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Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies Clinical Trials Information presented on Clinical Trials Search is not designed to be a substitute for proven healthcare advice, travels to or treatment by using a genuine medical doctor. We are not physicians. Always confer with your doctor on Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies Clinical research trials and Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies healthcare trials take place in many of cities across the United States of America. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally evaluate the effectiveness of new drugs. The function of the studies / undertakings is to answer specific human medical questions. Clinical trials are a popular means for mDs, government agencies, and private sector companies to find treatments for all forms of conditions, including Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies. Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies Clinical Trials and other clinical trials allow for volunteers to access medical treatment alternatives before they are available to the masses. Many times the test subjects undergo treatment for without cost, and occasionally they are compensated for their time. Occasionally there is a cost for a Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies clinical trial. Test subjects oftentimes recieve the best healthcare possible for their Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies condition. Hazards are a reality, nonetheless, and might include additional or frequent doctor trips, healthcare hazards (perhaps life-jeopardizing), and/or the treatment being ineffective. Trials are federally regulated with rigid guidelines to protect clinical trials subjects.
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Home > "B" Clinical Trials Conditions > Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies  Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies
Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies
For Condition: Thalassemia,Hemoglobinopathy,Sickle Cell Anemia
Status: Terminated
Sponsor(s): Baylor College of Medicine , Texas Children's Hospital,The Methodist Hospital
Synopsis: The major goal of this study is to determine the risks and benefits of stem cell transplants in combination with a newer, less toxic conditioning chemotherapy treatment in patients with severe sickle cell disease (SCD) or sickle hemoglobin variants (hemoglobin SC or hemoglobin SB0/+), or homozygous b0/+ thalassemia or severe B0/+ thalassemia variants. Participation in this project will be for one year, with follow up evaluations done every 6 months thereafter for 10 years or until participants are 18 years old.
Details: To do the stem cell transplant, we must first kill most of the cells in the bone marrow that make the sickle hemoglobin or abnormal blood cells of severe beta thalassemia. We will do this by using a single dose of body irradiation and two drugs called Fludarabine and Campath-IH. The treatment schedule is as follows: Day - 6: Total body irradiation Day - 5: Fludarabine and Campath 1H Day - 4: Fludarabine and Campath 1H Day - 3: Fludarabine and Campath 1H Day - 2: Fludarabine and Campath 1H Day - 1: REST Day 0: Stem Cell Transplant (infusion) After the drug treatment, participants will be given healthy stem cells from a related donor that partially matches their HLA (immune) type, most likely from a parent or sibling. This is known as the stem cell transplant. The healthy stem cells will be put into a blood vein in the same way that transfusions are given. The cells then travel to the right places in the body, where they should grow and make new blood cells that do not sickle.
Eligibility:
Study Type: Interventional, Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Minimum Age/Maximum Age: /65 Years
Genders: Both
Protocol Entry Criteria: Inclusion: - Patients with a haploidentical related HLA donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ thalassemia and at least one of the following conditions: 1) previous central nervous system vaso-occlusive episode with or without residual neurologic findings; 2) frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy; 3) recurrent SCD chest syndrome events, which necessitate chronic transfusion therapy; 4) severe anemia, which prevents acceptable quality of life and necessitates chronic transfusion therapy. - Patients with a haploidentical related HLA donor and homozygous b0/+ thalassemia or severe variants of b0/+ thalassemia and require chronic transfusion therapy. - Women of childbearing potential must have a negative pregnancy test. - Between the ages of birth and 65 years. Exclusion: - HLA identical or 5/6 HLA matched sibling donor - Biopsy proven chronic active hepatitis or portal fibrosis. - SCD chronic lung disease > stage 3 Severe renal dysfunction defined as creatinine clearance <40 ml/min/1.73 M2. - Severe cardiac dysfunction defined as shortening fraction <25%. - HIV infection. - Unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate Stem Cell Transplant. - Patient or guardian(s) unable to understand the nature and risks inherent in the stem cell transplant process. - Pregnant or lactating females and those unwilling to use acceptable contraception.
Total Enrollment: 15
Location and Contact Information:
Overall Study Official:
MalcolmBrenner, Principal Investigator, Baylor College of Medicine
The Methodist Hospital
Houston, Texas, 77030
United States
Texas Children's Hospital
Houston, Texas, 77030
United States
Additional Information:
Study ID Numbers: H8750; Smallo
Study Start Date: August 2000
Record last reviewed: December 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00040417
Other Sickle Cell Anemia Studies:
1. Phase I/II Study of Nonmyeloablative Allogeneic Bone Marrow Transplantation in Patients With High Risk Hemoglobinopathy
2. L-glutamine Therapy for Sickle Cell Anemia
3. Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies
4. Deferoxamine for the Treatment of Hemochromatosis
5. Bone Marrow transplant from related donor for patients with high risk hemoglobinopathies
Related Studies:
Other Sickle Cell Anemia Clinical Trials
Other Texas Clinical Trials
Other Houston Clinical Trials
Bone marrow transplant from donor using less toxic conditioning for patient with high risk hemoglobinopathies
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