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Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias Clinical Trials Information presented on Clinical Trials Search is not intended to be a substitute for qualified health advice, trips or treatment by using a genuine doctor. We aren't doctors. Always consult your mD on Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias conditions. Clinical Trials Search.org is a site committed to listing clinical research studies in human subjects. Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias Clinical research trials and Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias health trials take place in a lot of of cities across the US. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally measure the potency of new drugs. The aim of the studies / projects is to answer specific human medical questions. Clinical trials are a popular manner for physicians, government agencies, and private sector corporations to discover remedies for all kinds of circumstances, like Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias. Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias Clinical Trials and other clinical trials allow for volunteers to have health treatment alternatives before they are available to the general public. Many times the test subjects obtain treatment for without cost, and occasionally they are paid for their time. Sometimes there is a cost for a Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias clinical trial. Subjects oftentimes recieve the most effective healthcare possible for their Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias condition. Hazards are a reality, however, and could include additional or frequent doctor visits, healthcare dangers (perhaps life-threatening), and/or the treatment being ineffective. Trials are federally governed with exacting guidelines to protect clinical trials subjects.
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Home > "I" Clinical Trials Conditions > Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias  Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias
Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias
For Condition: Congenital Hemolytic Anemia,Diamond-Blackfan Anemia
Status: Recruiting
Sponsor(s): National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) ,
Synopsis: People with severe congenital anemias, such as sickle cell anemia, thalassemia, and Diamond Blackfan anemia, have been cured with bone marrow transplantation (BMT). The procedure, however, is limited to children younger than the age of 16 because the risks are lower for children than for adults. The purpose of this study is to explore the use of a BMT regimen that, instead of chemotherapy, uses a low dose of radiation, combined with two immunosuppressive drugs. This type BMT procedure is described as nonmyeloablative, meaning that it does not destroy the patient's bone marrow. It is hoped that this type of BMT will be safe for patients normally excluded from the procedure because of their age and other reasons. To participate in this study, patients must be between the ages of 18 and 65 and have a sibling who is a well-matched stem-cell donor. Beyond the standard BMT protocol, study participants will undergo additional procedures. The donor will receive G-CSF by injection for five days; then his or her stem cells will be collected and frozen one month prior to BMT. Approximately one month later, the patient will be given two immune-suppressing drugs, Campath 1-H and Sirolimus, as well as a single low dose of total body irradiation and then the cells from the donor will be infused. Prior to their participation in this study, patients will undergo the following evaluations: a physical exam, blood work, breathing tests, heart-function tests, chest and sinus x-rays, and bone-marrow sampling.
Details: Nonmyeloablative allogeneic peripheral blood stem cell transplants are currently being investigated in phase I/II trials assessing engraftment, efficacy, and toxicity at a number of transplant centers. Preliminary data, including our own experience with over 100 patients, have shown a high rate of complete donor engraftment with a relatively low toxicity profile. The decreased risk of transplant-related complications associated with nonmyeloablative transplants expands eligibility to patients with nonmalignant hematological disorders curable by allogeneic transplantation; however, significant toxicity with current regimens persists including severe GVHD leading to significant morbidity and mortality. Moreover, mixed chimerism has been shown to be sufficient to induce clinical remissions in children with nonmalignant hematologic disorders undergoing conventional allogeneic transplantation. Therefore, newer regimens need to be developed that are more applicable to patients with non-malignant disorders in whom no graft vs. leukemia effect is needed, and where mixed chimerism is sufficient for disease amelioration. In this protocol, we propose transplantation in patients with severe congenital anemias including sickle cell anemia (SCA), thalassemia, or Diamond Blackfan anemia (DBA), considered at high risk for complications from or ineligible for standard BMT, with allogeneic peripheral blood stem cells from an HLA identical sibling using a novel immunosuppressive regimen without myeloablation in an attempt to further decrease the transplant related morbidity/mortality. The low intensity nonmyeloablative conditioning regimen will consist of low dose radiation, Alemtuzumab (Campath® (Registered Trademark)) and Sirolimus (Rapamune® (Registered Trademark)) as a strategy to provide adequate immunosuppression to allow sufficient engraftment for clinical remission with a lower risk of graft versus host disease development. T-cell replete, donor-derived, granulocyte colony stimulating factor (G-CSF) mobilized peripheral blood stem cells (PBSC) will be used to establish hematopoietic and lymphoid reconstitution, with additional T-cell infusions in those patients with unstable engraftment. The primary endpoint of this study is donor engraftment. Other end points include degree of donor-host chimerism necessary for long-term graft survival and disease amelioration, incidence of acute and chronic graft versus host disease (GVHD), incidence of graft rejection, transplant related morbidity, as well as disease-free and overall survival.
Eligibility:
Study Type: Interventional, Treatment, Safety/Efficacy
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: INCLUSION CRITERIA: RECIPIENTS: Patients with Hb SS, SC, or Sb-thalo at high risk for disease related mortality or morbidity as defined by having one or more of the following: a previous neurologic event (either symptomatic or found by imaging alone), more than 2 painful crises per year for the last 2 years each requiring hospitalization, a previous acute chest syndrome, evidence of renal damage but with a creatinine clearance of greater than 50 % of normal, red cell alloimmunization, stage I or II sickle chest, (Stage I patients have normal oxygen saturation but 80% of predicted normal pulmonary function tests. Stage II patients also have normal oxygen saturation but 60% of predicted normal pulmonary function tests.) osteonecrosis of multiple joints pulmonary hypertension as measured by a TRV jet of greater than 2.5m/s and having failed hydroxyurea, as defined by a failure to achieve a hematologic response and/or clinical response where clinical/hematologic response is defined as a significant decrease in the number of crises experienced after a 6 month trial or a 2-3 fold increase in the hemoglobin F level unless has renal insufficiency preventing hydroxyurea use, or indication for transplant is or includes a neurologic event. or Patients with transfusion dependent Diamond Blackfan anemia or Patients with Thalassemia with Class II or III Iron overload Ages 18- 65 6/6 HLA matched family donor available Ability to comprehend and willing to sign an informed consent Negative Serum B-HCG INCLUSION CRITERIA: DONOR 6/6 HLA identical family donor Age greater than 18 Fit to receive G-CSF and give peripheral blood stem cells (normal blood counts, normotensive and no history of stroke) Ability to comprehend and willing to sign an informed consent; assent obtained from minors EXCLUSION CRITERIA: RECIPIENT (any of the following would exclude the subject from participating) Age less than 18 years or greater than 65 years ECOG performance status of 3 or more. Diffusion capacity of carbon monoxide (DLCO) less than 60% predicted. Left ventricular ejection fraction: less than 40%. Serum bilirubin greater than 4 mg/dl Transaminases greater than 5x upper limit of normal Psychiatric disorder or mental deficiency severe enough as to make compliance with the BMT treatment unlikely, and making informed consent impossible. Major anticipated illness or organ failure incompatible with survival from PBSC transplant. Pregnant or lactating EXCLUSION CRITERIA: DONOR: (any of the following would exclude the donor from participating) Age less than 18 Pregnant or lactating Donor unfit to receive G-CSF and undergo apheresis. (Uncontrolled hypertension, history of congestive heart failure or unstable angina, thrombocytopenia) HIV positive Hemoglobin SS, SC, or Sbeta thal0, or beta thalassemia intermedia Abnormal Red Cell Adenosine Deaminase level
Total Enrollment: 25
Location and Contact Information:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) *Recruiting*
Bethesda, Maryland, 20892
United States
Recruiting Patient and Public Liaison Office 1-800-411-1222
Additional Information:
Study ID Numbers: 030170; 03-DK-0170
Study Start Date: May 19, 2003
Record last reviewed: April 16, 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00061568
Other Congenital Hemolytic Anemia Studies:
1. Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias
Related Studies:
Other Congenital Hemolytic Anemia Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias
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