Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia (Mafia)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00590460|
Recruitment Status : Terminated (slow accrual)
First Posted : January 10, 2008
Results First Posted : August 9, 2012
Last Update Posted : May 24, 2018
|Condition or disease||Intervention/treatment||Phase|
|Fanconi Anemia Severe Aplastic Anemia||Biological: CAMPATH-1H Biological: Anti-CD45 Drug: Fludarabine Procedure: Stem cell infusion||Phase 1 Phase 2|
If clinically feasible (no aplasia, no active malignancy), the recipients marrow will be harvested and cryopreserved as a back up for use if non-engraftment/rejection is followed by failure to undergo autologous reconstitution.
For HLA Mismatched donors, harvested peripheral blood stem cells will be enriched for CD34 cells using the Clinimacs CD34 Reagent system.
Fludarabine will be given as 5 daily intravenous infusions. Campath-1H will be given as 3 daily intravenous infusions and will be followed by Anti-CD45 which will be given as four daily intravenous infusions that will be completed two days prior to stem cell infusion. Diphenydramine will be administered intravenously every 4 hours during the period of the course of each infusion.
Day -8 Campath 1H as per CAGT SOP Fludarabine 30 mg/m2 -7 Campath 1H as per CAGT SOP Fludarabine 30 mg/m2 -6 Campath 1H as per CAGT SOP Fludarabine 30 mg/m2 -5 YTH 24/54 400ug/kg over 6 hr Fludarabine 30 mg/m2 -4 YTH 24/54 400ug/kg over 6 hr Fludarabine 30 mg/m2 -3 YTH 24/54 400ug/kg over 6 hr -2 YTH 24/54 400ug/kg over 6 hr -1 -0 Stem Cell Infusion
GVHD prophylaxis will be achieved through positive selection for CD34 resulting in > 3 log T cell depletion. Previous reports have indicated that there is a low frequency of severe (Grade II/IV) GvHD after haploidentical transplants if recipients receive stem cell populations containing <5 x 10e4 CD3 positive T cells. We hope to achieve such levels with our CD34 enrichment protocol. However, pharmacologic prophylaxis will be added if the CD34 selected product contains more than 5 x 10e4 CD3+ve T cells/kg recipient weight. In addition, Campath 1H persists in the recipient circulation through the immediate transplant period and will contribute anti-GVHD activity, in vivo.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Cd45 (Yth-24 and Yth 54) and Cd52 (Campath-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Donor Stem Cell Transplantation of Patients With Fanconi Anemia|
|Study Start Date :||July 2001|
|Actual Primary Completion Date :||September 2009|
|Actual Study Completion Date :||September 2009|
Experimental: Single Arm Study: Stem Cell Transplant
CAMPATH-1H Anti-CD45 Fludarabine Stem Cell Infusion
Given intravenous on days -8, -7, and -6
Other Name: AlemtuzumabBiological: Anti-CD45
Given intravenous on days -5, -4, -3 and -2
dose is 400 micrograms/kg
Procedure: Stem cell infusion
Given intravenous on days -8, -7, -6, -5 and -4
Dose is 30 mg/m2
Stem cells are infused on day 0
- Number of Patients With Donor Engraftment [ Time Frame: 100 Days ]Number of patients with engraftment of at least 65% of donor cells 100 days after transplantation
- Number of Patients With Graft Failure [ Time Frame: 100 days ]Graft failure is defined as engraftment of less than 65% of donor cells 100 days after transplantation.
- Patients With Treated Related Death [ Time Frame: 100 days ]Number of patients with treated related death
- Days to Absolute Neutrophil Count (ANC) of 500/mm3 [ Time Frame: 30 Days ]Number of days to Absolute neutrophil count (ANC) of 500/mm3
- Days to Platelet Count of 20,000/mm3 Without Transfusions [ Time Frame: 30 Days ]Number of days to Platelet count of 20,000 / mm3 without transfusions
- Patients With Grade II - IV Acute Graft Versus Host Disease (GVHD) [ Time Frame: 100 days ]Number of patients with grade II - IV acute Graft versus Host Disease (GVHD)
- Number of Patients Alive at 1 Year Post Transplant [ Time Frame: 1 year ]Number of patients alive at 1 year post allogeneic stem cell transplant
- Patients With Limited Chronic GVHD From Day 100 to 365 [ Time Frame: 365 days ]Number of patients with limited chronic GVHD from day 100 to 365
- Patients With Extensive Chronic GVHD From Day 100 to 365 [ Time Frame: 365 days ]Number of patients with extensive chronic GVHD from day 100 to 365.
- Patients With Grade III - IV Acute GVHD [ Time Frame: 100 days ]Number of patients with Grade III-IV acute GVHD
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00590460
|United States, Texas|
|Houston, Texas, United States, 77030|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Malcolm Brenner, M.B., Ph.D.,||Baylor College of Medicine|