• Organization: The Emmes Company

amendizabal@emmes.com

(301) 251-1161

Study Officials

• Mary Horowitz, MD, MS

  Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: No parties are masked in this trial.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Two arms will enroll and have data collected on them simultaneously.

Study Record Dates

• First Submitted: December 16, 2013
• First Posted: December 20, 2013
• Study Start: December 16, 2013
• Primary Completion: October 5, 2021
• Study Completion: October 5, 2021
• Last Updated: March 3, 2023
• Results First Posted: July 29, 2022

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF
• Time Frame: Within 6 months of official study closure at participating sites.
• Access Criteria: Available to the public

Locations

US (37)