TBI Dose de-Escalation for Fanconi Anemia - Tabular View

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TBI Dose de-Escalation for Fanconi Anemia

This study is currently recruiting participants.

Study NCT00352976. Last updated on June 16, 2008. Information provided by University of Minnesota

This Tabular View shows the required WHO registration data elements as marked by ^†

Descriptive Information Fields

Brief Title ^†

TBI Dose de-Escalation for Fanconi Anemia

Official Title ^†

Total Body Irradiation Dose De-Escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell Transplantation

Brief Summary

This is a single arm, TBI trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.

Detailed Description

Study Treatment: 1. If the subject is to receive total body irradiation with thymic shielding, it will be given six days before the stem cells are given (day -6). 2. Day -5 through Day -2, subjects will receive a chemotherapy regimen of Fludarabine, Cyclophosphamide, ATG, and Methylprednisone (a steroid used to help make sure the transplant "takes") via central line (i.e. Hickman or Broviac). On days -5 to -1, subjects will receive ATG and Methylprednisone. The methylprednisone will continue until about three weeks after transplant.3. Starting Day -3, subjects will be given cyclosporin A (CSA) therapy to help prevent graft-versus-host-disease. We will continue to give CSA until about six months after the transplant. 4. If the subject is receiving bone marrow or "peripheral" stem cells (cells collected from the donor's arm via a cell separator), on the day of transplantation, the stem cells taken from the donor will be put into a machine which will separate the lymphocytes (the cells that cause graft-versus-host disease [GVHD]) from the stem cells. If the subject is receiving an umbilical cord blood, the lymphocytes will not be removed because the risk of GVHD is not as high. Otherwise all patients will receive the same treatment. The stem cells are given as an infusion into the subject's existing catheter over 1-2 hours on day 0.5. On the day after transplant (day +1) subjects will be given G-CSF to stimulate the growth of the transplanted cells. 6. While receiving treatment and until the subject's blood counts recover he/she will have daily blood tests, and several bone marrow biopsies and aspirates. After recovery, subjects will be seen once a month for a health assessment and blood tests until at least 3 months after the cells have been infused. Additional blood tests or assessments may be done as medically indicated.

Study Phase

Phase II, Phase III

Study Type ^†

Interventional

Study Design ^†

Treatment, Non-Randomized, Open Label, Uncontrolled, Crossover Assignment, Safety/Efficacy Study

Primary Outcome Measure ^†

Incidence of neutrophil recovery (absolute neutrophil count ≥500/µL for three consecutive days) . [ Time Frame: by day 42 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measure ^†

Incidence of grade ≥3 regimen related toxicity . [ Time Frame: at day 100 ] [ Designated as safety issue: Yes ]
Incidence of secondary graft failure at 100 days. [ Time Frame: 100 days ] [ Designated as safety issue: No ]
Incidence of acute graft-versus-host disease (GVHD) [ Time Frame: at 100 days. ] [ Designated as safety issue: No ]
Incidence of chronic GVHD . [ Time Frame: at one year ] [ Designated as safety issue: No ]
Probability of survival . [ Time Frame: at one year ] [ Designated as safety issue: No ]
Incidence of infections . [ Time Frame: at 100 days, 6 months and one year ] [ Designated as safety issue: No ]
Immune reconstitution . [ Time Frame: at 100 days, 6 month and one year ] [ Designated as safety issue: No ]

Condition ^†

Fanconi Anemia

Intervention ^†

Drug: Cyclophosphamide
Drug: Fludarabine
Drug: ATG
Procedure: Total Body Irradiation
Procedure: Bone Marrow Transplantation

MEDLINE PMIDs

Links

Recruitment Information Fields

Recruitment Status ^†

Recruiting

Enrollment ^†

Start Date ^†

May 2006

Completion Date

May 2016

Eligibility Criteria ^†

Inclusion Criteria:

Patients must have a ≤1 antigen mismatched HLA-A, B, DRB1 unrelated donor or ≤1 antigen mismatched related (non-HLA-matched sibling) or ≤2 antigen mismatched unrelated UCB donor.
Patients with FA must have aplastic anemia (AA), myelodysplastic syndrome without excess blasts, or high risk genotype.
Adequate major organ function.
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.
Refractory anemia with excess blasts, or leukemia.
Active CNS leukemia at time of HSCT.
History of squamous cell carcinoma of the head/neck/cervix within 2 years of HSCT.
Pregnant or lactating female.
Prior radiation therapy that prevents further TBI.
Patients with advanced MDS (.i.e. RAEB or RAEBt or acute leukemia) will be excluded from this study

Gender

Both

Ages

Accepts Healthy Volunteers

Contacts ^††

Contact: Margaret L MacMillan, M.D.

612-626-2778

macmi002@umn.edu

Location Countries ^†

United States

Administrative Information Fields

NCT ID ^†

NCT00352976

Organization ID

0605M85788

Secondary IDs ^††

Study Sponsor ^†

University of Minnesota

Collaborators ^††

Investigators ^†

Principal Investigator:

Margaret L MacMillan, M.D.

University of Minnesota Medical Center

Information Provided By

University of Minnesota

Verification Date

June 2008

First Received Date ^†

July 14, 2006

Last Updated Date

June 16, 2008

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.