Bone Marrow Transplant With Abatacept for Non-Malignant Diseases (NCT01917708) | Clinical Trial Compass
CompletedPhase 1
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
United States10 participantsStarted 2014-01
Plain-language summary
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.
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.
Inclusion criteria
. Glanzmann thrombasthenia
. Wiskott-Aldrich syndrome or other combined immune deficiency
. Chronic-granulomatous disease
. Severe congenital neutropenia (with resistance to granulocyte-colony stimulating factor (GCSF) or chronic requirement of GCSF doses ≥10 mcg/kg)
. Leukocyte adhesion deficiency
. Shwachman-Diamond syndrome
. Diamond-Blackfan anemia ((transfusion dependent, including steroid failure or inability to wean steroids)
. Thalassemia major
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 testing whether adding abatacept to bone marrow transplant was tolerable — since it was a Phase 1 study focused on safety rather than proving it works better, what do we actually know so far about whether abatacept improved outcomes for my specific condition?
2The trial included a wide range of serious non-malignant diseases like sickle cell disease, thalassemia, and Wiskott-Aldrich syndrome — how relevant are the results from this completed study to my particular diagnosis, and did patients with my condition respond differently than others in the trial?
3Since this trial has already completed, has the data been published, and if so, what did it show about the safety and tolerability of abatacept alongside bone marrow transplant — and does that change how my transplant team might approach my care?
4Bone marrow transplant itself carries significant risks — does adding abatacept to the regimen, as studied in this trial, appear to increase, decrease, or not meaningfully change those risks based on what was observed?
5Given that this trial is now completed and was only Phase 1, are there follow-up or Phase 2 trials now open that build on these findings, or would a more established standard transplant approach without abatacept be a safer or better-studied path for me right now?
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.