Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Thre… (NCT00006056) | Clinical Trial Compass
UnknownNot Applicable
Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders
United States40 participantsStarted 2000-03
Plain-language summary
OBJECTIVES: I. Determine the efficacy of unrelated donor hematopoietic stem cell transplantation in the treatment of patients with life threatening hemophagocytic disorders.
II. Determine the rate of disease free survival, incidence of graft failure, and incidence of graft versus host disease in these patients after undergoing this treatment regimen.
Who can participate
Age range
0 Years – 55 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.
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Patients diagnosed with any of the following active but stable, or nonactive/quiescent, hemophagocytic disorders:
* Hemophagocytic lymphohistiocytosis (HLH)
* Fever greater than 38.5 degrees Celsius
* Splenomegaly (greater than 3 cm below costal margin)
* Hemophagocytosis in bone marrow or spleen or lymph nodes
* Disease may be confirmed by positive family history
* No evidence of malignancy
* Hypertriglyceridemia and/or hypofibrinogenemia
* Fasting triglycerides at least 2.0 mmol/L or at least 3 standard deviations above normal for age
* Fibrinogen no greater than 1.5 g/L or no greater than 3 standard deviations above normal
* Cytopenia (affecting at least 2 of 3 lineages in the peripheral blood)
* Hemoglobin less than 9.0 g/L
* Platelet count less than 100,000/mm3
X-linked lymphoproliferative disorder (XLP)
Two or more maternally related males manifesting at least one of the following XLP phenotypes:
* Fulminant infectious mononucleosis
* Dysgammaglobulinemia
* Malignant lymphoma/lymphoproliferative disorder
* Aplastic anemia
* Lymphoid granulomatosis/vasculitis OR
* A maternally related male in an established XLP kindred who has strong genetic (RFLP) linkage to the XLP locus
Chediak-Higashi syndrome
Partial oculocutaneous albinism (hair, skin, eyes)
Frequent bacterial infections
Large peroxidase positive granules in leukocytes of peripheral blood or bone marrow
Positive family history or parental consanguinity…
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.