Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Stopped: drug company is no longer making the drug
United States3 participantsStarted 2010-09
Plain-language summary
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications. Much of the risk stems from the use of high dose radiation and chemotherapy for conditioning, the treatment administered just prior to transplant that eliminates the patients' marrow and immune system, effectively preventing rejection of the donors' cells. Attempts to make blood and marrow transplantation safer for children with non-malignant diseases by using lower doses of radiation and chemotherapy have largely failed because of a high rate of graft rejection.
In many such cases, it is likely that the graft is rejected because the recipient is sensitized to proteins on donor cells, including bone marrow cells, by blood transfusions. The formation of memory immune cells is a hallmark of sensitization, and these memory cells are relatively insensitive to chemotherapy and radiation. Alefacept, a drug used to treat psoriasis, on the other hand, selectively depletes these cells. The investigators are conducting a pilot study to begin to determine whether incorporating alefacept into a low dose conditioning regimen can effectively mitigate sensitization and, thereby, prevent rejection of allogeneic blood and marrow transplants for multiply transfused children with non-malignant hematological diseases.
Who can participate
Age range
21 Years
Sex
ALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Must be between the ages of 0-21 years at the time of admission for transplant
. Must have been transfused with at least five platelet, erythrocyte or granulocyte units (partial or full)
. Must have one of the following diseases:
. Must have an available HLA identical sibling (HLA matched related), a non-HLA identical parent or sibling who is matched at least seven of eight loci (mismatch can be at an allele or antigen level), an unrelated adult donor who is matched at least seven of eight loci (mismatch can be at an allele or antigen level) or an unrelated cord blood unit that is matched at five of six loci (A (antigen level), B (antigen level), DRB1 (allele level)) and provides a minimum pre-cryopreservation TNC dose of 5.0 x 107 TNC/kg recipient weight.
Exclusion criteria
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 was terminated before completing — do you know why it was stopped, and does that affect whether alefacept-based pre-conditioning is still considered a viable option for my condition?
2Since the trial was measuring 'full donor engraftment' as its main goal, how confident can we be in alefacept's ability to help the transplanted cells take hold, given that the study didn't finish collecting that data?
3This trial covered a wide range of blood and immune disorders — for my specific diagnosis, is there other completed research on alefacept or similar pre-conditioning approaches that might give us more reliable safety and effectiveness information?
4Because this trial was listed as Phase NA, meaning it was likely an early feasibility study, what would it mean for my safety to consider a treatment approach that hasn't been fully tested through standard trial phases?
5Given that this trial was terminated, would you recommend I look at a different active clinical trial or consider standard transplant pre-conditioning regimens instead, and how do those options compare for someone with my diagnosis?
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
Feasibility of Alefacept Pre-conditioning, Measured by Number of Subjects With Full Donor Engraftment
. Hemophagocytic lymphohistiocytosis or other disorder characterized by NK cell dysfunction, since alefacept's effect is mediated by NK cells.
. Biopsy proven cirrhosis (score IV).
. SCD chronic lung disease ≥ stage III (see appendix 1)
. Severe renal dysfunction defined as estimated GFR of \<30 ml/min.
. Severe cardiac dysfunction defined as shortening fraction \< 25%.
. Severe neurologic impairment other than hemiplegia alone, defined as full scale IQ ≤ 70, quadriplegia or paraplegia, inability to ambulate, inability to communicate without assistive device, or any impairment resulting in decline of Lansky performance score to \<50%.
. Karnofsky or Lansky functional performance score \< 50%