Recruiting

HCT & GT

Sponsor:

Medical College of Wisconsin

Code:

NCT07227155

Conditions

Aplastic Anemias

Hemoglobinopathies

Bone Marrow Failure

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Accepted

Study Details

Brief summary:

A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases.

Conditions

Aplastic Anemias

Hemoglobinopathies

Bone Marrow Failure

Study ID

NCT07227155

Start date

May 15, 2026

Status verified date

Aug, 2026

Completion date

Mar 30, 2031

Anticipated

Primary completion date

Mar 30, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Accepted

HCT/GT Inclusion:

1. Patients with a diagnosis of Aplastic Anemia (AA), hemoglobinopathies or bone marrow failure from other causes except for malignant diseases will be eligible for enrollment on this protocol:

1. AA will be defined as having peripheral blood cytopenias with a hypocellular bone marrow for age and a clinical diagnosis of aplastic anemia as determined by their treating physicians.
2. Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study.
3. Individuals with bone marrow failure due to clinical or molecularly diagnosed inherited bone marrow failure, inborn errors of immunity or other cause will be included.
2. Patients must receive an HCT or GT for management of their underlying disease. Allogeneic transplants including all conditioning regimens, donors, and GVHD prophylaxis regimens are eligible. This study does not define how the transplant or transplant-supportive care will be performed.
3. Patients or their legal guardian must consent to participate in the CIBMTR "Protocol for a Research Database for Hematopoietic Cell Transplantation and Marrow Toxic Injuries" (NCT 1166009) to allow linkage with the longitudinal clinical data collected by CIBMTR.
4. All ages minorities, sexes and genders are eligible for the study, but participants must weigh at least 10 kilograms (kg) at the time of study enrollment given the volume and number of blood draws required.
5. All participants or parent/legal guardian must sign an informed consent for this study. If there are questions regarding a patient's eligibility for the study, contact the Protocol Team for review and discussion by emailing bmtctn2402@emmes.com.

HCT/GT Exclusion

1. Patients with aplastic anemia or hemoglobinopathies who are not pursuing allogeneic HCT or GT.
2. Active malignancy.
3. Hematologic malignancy or therapy for a prior hematologic malignancy in the previous five 5 years.
4. Weight ≤ 10.0 kg at time of study enrollment.
5. Prior autologous or allogeneic transplant.

Related Donor Inclusion:

1\. All related donors for eligible recipients undergoing allogeneic HCT for AA, hemoglobinopathies, or bone marrow failure as defined in the recipient eligibility criteria above are eligible. Note: HCT recipient participants will remain eligible if the related donor declines to participate in the study.

Related Donor Exclusion:

1\. Donor weight ≤ 10.0 kg at time of study enrollment

Study Design

Enrollment

375 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

  • Develop a biorepository that will establish mechanistic insights and biologic correlates to key casues of fialure of HCT and GT for Non-malignant Diseases (NMD). [ Time Frame: 5 Years ]
  • Identify new therapeutic targets for NMD and HCT or GT related complications leading to development of more targeted and effective therapies. [ Time Frame: 5 Years ]

Central Contacts and Locations

Central contacts

Locations

City of Hope

Recruiting

Duarte, California, United States, 91010

Contacts

Principal Investigator:

Ryotaro Nakamura, MD

University of Florida

Recruiting

Gainesville, Florida, United States, 32611

Contacts

Giselle Morre-Higgs

352-273-9050mooregj@ufl.edu

Principal Investigator:

Jordan Milner, MD

Children's Healthcare of Atlanta

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

Principal Investigator:

Shanmuganathan Chandrakasan, MD

Lurie Children's Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Principal Investigator:

Sonali Chaudhury, MD

St. Louis Children's

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Principal Investigator:

Shalini Shenoy, MD

Washington University

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Principal Investigator:

Iskra Pusic, MD

Memorial Sloan Kettering Cancer Center

Recruiting

New York, New York, United States, 10065

Contacts

Principal Investigator:

Roni Tamari, MD

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Principal Investigator:

Mitchell Horwitz, MD

Oregon Health and Science University

Recruiting

Portland, Oregon, United States, 97239

Contacts

Principal Investigator:

Evan Shereck, MD

Vanderbilt University Medical Center

Recruiting

Nashville, Tennessee, United States, 37235

Contacts

Melanie Hallowell Hallowell

615-875-0092melanie.hallowell@vumc.org

Principal Investigator:

Jim Connelly, MD

Baylor College of Medicine

Recruiting

Houston, Texas, United States, 77030

Contacts

Principal Investigator:

Alexander Ngwube, MD

Virginia Commonwealth University

Recruiting

Richmond, Virginia, United States, 23298

Contacts

Principal Investigator:

Elizabeth Krieger, MD

Fred Hutchinson Cancer Center

Recruiting

Seattle, Washington, United States, 98109

Contacts

Principal Investigator:

Neel Bhatt, MD

Children's Hospital of Wisconsin

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Principal Investigator:

Julie Talano, MD

More Information

Sponsor

Medical College of Wisconsin

Last update posted

Aug 27, 2026

Last verified

Aug, 2026

Keywords

  • Biorepository
  • Biospecimens
  • Allogenic Transplant
  • Gene Therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Medical College of Wisconsin on 2026-08-27.