Expired Study
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Minneapolis, Minnesota 55455


Purpose:

The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched sibling) stem cell transplants.


Study summary:

All subjects will be given the same treatment regimen of total body irradiation (TBI), Fludarabine, Cyclophosphamide, and anti-thymocyte globulin (ATG), followed by an alternate donor stem cell transplant. Since this treatment regimen has been given before, without thymic shielding, we will compare the outcomes of these patients with the historical data from subjects who did not receive thymic shielding.


Criteria:

Inclusion Criteria: - Patients must be less than (<) 18 years of age with a diagnosis of Fanconi anemia. - Patients must have an HLA-A, B, DRB1 identical unrelated donor or less than or equal to (≤)1 antigen mismatched related (non-HLA-matched sibling) or <1 antigen mismatched unrelated UCB 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 aplastic anemia (AA), myelodysplastic syndrome without excess blasts, or high risk genotype as defined below. - Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions - Platelet count <20 x 10^9/L - ANC <5 x 10^8/L - Hgb <8 g/dL - Myelodysplastic syndrome with multilineage dysplasia with or without chromosomal anomalies - High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations, or BRCA1 or 2 mutations) - Adequate major organ function including - Cardiac: ejection fraction greater than (>)45% - Hepatic: bilirubin, AST/ALT, ALP <2 x normal - Karnofsky performance status >70% or Lansky performance status >50% - Women of child-bearing age must be using adequate birth control and have a negative pregnancy test Exclusion Criteria: - Available HLA-genotypically identical related donor - History of gram negative sepsis or systemic fungal infection (proven or suspected based on radiographic studies) - Refractory anemia with excess blasts, or leukemia - Active central nervous system (CNS) leukemia at time of hematopoietic cell transplant (HCT) - History of squamous cell carcinoma of the head/neck/cervix within 2 years of HCT - Pregnant or lactating female - Prior radiation therapy preventing use of total body irradiation (TBI) 450 centigray (cGy)


Study is Available At:


Original ID:

0312M54991


NCT ID:

NCT00167206


Secondary ID:

MT2003-18


Study Acronym:


Brief Title:

Stem Cell Transplantation for Fanconi Anemia


Official Title:

A Study of Thymic Shielding in Recipients of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Stem Cell Transplantation in Patients With Fanconi Anemia


Overall Status:

Terminated


Study Phase:

Phase 1/Phase 2


Genders:

N/A


Minimum Age:

N/A


Maximum Age:

18 Years


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

Masonic Cancer Center, University of Minnesota


Oversight Authority:

United States: Institutional Review Board


Reasons Why Stopped:

Treatment with thymic shielding found safe,


Study Type:

Interventional


Study Design:


Number of Arms:

1


Number of Groups:

0


Total Enrollment:

16


Enrollment Type:

Actual


Overall Contact Information

Official Name:Margaret MacMillan, MD
Principal Investigator
Masonic Cancer Center, University of Minnesota

Study Dates

Start Date:March 2004
Completion Date:December 2008
Completion Type:Actual
Primary Completion Date:December 2008
Primary Completion Type:Actual
Verification Date:August 2019
Last Changed Date:August 21, 2019
First Received Date:September 9, 2005
First Results Date:July 7, 2009

Study Outcomes

Outcome Type:Primary Outcome
Measure:Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment
Time Frame:Day 42 after hematopoietic cell transplant
Safety Issues:False
Description:Calculated from Day 1 of hematopoietic cell transplant to Day 42 post-transplant. Hematopoietic recovery and engraftment is defined as the first of three consecutive days the patient's absolute neutrophil count is greater than or equal to 0.5X10^9/Liter.
Outcome Type:Secondary Outcome
Measure:Number of Patients Who Exhibited Secondary Graft Failure
Time Frame:Day 100 after hematopoietic cell transplant
Safety Issues:False
Description:Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. A complication after Bone Marrow Transplant in which the transplanted stem cells do not grow in the recipient's bone marrow and thus do not produce new blood cells.
Outcome Type:Secondary Outcome
Measure:Number of Patients With Acute Graft Versus-Host Disease (aGVHD)
Time Frame:Day 100 after hematopoietic cell transplant
Safety Issues:False
Description:Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" an
Outcome Type:Secondary Outcome
Measure:Number of Patients With Chronic Graft Versus-Host Disease (GVHD)
Time Frame:1 year after hematopoietic cell transplant
Safety Issues:False
Description:Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and
Outcome Type:Secondary Outcome
Measure:Number of Patients Who Exhibited Regimen-related Toxicity (RRT)
Time Frame:1 year after hematopoietic cell transplant
Safety Issues:False
Description:Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. Regimen-related toxicity involves harmful effects in an organism through exposure to the treatment given.
Outcome Type:Secondary Outcome
Measure:Immune Reconstitution - Mean Value (1 Year)
Time Frame:1 year post-transplant.
Safety Issues:False
Description:Calculated mean value of patient CD4 values collected at intervals from Day 30 through 1 year post-transplant.
Outcome Type:Secondary Outcome
Measure:Immune Reconstitution - Mean Value (2 Years)
Time Frame:at 2 years after transplant
Safety Issues:False
Description:Calculated mean value of patient CD4 values collected at intervals from Day 30 through 2 years post-transplant.
Outcome Type:Secondary Outcome
Measure:Number of Patients Alive at 1 Year
Time Frame:1 year after transplant
Safety Issues:False
Description:Calculated from Day 1 of hematopoietic cell transplant to 1 year post-transplant.
Outcome Type:Secondary Outcome
Measure:Number of Patients Alive at 2 Years
Time Frame:2 years after transplant
Safety Issues:False
Description:Calculated from Day 1 of hematopoietic cell transplant to 2 years post-transplant.

Study Interventions

Intervention Type:Procedure
Name:Hematopoietic Stem Cell Transplant
Description:Bone marrow failure may be treated by giving patients stem cells that come from someone else. This is called a stem-cell transplant. As part of the transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts
Arm Name:HSCT Patients
Other Name:Bone Marrow Transplant
Intervention Type:Procedure
Name:Thymic Shielding During Radiation
Description:protecting the thymus during total body radiation (450 cGy administered)
Arm Name:HSCT Patients
Other Name:TBI
Intervention Type:Procedure
Name:Total Body Irradiation
Description:Six days before the stem cells are given (day -6), subjects will receive total body irradiation with thymic shielding. Thymic shielding is done by placing a piece of lead on the chest during the irradiation treatment so that the irradiation beams do not go to the thymus.
Arm Name:HSCT Patients
Other Name:Radiation Therapy, Therapuetic Radiation
Intervention Type:Drug
Name:Cyclophosphamide, Fludarabine
Description:Day -5 through Day -2, subjects will receive a chemotherapy regimen of Fludarabine, Cyclophosphamide via central line
Arm Name:HSCT Patients
Other Name:Cytoxan, Fludara

Study Arms

Study Arm Type:Experimental
Arm Name:HSCT Patients
Description:Patients who received total body irradiation (450 cGy [centigray]) with thymic shielding prior to chemotherapy regimen and Hematopoietic Stem Cell Transplant (HSCT)

Study Agencies

Agency Class:Other
Agency Type:Lead Sponsor
Agency Name:Masonic Cancer Center, University of Minnesota

Sample and Retention Information

There are no available Sample and Retention Information

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: January 21, 2020

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