TBI Dose De-escalation for Fanconi Anemia

Status Completed

Clinical Trial Summary

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

Clinical Trial Description

Study Treatment: Patients will receive voriconazole (antifungal therapy) by mouth beginning 1 month prior to conditioning therapy, if possible. 1) The subject is to receive total body irradiation (300 cGy) 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 and Cyclophosphamide via central line (i.e. Hickman or Broviac). Starting Day -3, patients will receive sirolimus therapy with a taper commencing on day +180 and also mycophenolate mofetil (MMF) through day +30 or for 7 days after engraftment, whichever day is later, if no acute graft-versus-host disease (GVHD). 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 Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00352976
Study type	Interventional
Source	Masonic Cancer Center, University of Minnesota
Contact
Status	Completed
Phase	Phase 2/Phase 3
Start date	May 18, 2006
Completion date	October 9, 2020

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT02931071` - Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1	Phase 2
Terminated	`NCT01319851` - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation	N/A
Recruiting	`NCT00084695` - Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases	Phase 2
Completed	`NCT00000603` - Cord Blood Stem Cell Transplantation Study (COBLT)	Phase 2
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Recruiting	`NCT01146210` - Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia	N/A
Completed	`NCT01082133` - Multicenter Transplant Study for Fanconi Anemia	Phase 2
Completed	`NCT00965666` - Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia	Early Phase 1
Terminated	`NCT00290628` - Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer	N/A
Recruiting	`NCT00027274` - Cancer in Inherited Bone Marrow Failure Syndromes
Terminated	`NCT01001598` - Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita	Phase 1/Phase 2
Recruiting	`NCT03206086` - Eltrombopag for People With Fanconi Anemia	Phase 2
Recruiting	`NCT03579875` - T Cell Receptor α/β TCD HCT in Patients With Fanconi Anemia	Phase 2
Recruiting	`NCT05598515` - Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia	N/A
Active, not recruiting	`NCT03476330` - Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia	Phase 2
Terminated	`NCT05910853` - Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
Completed	`NCT03609840` - Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Completed	`NCT00479115` - Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100	Phase 1/Phase 2
Terminated	`NCT03600909` - A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia	Phase 2
Completed	`NCT03398824` - Pilot Study of Metformin for Patients With Fanconi Anemia	Phase 2

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.