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Aplastic AnemiasHOPE ResourceNCT07227155

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

An observational study of Aplastic Anemias and Hemoglobinopathies, run by Medical College of Wisconsin at 14 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Any ageHealthy volunteers can take part
How many people
375 participants
Where
14 sitesUnited States
Study dates
15 May 2026to 30 Mar 2031
How long will I take part?
5 Years
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • Healthy volunteers accepted
  • Child, Adult, Older adult

Study population: Patients undergoing HCT or GT for treatment of Non-malignant blood disorders: Aplastic Anemia, Hemoglobinopathies or Bone Marrow Failure. Related donors of enrolled participants undergoing HCT are also eligible for the study.

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:
  2. 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.
  3. Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study.
  4. 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.
  5. 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.
  6. 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.
  7. 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.
  8. 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

Trial locations and contacts

14 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Megan Scott contact

301-251-1161

mscott@emmes.com

14 sites in 1 country

United States14 sites, 14 recruiting

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329

    Judson Russell, 404-785-7263, judson.russell@choa.org

    Shanmuganathan Chandrakasan, MD

    Recruiting

  • Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

    Morgan Kearney, 312-227-4067, mkearney@luriechildrens.org

    Sonali Chaudhury, MD

    Recruiting

  • City of Hope

    Duarte, California, 91010

    Miranda Lee, 626-218-4655, mirlee@coh.org

    Ryotaro Nakamura, MD

    Recruiting

  • Duke University Medical Center

    Durham, North Carolina, 27710

    Quinna Lawson, 916-613-0996, quinna.marshburn@duke.edu

    Mitchell Horwitz, MD

    Recruiting

  • University of Florida

    Gainesville, Florida, 32611

    Giselle Morre-Higgs, 352-273-9050, mooregj@ufl.edu

    Jordan Milner, MD

    Recruiting

  • Baylor College of Medicine

    Houston, Texas, 77030

    Deb Dowlin, 713-798-7371, deborah.dowlin@bcm.edu

    Alexander Ngwube, MD

    Recruiting

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226

    Kira Mielke, 414-955-5915, kmielke@mcw.edu

    Julie Talano, MD

    Recruiting

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37235

    Melanie Hallowell Hallowell, 615-875-0092, melanie.hallowell@vumc.org

    Jim Connelly, MD

    Recruiting

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

    Alyssa Kamrowski, 347-798-9213, kamrowsa@mskcc.org

    Roni Tamari, MD

    Recruiting

  • Oregon Health and Science University

    Portland, Oregon, 97239

    Denise Lackey, 503-494-8311, lackey@ohsu.edu

    Evan Shereck, MD

    Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia, 23298

    Mehreen Qureshi, 804-628-2229, mehreen.qureshi@vcuhealth.org

    Elizabeth Krieger, MD

    Recruiting

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109

    Sheri Ballard, 260-667-2814, sballard@fredhutch.org

    Neel Bhatt, MD

    Recruiting

  • St. Louis Children's

    St Louis, Missouri, 63110

    Lissy Keller, 314-286-1168, kellerl@wustl.edu

    Shalini Shenoy, MD

    Recruiting

  • Washington University

    St Louis, Missouri, 63110

    Maggie Nash, 314-273-5936, nashm@wustl.edu

    Iskra Pusic, MD

    Recruiting

Other trials in United States

About this study

Official title: Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (BMT CTN 2402)

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.

Read the detailed description

This is 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. The enrollment goal is 375 participants and up to all consenting 100 related donors (approximately 100 are anticipated), accrued over 4 years. Participants will be followed until the end of study, but continued collection of specimens and clinical data may continue to allow up to 15 years of follow-up for all enrolled participants if funds are available. Participants with a diagnosis of Aplastic Anemia or Hemoglobinopathies or bone marrow failure undergoing HCT or GT for management of their underlying disease. There is no treatment intervention in this study. Treatment will be at the provider's discretion. Participants will provide blood and other biospecimens pre and post-intervention at specified intervals. 375 participants and approximately 100 related donors will be enrolled from 35 sites.

Keywords
Biorepository, Biospecimens, Allogenic Transplant, Gene Therapy
Medical subject headings
Anemia, Aplastic, Hemoglobinopathies, Bone Marrow Failure Disorders

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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).

    Retention of biospecimen samples with DNA

    Measured: 5 Years

  2. Identify new therapeutic targets for NMD and HCT or GT related complications leading to development of more targeted and effective therapies.

    Retention of biospecimen samples with DNA

    Measured: 5 Years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Nov 2025
  2. Study start15 May 2026
  3. Primary completion30 Mar 2031 (estimated)
  4. Study completion30 Mar 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Case only
Time perspective
Prospective
Enrolment
375 participants (estimated)
Target duration
5 Years
IDs, dates and publications
ClinicalTrials.gov ID
NCT07227155
Sponsor's protocol ID
BMT CTN 2402
Acronym
HOPE Resource
Status last verified
Aug 2026
First submitted
7 Nov 2025
Last updated
27 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

An observational study of Aplastic Anemias and Hemoglobinopathies, run by Medical College of Wisconsin at 14 sites in United States.

The full registered description is in the About this study section and the official record.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 27 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Megan Scott (301-251-1161, mscott@emmes.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the 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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07227155.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2026; AzTrials last refreshed its copy on 26 Oct 2026.

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