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New treatment option for Severe Aplastic Anemia (SAA)
Official title Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
ClinicalTrials.gov ID: NCT03520647
What this study is testing
What is Cyclophosphamide?
Cyclophosphamide is an investigational medicine, given as an once-weekly injectable medicine, being studied as a potential treatment for severe aplastic anemia (saa).
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
- Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 5 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 75
You may be able to join if
- - RECIPIENT:
- Diagnosed with severe aplastic anemia with bone marrow cellularity \<30% (excluding lymphocytes) associated with RBC or platelet transfusion...
- PNH that is either refractory to treatment with eculizumab/ravulizumab or occurs in patients who don t have access to treatment with eculizumab...
- people with severe aplastic anemia, hypoplastic MDS or PNH with associated bone marrow failure syndromes who have intolerance of or failure to...
- Availability of at least one HLA- haploidentical related donor (i.e. \>= 5/10 HLA match: HLA-A, B, C, DR, and DQ loci) to serve as a stem cell donor...
You likely can't join if
- RECIPIENT (ANY OF THE FOLLOWING):
- Availability of an HLA identical (12/12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft...
- The patient is deemed to be a candidate for a 12/12 HLA matched unrelated stem cell transplant (availability of a donor and resources required for...
- ECOG performance status of 2 or more.
- Major anticipated illness or organ failure incompatible with survival from transplant.
- Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from...
See the full eligibility criteria
- - RECIPIENT:
- Diagnosed with severe aplastic anemia with bone marrow cellularity \<30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and/or neutropenia (absolute neutrophil count less than or equal to...
- PNH that is either refractory to treatment with eculizumab/ravulizumab or occurs in patients who don t have access to treatment with eculizumab associated with either a) life- threatening thrombosis and/or b) cytopenia...
- people with severe aplastic anemia, hypoplastic MDS or PNH with associated bone marrow failure syndromes who have intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have...
- Availability of at least one HLA- haploidentical related donor (i.e. \>= 5/10 HLA match: HLA-A, B, C, DR, and DQ loci) to serve as a stem cell donor for the allogeneic transplant.
- Availability of a backup stem cell source in the event of graft rejection:
- at least one additional haploidentical related alternative donor (i.e. HLA- haploidentical related donor (i.e. \>= 5/10 HLA match: HLA-A, B, C, DR, and DQ loci) or \>= 9/10 HLA matched unrelated donor who is available...
- umbilical cord blood unit/s that can be used for a salvage cord blood transplant in the event that the haplo-transplant has been rejected
- The patient does not have any HLA antibodies detectable against any of the mismatched HLA alleles expressed by the haplo-donor.
- Ages 4-60 years inclusive.
- Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to people aged 4-17 years with formal consent being obtained from parents or legal guardian.
- RECIPIENT (ANY OF THE FOLLOWING):
- Availability of an HLA identical (12/12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral...
- The patient is deemed to be a candidate for a 12/12 HLA matched unrelated stem cell transplant (availability of a donor and resources required for such a transplant).
- ECOG performance status of 2 or more.
- Major anticipated illness or organ failure incompatible with survival from transplant.
- Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.
- HIV positive.
- Diagnosis of Fanconi s anemia (by chromosome breakage study).
- Diffusion capacity of carbon monoxide (DLCO) \<40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and...
- Left ventricular ejection fraction \<40% (evaluated by ECHO)
- Transaminases \> 5x upper limit of normal.
- Direct bilirubin \>3 mg/dl.
- Creatinine clearance \< 50 cc/min/BSAm\^2 by 24-hour urine collection adjusted by body surface area.
- Serum creatinine \> 2.5 mg/dl
- Presence of an active infection not adequately responding to appropriate therapy.
- History of a malignant disease liable to relapse or progress within 5 years. INCLUSION CRITERIA - DONOR: Donor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing...
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, 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 study runs about 5 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, 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.