CompletedPhase 2/3

Stem Cell Transplant for Bone Marrow Failure Syndromes

United States10 participantsStarted 2000-06

Plain-language summary

The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.

Who can participate

Age range

35 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: * Patients eligible for transplantation under this protocol will be \<35 years of age, and will be diagnosed with: * a bone marrow failure syndrome unresponsive to available therapy, including but not limited to Diamond-Blackfan anemia, Shwachman Diamond syndrome or Kostmann's neutropenia but exclusive of aplastic anemia. * Diamond Blackfan Anemia: * Patients must show evidence of steroid resistance requiring equivalent of \>6 transfusions yearly despite steroid therapy. * Evidence of developing aplasia or myelodysplasia will also be criteria for transplantation. * Kostmann's Neutropenia, Shwachman-Diamond syndrome: * Patients must have been previously diagnosed as having a clinical picture characteristic of Shwachman-Diamond syndrome (exocrine pancreatic insufficiency, growth retardation, metaphyseal dysostosis, neutropenia), or must have a bone marrow aspirate consistent with Kostmann's neutropenia, with no evidence of acute leukemia. * Patients must have failed therapy with granulocyte-colony stimulating factor (G-CSF), as determined by an inability to maintain an absolute neutrophil count (ANC) \>750 cells/ml(3), or manifesting recurrent infections despite G-CSF administration resulting in life threatening infections or repeated hospitalizations (\<4 /year). Exclusion Criteria: * Patients \>35 years of age * Karnofsky score \<70% * Hepatic dysfunction as determined by bilirubin \>3.0, ALT \>150, or active hepatitis * Pulmonary function …

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.

1Since this trial studied stem cell transplantation specifically for bone marrow failure conditions like Diamond-Blackfan Anemia, Kostmann's Neutropenia, or Shwachman-Diamond Syndrome, can you tell me how the 2-year survival results turned out, and what that might mean for someone in my situation?
2Because this was a Phase 2/3 trial, what do we know so far about both the effectiveness and the safety profile of this transplant approach, and are there still unknowns I should be aware of?
3Given that this trial is now completed, have its findings changed how stem cell transplantation is used as a standard treatment for my specific diagnosis, or are there newer approaches that have emerged since?
4How does stem cell transplantation studied in this trial compare to the other treatment options currently available to me, like blood transfusions or growth factors, in terms of risks and long-term outcomes?
5Would my current health status, disease severity, or available donors make me a realistic candidate to discuss transplantation with my care team, based on what this type of trial typically required from participants?

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

Number of Patients Alive (Survival) at 2 Years

Timeframe: 2 years

Trial details

NCT IDNCT00176878

SponsorMasonic Cancer Center, University of Minnesota

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2009-03

Results submitted2009-06-18

View on ClinicalTrials.gov More Diamond-Blackfan Anemia trials

Who can participate

Age range

35 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.