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

NCT03600909 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2022-04-12

Study results available
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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

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.

Sponsors & Collaborators

Principal Investigators

  • Farid Boulad, MD · Memorial Sloan Kettering Cancer Center

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-05-15
Primary Completion
2021-04-09
Completion
2021-04-09
FDA Drug
Yes
FDA Device
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03600909 on ClinicalTrials.gov