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Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia Clinical Trials Data presented on Clinical Trials Search is not meant to be a substitute for qualified medical advice, visits or professional assistance with a genuine dr.. We are not doctors. Always consult your mD about Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia Clinical research trials and Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia medical trials take place in many of places throughout the U.S.A.. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials usually evaluate the effectiveness of new does drugs. The purpose of the studies / projects is to solve specific human healthcare questions. Clinical trials are a popular way for mDs, government agencies, and private sector companies to find cures for all varieties of conditions, like Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia. Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia Clinical Trials and other clinical trials allow for volunteers to have health treatment options before they are available to the masses. Many times the human subjects acquire professional assistance for free of charge, and sometimes they are compensated for their time. Occasionally there is a cost for a Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia clinical trial. Test subjects typically obtain the finest healthcare available for their Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia condition. Dangers are a reality, nevertheless, and might include additional or frequent doctor trips, medical dangers (possibly life-jeopardising), and/or the treatment being ineffectual. Trials are federally regulated with strict guidelines to protect clinical trials patients.
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Home > "S" Clinical Trials Conditions > Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia  Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia
Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia
For Condition: Anemia, Sickle Cell,Hemoglobin SC Disease,Thalassemia Major,Hemoglobinopathies,Thalassemia
Status: Recruiting
Sponsor(s): National Institute of Allergy and Infectious Diseases (NIAID) ,
Synopsis: The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation.
Details: Hemoglobinopathies, such as sickle cell disease and thalassemia major, are genetic diseases associated with significant morbidity and premature death. Allogeneic bone marrow transplantation (BMT) is the only potential cure for severe hemoglobinopathies. Typical regimens have used high doses of chemotherapy or chemo-radiotherapy to ablate recipient hematopoiesis and to prevent graft rejection. The widespread use of this treatment has been limited by toxicity, risk of end-organ damage, and donor availability. This study will use a non-myeloablative regimen of fludarabine and Busulfex to attempt to generate consistent engraftment with donor hematopoietic stem cells in patients with severe hemoglobinopathy. G-CSF mobilization of the donor’s peripheral blood white blood cells will precede donor apheresis. A non-myeloablative conditioning regimen of fludarabine and Busulfex will be administered to patients prior to allogeneic peripheral blood stem cell infusions. FK506 and prednisone will be administered for graft versus host disease prophylaxis. Patients will be evaluated for engraftment, donor:host hematopoietic chimerism, toxicity, and hemoglobinopathy.
Eligibility:
Study Type: Interventional, Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Minimum Age/Maximum Age: 18 Years/
Genders: Both
Protocol Entry Criteria: Inclusion criteria: --All patients must: - Have related donors who are identical at 6 HLA loci (A, B and DR) by molecular typing - Have a performance status from 0-2 - Give written informed consent --Patients with sickle cell disease should have 1 or more of the following: - Acute chest syndrome requiring recurrent hospitalization or exchange transfusion - Nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours - Recurrent vaso-occlusive pain (2 episodes or more per year) or recurrent priapism - Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate 30-50 percent of normal predicted value) - Bilateral proliferative retinopathy and major visual impairment in at least 1 eye - Osteonecrosis of multiple joints --Patients with thalassemia should have 1 or more of the following: - Transfusion dependence, defined as a transfusion requirement of greater than or equal to 6 units of packed red blood cells over the past 12 months - Iron overload, defined as serum ferritin greater than 500 in the absence of infection or biopsy-proven iron overload - Presence of 2 or more alloantibodies against red cell antigens Exclusion criteria: - Pregnancy - Acute hepatitis (transaminases greater than 3 times the normal value) - Cardiac ejection fraction less than 30 percent - Severe renal impairment (glomerular filtration rate less than 30 percent of predicted normal value) - Severe residual functional neurologic impairment (other than hemiplegia alone) - Seropositivity for HIV
Total Enrollment: 20
Location and Contact Information:
Overall Study Official:
CatherineWu, Principal Investigator, Dana-Farber Cancer Institute, Harvard Medical School
Dana-Farber Cancer Institute/Harvard Cancer Center, Brigham and Women's Hospital and Massachusetts General Hospital *Recruiting*
Boston, Massachusetts, 02115
United States
Recruiting Catherine Wu 617-632-5943
Additional Information:
Study ID Numbers: DAIT DF/HCC 01-098; P01 A 129530
Study Start Date:
Record last reviewed: September 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00034528
Other Thalassemia Studies:
1. Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia
2. A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders
3. The Effect of Oral Magnesium Pidolate on How Often Painful Crises Happens in Patients with Hemoglobin SC Disease
Related Studies:
Other Thalassemia Clinical Trials
Other Massachusetts Clinical Trials
Other Boston Clinical Trials
Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia
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