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New treatment option for Severe Aplastic Anemia
Official title Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
ClinicalTrials.gov ID: NCT02828592
What this study is testing
What is Fludarabine?
Fludarabine is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for severe aplastic anemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 75
You may be able to join if
- Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
- Age \<= 65 years for previously treated and \<= 75 years for previously treated patients
- KPS \>= 70%
- Aplastic Anemia that meets the following criteria: Peripheral Blood (must fulfill 2 of 3):
- \<500 PMN/mm3
You likely can't join if
- poor cardiac function (LVEF \<40%)
- poor pulmonary function (FEV1 \& FVC \<50% predicted)
- poor liver function (bili \>= 2mg/dL)
- poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40mL/min)
- prior allogeneic transplant
See the full eligibility criteria
- Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
- Age \<= 65 years for previously treated and \<= 75 years for previously treated patients
- KPS \>= 70%
- Aplastic Anemia that meets the following criteria: Peripheral Blood (must fulfill 2 of 3):
- \<500 PMN/mm3
- \<20,000 platelets
- absolute reticulocyte count \<40,000/microL Bone Marrow (must be either):
- markedly hypocellular (\<25% of normal cellularity)
- moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above
- poor cardiac function (LVEF \<40%)
- poor pulmonary function (FEV1 \& FVC \<50% predicted)
- poor liver function (bili \>= 2mg/dL)
- poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40mL/min)
- prior allogeneic transplant
The study team makes the final eligibility decision.
Where it's taking place
- Atlanta, Georgia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Atlanta, Georgia, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.