NCT04965597 - Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

April 19, 2022

Primary Completion Date

February 28, 2026

Study Completion Date

March 31, 2026

Conditions

Bone Marrow Failure SyndromeCongenital Amegakaryocytic ThrombocytopeniaDiamond-Blackfan AnemiaHereditary Sideroblastic AnemiaParoxysmal Nocturnal HemoglobinuriaShwachman-Diamond SyndromeHematologic Neoplasm With Germline GATA2 MutationHematologic Neoplasm With Germline SAMD9 MutationHematologic Neoplasm With Germline SAMD9L Mutation

Interventions

DRUG

Treosulfan

Given IV

DRUG

Fludarabine Phosphate

Given IV

DRUG

Tacrolimus

Given IV and PO

DRUG

Methotrexate

Given IV

BIOLOGICAL

Lapine T-Lymphocyte Immune Globulin

Given IV

PROCEDURE

Peripheral Blood Stem Cell Transplantation

Undergo PBSC

PROCEDURE

Allogeneic Bone Marrow Transplantation

Undergo bone marrow transplant

OTHER

Quality-of-Life Assessment

Ancillary studies

PROCEDURE

Echocardiography

Undergo ECHO

PROCEDURE

Multigated Acquisition Scan

Undergo MUGA

PROCEDURE

Bone Marrow Biopsy

Undergo bone marrow biopsy and aspiration

PROCEDURE

Bone Marrow Aspiration

Undergo bone marrow biopsy and aspiration

PROCEDURE

Biospecimen Collection

Undergo blood sample collection

PROCEDURE

X-Ray Imaging

Undergo chest x-ray

PROCEDURE

Computed Tomography

Undergo chest CT

Trial Locations (24)

10065

Memorial Sloan Kettering Cancer Center, New York

11040

Cohen Children's Hospital of NY, Queens

14203

Roswell Park Comprehensive Cancer Center, Buffalo

19104

Children's Hospital of Philadelphia, Philadelphia

21231

Johns Hopkins University, Baltimore

27705

Duke University Medical Center, Durham

30329

Children's Healthcare of Atlanta, Atlanta

37232

Vanderbilt University Medical Center, Nashville

43205

Nationwide Children's Hospital, Columbus

45229

Cincinnati Children's Hospital, Cincinnati

48109

University of Michigan Medical Center, Ann Arbor

53226

Medical College of Wisconsin/Children's Hospital of Wisconsin, Milwaukee

55455

University of Minnesota, Minneapolis

63110

St. Louis Children's Hospital, St Louis

77030

MD Anderson Cancer Center, Houston

Texas Children's Hospital, Houston

80045

Children's Hospital Colorado, Aurora

84113

Primary Children's/University of Utah, Salt Lake City

90027

Children's Hospital Los Angeles, Los Angeles

92123

Rady Children's Hospital/UCSD, San Diego

94143

University of California San Francisco, San Francisco

97239

Oregon Health & Science University, Portland

98109

Fred Hutch/University of Washington Cancer Consortium, Seattle

02115

Boston Children's Hospital, Boston

All Listed Sponsors

collaborator

Blood and Marrow Transplant Clinical Trials Network

NETWORK

collaborator

National Cancer Institute (NCI)

NIH

collaborator

National Marrow Donor Program

OTHER

collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

lead

Fred Hutchinson Cancer Center

OTHER