Stem Cell Transplant for Hematologic Diseases (HIMSUM)
Patients are being asked to participate in this study because they have a cancer in their blood, Fanconi's Anemia, or have been unsuccessfully treated for bone marrow failure such as Aplastic Anemia or Paroxysmal Nocturnal Hemoglobinuria. Any of these conditions could benefit from an allogeneic stem cell transplant using a donor that is related to the patient.
Stem cells are created in the bone marrow. They grow into different types of blood cells that the patient needs, including red blood cells, white blood cells, and platelets. In a transplant, the patient's own stem cells are killed and then replaced by stem cells from the donor.
Usually, patients are given very strong doses of chemotherapy prior to receiving a stem cell transplant. However, because of the patient's condition, they have a high risk of experiencing life-threatening treatment-related side-effects. Recently, some doctors have begun to use chemotherapy that does not cause as many side-effects before patients receive a transplant.
This research study adds CAMPATH 1H to a low-dose chemotherapy regimen, followed by an allogeneic stem cell transplantation. We want to see whether adding CAMPATH 1H to the transplant medications helps in treating the disease. We also want to see whether there are fewer life-threatening side-effects from the treatment. CAMPATH 1H is a drug that is still being studied. CAMPATH 1H stays active in the body for a long time after patients receive it, which means it may work longer at preventing graft-versus-host-disease (GvHD) symptoms.
Biological: Campath 1H
Procedure: Stem Cell Transplant
Radiation: Total Body Irradiation (TBI)
Drug: FK506 (Tacrolimus) or Cyclosporine
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase I/II Study of Allogeneic Stem Cell Transplantation for Patients With Hematologic Diseases Using Haploidentical Family Donors and Sub-Myeloablative Conditioning With Campath 1H|
- Transplant Related Mortality (TRM) [ Time Frame: 100 days ] [ Designated as safety issue: No ]Percentage of patients with transplant related mortality
- Time in Days to ANC Engraftment [ Time Frame: 30 days ] [ Designated as safety issue: No ]Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days.
- Donor Chimerism Engraftment of Greater Than 50% [ Time Frame: 30 days ] [ Designated as safety issue: No ]Number of patients that engrafted who showed a chimerism (donor cells) of greater than 50% in the first 30 days
- Acute Graft Versus Host Disease [ Time Frame: 100 days ] [ Designated as safety issue: Yes ]Number of patients with Acute Graft Versus Host Disease within 100 days post-transplant
- Chronic Graft Versus Host Disease [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]Number of patients with Chronic Graft Versus Host Disease within 1 year post-transplant
- 2-year Relapse-free Survival [ Time Frame: 2 years ] [ Designated as safety issue: No ]Relapse-free survival (RFS) was calculated from the time of transplant to the date of relapse, death, or last follow-up, whichever occurred first. Survival data were analyzed by Kaplan-Meier method.
- 2-year Overall Survival [ Time Frame: 2 years ] [ Designated as safety issue: No ]Overall survival (OS) was calculated from the time of transplant to death from any cause or censored at last follow-up. Survival data were analyzed by Kaplan-Meier method.
- Number of Patients Who Engrafted With the Isolex/CLINIMACs System [ Time Frame: 30 days ] [ Designated as safety issue: No ]Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days.
- Median Time to Engraftment With the Isolex/CLINIMACs System [ Time Frame: 30 days ] [ Designated as safety issue: No ]Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days.
|Study Start Date:||August 2000|
|Study Completion Date:||September 2014|
|Primary Completion Date:||September 2014 (Final data collection date for primary outcome measure)|
Experimental: Stem Cell Transplant
Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF.
Biological: Campath 1H
Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP.Drug: Fludarabine
Day -5 to Day -2: Fludarabine 30 mg/m2.Procedure: Stem Cell Transplant
Day 0: Donor stem cells infused.Radiation: Total Body Irradiation (TBI)
Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute.Drug: FK506 (Tacrolimus) or Cyclosporine
Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.
Before treatment begins, stem cells will be collected from the donor's blood or bone marrow. The stem cells will be collected and frozen before we start to give the patient chemotherapy.
After admission to the hospital, patients will receive total body irradiation (very strong x-rays that kill cells in the bone marrow), Fludarabine and Campath 1H prior to the stem cell transplant (infusion of the donor's stem cells).
Starting 7 days after the transplant, the patient will be given G-CSF by subcutaneous injection, until a blood test shows that numbers of granulocytes (a type of white blood cell) in the blood are more than 1,000/uL. This is to help increase blood cell counts.
After transplantation, the patient will undergo several evaluations at different times. These are standard evaluations and tests performed for any patient who has received a stem cell transplant, as part of routine clinical monitoring.
We will also be looking at the patient's immune function (how the body protects itself to prevent and fight infections and diseases). To do this, blood tests will be performed at regular intervals (every 3 to 6 months) for 2 years.
Depending on how well the donor stem cells work in the body after the transplant, the patient may receive one or more Donor Leukocyte Infusions (DLI). This is when leukocytes (a type of white blood cell) collected from the same donor that provided the stem cells are given to the patient through a central line into a vein.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00058825
|United States, Texas|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|The Methodist Hospital|
|Houston, Texas, United States, 77030|
|Principal Investigator:||George Carrum, MD||Baylor College of Medicine; The Methodist Hospital|