NCT02928991
Fludarabine Based RIC for Bone Marrow Failure Syndromes
Active, Not Recruiting
EARLY_PHASE1Up to 22InterventionalTreatmentChildren's Hospital of PhiladelphiaInvestigator-initiated
~25 participants
Updated 2026-05-28 on ClinicalTrials.gov
What's tested:MRD-BMT with Fludarabine-based RIC for Acquired AAMRD-BMT with Fludarabine-based RIC for iBMF with trilineage aplasiaMRD-BMT with Fludarabine-based RIC for iBMF without trilineage aplasia
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Rate of graft failure
Measured over Up to 1 year post transplant
+2 more outcomes measured
Conditions
Where it's being run
1 sites across 1 statesPennsylvania1
Study leadership
- Timothy Olson, MD, PhD · PRINCIPAL_INVESTIGATOR · Children's Hospital of Philadelphia
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Patients with severe or very severe acquired AA, defined by:
Bone marrow biopsy demonstrating cellularity of \<25% (at least 2 weeks from last dose of G-CSF), in addition to 2 of the following: absolute neutrophil count (ANC) \<500/µL, platelets \< 20,000/µL and absolute reticulocytes \<40,000/µL
Negative evaluation for inherited bone marrow failure conditions and negative evaluation for dysplasia or cytogenetic abnormalities associated with myelodysplastic syndromes
Patients with concurrent paroxysmal nocturnal hemoglobinuria (PNH) clones are eligible, as long as they meet criteria for severe or very severe aplastic anemia as defined above
Patients with clinically diagnosed and/or genetically proven iBMF syndromes, resulting in chronic red blood cell or platelet-transfusion dependence and/or an absolute neutrophil count \<500/µL. These disorders include, but are not limited to:
Fanconi Anemia
Dyskeratosis Congenita
Severe Congenital Neutropenia
Diamond-Blackfan Anemia
Congenital Dyserythropoietic/Sideroblastic Anemias
Congenital Amegakaryocytic Thrombocytopenia
Shwachman-Diamond Syndrome 3. Lansky or Karnofsky performance \>60 4. HLA matched related donor available. 5. No active untreated infection 6. Females of childbearing potential must have negative pregnancy test.
Serum creatinine \<1.5xupper limit of normal for age Hepatic: Transaminases \<5x normal
Cardiac shortening fraction \>27%
Bilirubin \<2.5x normal (unless elevation due to Gilberts disease).
Donor selection will comply with U.S. Food and Drug Administration's Code of Federal Regulations
Fully HLA-matched related donor.
Donor must be at least 6 months of age
Donor suitable for bone marrow collection and meets eligibility for donation, including fulfilling infectious disease criteria as per SOP, including HIV, Hepatitis B, Hepatitis C Polymerase chain reaction (PCR) negative.
If subject has confirmed iBMF syndrome, donor must be evaluated for this disorder and testing must be negative
Children's Hospital of Philadelphia (CHOP) bone marrow transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases.
Donor evaluation and collection procedure as per CHOP Standard Operating Procedures (SOP)
Exclusion
Uncontrolled bacterial, viral or fungal infections
HLA matched related donor unable to donate bone marrow.
No eligible fully HLA-matched related donor
Pregnant females
Patients with a clinical diagnosis of Myelodysplastic syndrome (MDS) defined by combination of bone marrow dysplasia and classic cytogenetic lesion (Monosomy 7, Trisomy 8 eg.), with or without excess blasts.
Patients with PNH without underlying bone marrow aplasia
What this trial measures
- Rate of graft failureUp to 1 year post transplant
Combined rate of primary and secondary graft failure. Primary graft failure is defined as no evidence of neutrophil engraftment by day +28 after stem cell infusion. Secondary graft failure is defined as an ANC\<100 for \>7-10 days after initial engraftment occurs and is confirmed by hypocellular bone marrow biopsy and donor engraftment \<20%.
- Time to neutrophil engraftmentUp to 1 year post transplant
The time from the day of transplant until neutrophil engraftment, which is defined as the first day of ANC \>500/ul for the first of 3 consecutive days.
- Transplant-related mortalityUp to 100 days post transplant