NCT03600909

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Memorial Sloan Kettering Cancer Center · Academic / Other
• Sex: All
• Age: 1 Month
• Healthy volunteers: Not accepted

Summary

The goal of this study is to see if the study therapy can decrease the chemotherapy-related side effects while maximizing the effectiveness of disease control. The physicians will also be studying the effect of removing T-cells from the donor"s stem cells before transplant. T-cells are a type of white blood cell that may help cause a serious side effect of transplant called Graft versus Host Disease (GVHD). The way it removes the T-cells from the donor stem cells is actually by selecting only the stem cells (called CD34 cells) by using a device called CliniMACS. This process is called CD34 selection. The CliniMACS® device is currently under the supervision of the FDA .

Conditions

• Fanconi Anemia
• Myelodysplastic Syndrome (MDS)
• Acute Myelogenous Leukemia (AML)

Interventions

Type	Name	Description
DRUG	Busulfan	A standard dose of busulfan, associated with excellent outcomes in our previous trial will be used for young patients with marrow aplasia (arm A). A higher dose of busulfan will be used in younger patients with MDS and AML (arm B) to maximize disease control. A lower dose of busulfan will be used in older patients (arm C) to minimize toxicity.
DRUG	Fludarabine	Arms A, B and C - Fludarabine will be given IV over 30 minutes daily for 4 days. The dose will be adjusted according to renal function according to Institutional guidelines.
DRUG	Cyclophosphamide	Arms A, B and C - Cytoxan will be given as a 1-2 hour infusion for 4 days. The dose will be adjusted according to patients ideal body weight for obese patients.
DRUG	Anti-Thymocyte Globulin (Rabbit)	Arms A, B and C - 4 doses will be given prior to transplant to promote engraftment
DEVICE	The CliniMACS device	The source of stem cells for all patients will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 4-6 days. T-cell depletion will be uniformly performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device)
DRUG	G-CSF	All patients will also receive G-CSF post-transplant to foster engraftment.

Timeline

• Start date: 2018-05-15
• Primary completion: 2021-04-09
• Completion: 2021-04-09
• First posted: 2018-07-26
• Last updated: 2022-04-12
• Results posted: 2022-04-12

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study
• FDA-regulated device study