Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young P… (NCT00301834) | Clinical Trial Compass
CompletedPhase 2
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
United States35 participantsStarted 2005-01
Plain-language summary
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Drugs used in chemotherapy, such as fludarabine and busulfan, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell, bone marrow , or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine together with methotrexate and methylprednisolone may stop this from happening.
PURPOSE: This phase II trial is studying how well giving alemtuzumab together with fludarabine and busulfan works when given before donor stem cell transplant in treating young patients with hematologic disorders.
Who can participate
Age range
21 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.
DISEASE CHARACTERISTICS:
* Diagnosis of 1 of the following hematologic conditions:
* Aplastic anemia with marrow aplasia, meeting all of the following criteria:
* Absolute neutrophil count \< 500/mm\^3
* Platelet and/or red cell transfusion dependent
* Chronic aplastic anemia, meeting all of the following criteria:
* Transfusion dependent
* Unresponsive to immunosuppressive therapy
* Alternative matched unrelated donor has been identified
* Congenital marrow failure syndrome, including any of the following (with closely matched related or unrelated donor):
* Primary red cell aplasia (Diamond-Blackfan syndrome)
* Congenital neutropenia (Kostmann's syndrome)
* Amegakaryocytic thrombocytopenia
* Congenital dyserythropoietic anemias
* Other severe acquired cytopenias in which a transplantation using a combined busulfan/cyclophosphamide conditioning regimen is indicated
* Hemoglobinopathy (with closely matched related or unrelated donor)
* β-thalassemia major
* Sickle cell anemia
* Hemoglobin E/β-thalassemia
* Severe immunodeficiency disease
* Chediak-Higashi disease
* Wiskott-Aldrich syndrome
* Combined immunodeficiency disease (Nezelof's)
* Hyper immunoglobulin M (IgM) syndrome
* Bare lymphocyte syndrome
* Chronic granulomatous disease
* Familial erythrohemophagocytic lymphohistiocytosis
* Other stem cell defects (e.g., osteopetrosis)
* Severe immune dysregulation/autoimmune diso…
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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
1
Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation