Fludarabine-Based Conditioning for Matched Related Donor Bone Marrow Transplantation in Patients With Bone Marrow Failure Syndromes

What's the purpose of this trial?

This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).

This trial is currently open and accepting patients.

What will happen during the trial?

You may be able to join this trial if you:

The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.

Patients 0-22 years with acquired aplastic anemia or a diagnosed inherited bone marrow failure syndrome, and a fully Human leukocyte antigen (HLA)-matched (10/10) related donor.

Inclusion Criteria:


1. Ages 0-22 years at time of enrollment
2. Diseases:

* 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.

Organ Function:

* 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 Criteria:

* 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 Criteria:

* 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

Additional Trial Information

Phase 0/1

Enrollment: 75 patients (estimated)

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Trial Locations

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Children's Hospital of Philadelphia

Philadelphia, PA

Open and Accepting
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