Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia

Summary

RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor stem cell transplant helps to remove the patient's cells to allow for the transplant cells to take and grow. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells can make an immune response against the body's normal cells. Giving antithymocyte globulin and removing the T cells from the donor cells before transplant and giving cyclosporine before and after transplant may stop this from happening.

PURPOSE: This phase I/II trial is studying the side effects of cyclophosphamide, fludarabine, and antithymocyte globulin followed by donor stem cell transplant and to see how well it works in treating patients with Fanconi anemia.

Conditions

• Fanconi Anemia

Interventions

BIOLOGICAL

Anti-Thymocyte Globulin

30 mg/kg/day will be administered after MP on days -6, -5, -4, -3 and -2.

DRUG

Cyclophosphamide

5 mg/kg is to be given as a 2 hour infusion, Days -6 through -3.

DRUG

Fludarabine

35 mg/m\^2 intravenously (IV) on days -6 through -2.

PROCEDURE

Hematopoietic Stem Cell Transplantation

Bone marrow or umbilical cord blood infusion on day 0.

DRUG

Methylprednisolone

Methylprednisolone (MP) 2 mg/kg/day intravenously every 24 hours will be given from day -6 until day -2 as a premedication for ATG.

DRUG

Filgrastim

5 mcg/kg per day intravenously (IV) continue until Absolute neutrophil count \> or = 2.5 x 10\^9/L

DRUG

Cyclosporine

Cyclosporine IV over 2 hours or orally every 8-12 hours beginning on day -3 and continuing until day 100, followed by a taper.

DRUG

Mycophenolate Mofetil

Day -3 through day +30 or for 7 days after engraftment, whichever day is later, if no acute GVHD. Engraftment is defined as 1st day of 3 consecutive days of absolute neutrophil count \[ANC\] \> 0.5 x 10\^9/L. MMF will be given at a dose of 15 mg/kg/dose every 8 hours PO (to a maximum dose of 1 gram).

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota

  lead OTHER

Principal Investigators

• Margaret L. MacMillan, MD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Max Age

59 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2000-02-17

Primary Completion

2020-10-10

Completion

2020-10-10

Countries

• United States

Study Locations