ACTIVE_NOT_RECRUITING

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia). This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA. The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms. This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.

Official Title

A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

Quick Facts

Study Start:2023-01-25
Study Completion:2029-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05600426

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:0 Years to 25 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Provision of signed and dated informed consent form for the randomized trial by patient and/or legal guardian.
  2. 2. Age ≤25 years old at time of randomized trial consent.
  3. 3. Confirmed diagnosis of idiopathic SAA, defined as:
  4. 1. Bone marrow cellularity \<25%, or \<30% hematopoietic cells.
  5. 2. Two of three of the following (in peripheral blood): neutrophils \<0.5 x 10\^9/L, platelets \<20 x 10\^9/L, absolute reticulocyte count \<60 x 10\^9/L or hemoglobin \<8 g/dL.
  6. 4. No suitable fully matched related donor available (minimum 6/6 match for HLA-A and B at intermediate or high resolution and DRB1 at high resolution using DNA based typing).
  7. 5. At least 2 unrelated donors noted on NMDP search who are well matched (9/10 or 10/10 for HLA-A, B, C, DRB1, and DQB1 using high resolution).
  8. 6. In the treating physician's opinion, no obvious contraindications precluding them from BMT or IST.
  1. 1. Presence of Inherited bone marrow failure syndromes (IBMFS). The diagnosis of Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow. Telomere length testing should be sent on all patients to exclude Dyskeratosis Congenita (DC), but if results are delayed or unavailable and there are no clinical manifestations of DC, patients may enroll. If patients have clinical characteristics suspicious for Shwachman-Diamond syndrome, this disorder should be excluded by pancreatic isoamylase testing or gene mutation analysis (note: pancreatic isoamylase testing is not useful in children \<3). Other testing per center may be performed to exclude IBMFS.
  2. 2. Clonal cytogenetic abnormalities or Fluorescence In-Situ Hybridization (FISH) pattern consistent with pre- myelodysplastic syndrome (pre-MDS) or MDS on marrow examination.
  3. 3. Known severe allergy to ATG.
  4. 4. Prior allogeneic or autologous stem cell transplant.
  5. 5. Prior solid organ transplant.
  6. 6. Infection with human immunodeficiency virus (HIV).
  7. 7. Active Hepatitis B or C. This only needs to be excluded in patients where there is clinical suspicion of hepatitis (e.g., elevated LFTs).
  8. 8. Female patients who are pregnant or breast-feeding.
  9. 9. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ.
  10. 10. Disease modifying treatment prior to study enrollment, including but not limited to use of androgens, eltrombopag, romiplostim, or immune suppression. Note: Supportive care measures such as G-CSF, blood transfusion support and antibiotics are allowable

Contacts and Locations

Principal Investigator

David Williams, MD
PRINCIPAL_INVESTIGATOR
Boston Children's Hospital
Michael Pulsipher, MD
PRINCIPAL_INVESTIGATOR
University of Utah
Bronwen Shaw, MD
PRINCIPAL_INVESTIGATOR
CIBMTR/Medical College of Wisconsin (MCW)

Study Locations (Sites)

University of Alabama at Birmingham
Birmingham, Alabama, 35249
United States
Phoenix Children's Hospital
Phoenix, Arizona, 85016
United States
Arkansas
Little Rock, Arkansas, 72202
United States
Loma Linda
Loma Linda, California, 92354
United States
Children's Hospital Los Angeles
Los Angeles, California, 90027
United States
UCLA
Los Angeles, California, 90095
United States
Children's Hospital & Research Center Oakland
Oakland, California, 94609
United States
Children's Hospital of Orange County
Orange, California, 92868
United States
Stanford
Palo Alto, California, 94304
United States
Rady Children's Hospital San Diego
San Diego, California, 92123
United States
University of California San Francisco
San Francisco, California, 94158
United States
Children's Hospital Colorado
Aurora, Colorado, 80045
United States
Yale University
New Haven, Connecticut, 06520
United States
Nemours Children's Hospital, Delaware
Wilmington, Delaware, 19803
United States
Children's National Hospital
Washington D.C., District of Columbia, 20010
United States
University of Florida
Gainesville, Florida, 32610
United States
University of Miami
Miami, Florida, 33136
United States
Nicklaus Children's Hospital
Miami, Florida, 33155
United States
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
United States
Children's Hospital of Atlanta/Emory
Atlanta, Georgia, 30322
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
University of Chicago
Chicago, Illinois, 60637
United States
Indiana University Hospital/Riley Hospital for Children
Indianapolis, Indiana, 46202
United States
Children's Hospital NOLA
New Orleans, Louisiana, 70118
United States
Maine Health
Scarborough, Maine, 04074
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
University of Michigan
Ann Arbor, Michigan, 48109
United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
United States
Mayo Clinic Rochestser
Rochester, Minnesota, 55902
United States
University of Mississippi Medical Center
Jackson, Mississippi, 39216
United States
Washington University in St. Louis
St Louis, Missouri, 63110
United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601
United States
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263
United States
Cohen Children's Medical Center of New York
New Hyde Park, New York, 11040
United States
Columbia University Medical Center
New York, New York, 10032
United States
University of North Carolina
Chapel Hill, North Carolina, 27599
United States
Levine Children's Hospital
Charlotte, North Carolina, 28203
United States
Duke University
Durham, North Carolina, 27705
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
Oregon Health & Science University
Portland, Oregon, 97239
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37203
United States
Children's Medical Center Dallas
Dallas, Texas, 75235
United States
Texas Children's Hospital
Houston, Texas, 77030
United States
Methodist Healthcare
San Antonio, Texas, 78229
United States
University of Utah/Primary Children's Hospital
Salt Lake City, Utah, 84112
United States
Children's Hospital of the King's Daughter
Norfolk, Virginia, 23507
United States
Fred Hutchinson Cancer Center
Seattle, Washington, 98109
United States
University of Wisconsin
Madison, Wisconsin, 53792
United States

Collaborators and Investigators

Sponsor: Boston Children's Hospital

  • David Williams, MD, PRINCIPAL_INVESTIGATOR, Boston Children's Hospital
  • Michael Pulsipher, MD, PRINCIPAL_INVESTIGATOR, University of Utah
  • Bronwen Shaw, MD, PRINCIPAL_INVESTIGATOR, CIBMTR/Medical College of Wisconsin (MCW)

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-01-25
Study Completion Date2029-12

Study Record Updates

Study Start Date2023-01-25
Study Completion Date2029-12

Terms related to this study

Additional Relevant MeSH Terms

  • Severe Aplastic Anemia