CompletedPhase 2

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

United States40 participantsStarted 2022-04-19

Plain-language summary

This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.

Who can participate

Age range

1 Year – 49 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Patient must be \>= 1.0 year of age and less than 50.0 years of age at the time of enrollment (i.e. patient must have celebrated their 1st birthday when enrolled and must NOT have celebrated their 50th birthday when enrolled; 49.99 years) * Underlying BMFD treatable by allogenic HCT * Shwachman-Diamond syndrome * Criteria for Diagnosis: * A pathogenic mutation(s) for Shwachman-Diamond syndrome * For those patients tested but lacking a genetic mutation they must meet both \*\*\* criteria below: * Exocrine pancreatic dysfunction as defined by at least one of the following: * Pancreatic isoamylase below normal (age \>= 3 years old), OR * Fecal elastase \< 200, AND * Bone marrow failure as evidence by at least one of the following: * Intermittent or persistent neutropenia (absolute neutrophil count \< 1,500/uL), OR * Hypo-productive anemia with a hemoglobin concentration below the age-related adjusted norms, OR * Unexplained macrocytosis, OR * Platelet count \< 150,000/uL without alternative etiology, OR * Hypocellular bone marrow * Indications for HCT: * Severe neutropenia (absolute neutrophil count \[ANC\] \< 500/uL), OR * Severe anemia (hemoglobin \< 8 g/dL) or transfusion-dependent anemia, OR * Severe thrombocytopenia (platelet count \< 20,000/uL) or transfusion-dependent thrombocytopenia, OR * Additional clinical or laboratory data may be considered fo…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1This trial used a treosulfan-based conditioning regimen instead of more traditional chemotherapy approaches before transplant — can you explain how treosulfan works differently and why that might or might not matter for my specific diagnosis?
2The trial is now completed but results may not be fully published yet — do you have access to any preliminary findings on GVHD-free event-free survival, and what would those results mean for someone in my situation?
3Since this was a Phase 2 trial, the safety and effectiveness data are still more limited than a Phase 3 study — how does that uncertainty factor into whether this approach is something you'd consider for my care right now?
4This trial covered a range of bone marrow failure conditions including Diamond-Blackfan Anemia, PNH, and GATA2-related disorders — given my specific diagnosis, do you think the results from this study would be relevant to my case, or were outcomes likely different across those subgroups?
5Graft-versus-host disease is one of the most serious risks after a bone marrow transplant — based on what this trial was measuring, can you walk me through what the realistic risks of GVHD still look like with a treosulfan conditioning approach compared to standard options?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Graft-Versus Host-Disease (GVHD)-Free Event-Free Survival (EFS)

Timeframe: 1 year post-HCT

Trial details

NCT IDNCT04965597

SponsorFred Hutchinson Cancer Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-02-18

View on ClinicalTrials.gov More Bone Marrow Failure Syndrome trials