DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



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

Information source: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov processed this data on November 27, 2014
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Fanconi Anemia

Intervention: anti-thymocyte globulin (Biological); cyclophosphamide (Drug); fludarabine phosphate (Drug); hematopoietic stem cell transplantation (Procedure); methylprednisolone (Drug); filgrastim (Drug); cyclosporine (Drug); Mycophenolate Mofetil (Drug)

Phase: Phase 1/Phase 2

Status: Recruiting

Sponsored by: Masonic Cancer Center, University of Minnesota

Official(s) and/or principal investigator(s):
Margaret L. MacMillan, MD, Principal Investigator, Affiliation: Masonic Cancer Center, University of Minnesota

Overall contact:
Margaret MacMillan, M.D., Phone: 612-626-2778, Email: macmi002@umn.edu

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.

Clinical Details

Official title: A Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi Anemia

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Graft failure

Secondary outcome:

Incidence of Acute Graft-Versus-Host Disease (GVHD)

Overall survival

Incidence of Chronic Graft-Versus-Host Disease (GVHD)

Transplant Related Deaths

Detailed description: OBJECTIVES: Primary

- To determine the probability of engraftment in patients with Fanconi anemia treated

with cyclophosphamide, fludarabine phosphate, and antithymocyte globulin followed by HLA-genotypically identical sibling donor hematopoietic stem cell transplantation that is T-cell depleted. Secondary

- To evaluate the incidence of acute graft-versus-host disease (GVHD) and chronic GVHD in

patients treated with this regimen.

- To evaluate the incidence of regimen-related toxicity in these patients.

- To evaluate the 1-year survival of patients treated with this regimen.

- To evaluate the incidence of late secondary malignancies (e. g., squamous cell carcinoma

of the head and neck or cervix) in patients treated with this regimen. OUTLINE:

- Preparative cytoreductive therapy: Patients receive cyclophosphamide IV over 2 hours on

days - 6 to -3 and fludarabine phosphate IV over 30 minutes and anti-thymocyte globulin

IV over 4-6 hours on days - 6 to -2.

- T-cell depleted donor hematopoietic stem cell transplantation: Patients undergo T-cell

depleted donor bone marrow or umbilical cord blood stem cell transplantation on day 0. Patients also receive filgrastim (G-CSF) IV beginning on day 1 and continuing until blood counts recover.

- Graft-versus-host disease prophylaxis: Patients receive cyclosporine IV over 2 hours or

orally every 8-12 hours beginning on day - 3 and continuing until day 100, followed by a

taper. Patients will receive Mycophenolate Mofetil (MMF) therapy beginning on 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. After completion of study therapy, patients are followed periodically.

Eligibility

Minimum age: N/A. Maximum age: 59 Years. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Patients must be <60 years of age with a diagnosis of Fanconi Anemia (FA).

- Patients must have an HLA-A, B, DRB1 identical sibling donor. Patients and donors

will be typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing.

- Patients with FA must have moderately severe aplastic anemia (AA), early

myelodysplastic syndrome (MDS) with no excess blasts with or without chromosomal abnormalities.

- In patients <18 years of age, moderately severe aplastic anemia is defined as

having at least one of the following:

- platelet count <40 x 10^9/L

- absolute neutrophil count (ANC) <10 x 10^8/L

- Hgb <9 g/dL

- In patients 18-60 years of age, moderately severe aplastic anemia is defined as

having at least one of the following:

- platelet count <20 x 10^9/L

- absolute neutrophil count ANC <5 x 10^8/L

- Hgb <8 g/dL

- Early myelodysplastic syndrome, with multilineage dysplasia with < 5% blasts,

with or without chromosomal anomalies.

- Adequate major organ function including:

- Cardiac: ejection fraction >45%

- Hepatic: no clinical evidence of hepatic failure (e. g. coagulopathy, ascites)

- Karnofsky performance status >70% or Lansky >50%

- Women of child bearing age must be using adequate birth control and have a negative

pregnancy test. Exclusion Criteria:

- Active bacterial infection within one week of hematopoietic cell transplant (HCT)

- Active fungal infection at time of HCT.

- Late MDS with greater than 5% blasts in bone marrow.

- Acute myelogenous leukemia (AML) or history of AML

- Malignant solid tumor (e. g. squamous cell carcinoma of the head/neck/cervix) within 2

years of HCT.

- Pregnant or lactating female.

Locations and Contacts

Margaret MacMillan, M.D., Phone: 612-626-2778, Email: macmi002@umn.edu

Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota 55455, United States; Recruiting
Patricia Kleinke, RN, Phone: 612-273-0857, Email: Kleink1@fairview.org
Margaret MacMillan, M.D., Principal Investigator
Additional Information

Clinical trial summary from the National Cancer Institute's PDQ® database

Starting date: February 2000
Last updated: May 15, 2014

Page last updated: November 27, 2014

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2014