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Home > "T" Clinical Trials Conditions > Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)  Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)
Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)
For Condition: Anemia (Cooley's),beta-Thalassemia,Blood Disease,Thalassemia,Osteoporosis
Status: Recruiting
Sponsor(s): National Heart, Lung, and Blood Institute (NHLBI) ,
Synopsis: To accelerate research in the management of thalassemia, standardize existing treatments, and evaluate new ones in a network of clinical centers. The emphasis will be on clinical trials that help identify optimal therapy. Therapeutic trials may involve investigational drugs, drugs already approved but not currently used, and drugs currently used.
Details: BACKGROUND: Cooley's anemia (beta-thalassemia major, hereafter referred to as thalassemia) is a severe, inherited blood disorder characterized by a quantitative defect in the synthesis of the beta chain of hemoglobin caused by any one of more than 100 known mutations in and around the beta globin gene cluster. The disease is characterized by severe anemia beginning in the first six to twelve months of life. If untreated, the life expectancy is less than five years of age. Chronic red blood cell transfusions to maintain hemoglobin levels between nine and 11 gm/dl ("hypertransfusion") alleviate the anemia and partially suppress erythropoiesis. The regular administration of red blood cells also improves growth, delays or prevents enlargement of the liver, and spleen, and prevents the development of bone abnormalities that cause fractures as well as disfiguring changes known as Cooley's facies. Transfusions carry risks of alloimmunization, iron overload, and blood transmitted infections. In the absence of effective iron chelation therapy, iron overload leads to numerous complications including delayed or absent sexual development, diabetes mellitus, cirrhosis, cardiac arrhythmias, and congestive heart failure. Non-chelated or poorly chelated patients usually die of heart disease by 20 to 30 years of age. The majority of patients with thalassemia major transfused prior to the late 1980's are infected with hepatitis C, and this may also prove to be a significant cause of morbidity and mortality in this hematologic disorder. The addition of chelation therapy with deferoxamine to the treatment of Cooley's anemia has dramatically improved the outcome for affected patients. With regular chelation therapy, the accumulation of excessive iron can be prevented. Studies have demonstrated that well-chelated patients have normal or only modest increases in liver iron, improved growth, sexual development, and most importantly, a markedly reduced chance of developing iron induced heart disease. In the past few years, several new approaches to the treatment of thalassemia have included marrow or stem cell transplantation, the use of young red blood cells ("neocytes" for transfusion), maintenance of a higher pre-transfusion hemoglobin level, new iron chelators, and the use of drugs such as hydroxyurea, erythropoietin, and butyrate compounds. It is recognized that even with a clinical network, the number of patients with Cooley's Anemia who can be enrolled in a research protocol is likely to be small. Therefore, although a randomized clinical trial may be the preferred way of assessing the clinical benefits of a new therapy, it may not be feasible in some instances, even using biomarkers or other surrogate outcome measures. Depending upon the specific questions being addressed, other study designs might be appropriate. These might include pre- and post-treatment assessment or historical control studies. In all cases, the proposed design, including sample size would be evaluated by the Protocol Review Committee. There is an urgent need to evaluate new and existing therapeutic approaches for persons with thalassemia, and to disseminate the findings to health care professionals, patients and the public. There are several reasons why a thalassemia clinical research network will accelerate clinical research and meet this need. The highly variable and sometimes complicated clinical manifestations of thalassemia often make it difficult to accumulate a large number of comparable patients in one center. Further, uniformity in treatment protocols may reduce the number of patients needed at each clinical center. Also, the Thalassemia Clinical Research Network mechanism will help pool the necessary clinical expertise and administrative resources to facilitate the conduct of multiple and novel therapeutic trials in a timely, efficient manner. This, in turn, would promote rapid dissemination of research findings to health care professionals. The Request for Applications was released in March, 1999. Awards were made in July, 2000. DESIGN NARRATIVE: Registry: The registry is collecting routine clinical care baseline data on patients with thalassemia. Specific aims include: determining the prevalence of thalassemia and its complications in North America; determining the availability of potential participants for the research network studies; using information from the registry to design clinical research studies relevant to the patient population and to the goals of the network; defining broadly the goals of future research in thalassemia. Osteoporosis Cross-Sectional Study: Cross-sectional observational data are collected on osteoporosis in thalassemia, including the prevalence of osteoporosis in thalassemia and the interaction of auxosomal, hematopoietic, endocrine, and genetic factors in the bone metabolism of patients with thalassemia. Osteoporosis is emerging as a major cause of morbidity in the aging thalassemic population. Fracture on minimal trauma and back pain due to vertebral compression fractures have become a common occurrence. The pathogenesis of osteoporosis is unclear and multifactorial. All patients in the thalassemia network will be entered into the cross sectional study of osteoporosis with an eligible subset entered into the intervention study. Osteoporosis Intervention Study: This is a randomized, controlled trial of combination bisphosphonate therapy plus supportive care versus supportive care alone for osteoporosis in thalassemia. The study will determine if osteoporosis can be improved with the addition of bisphosphonate therapy using intravenous pamidronate rather than the standard care of calcium and vitamin D supplementation alone. The study will also address whether certain factors impact on the response to treatment. These factors will be evaluated on enrollment and include treatment regimens, complications of iron overload, endocrine and genetic factors, and immune function. Response to treatment will be monitored by Bone Mineral Density (BMD) upon enrollment and annually during the two year treatment period and at completion of the study in year three. Markers of bone turnover will be performed on enrollment and quarterly during the study. One hundred patients with thalassemia major and intermedia will be enrolled with approximately fifty patients in each treatment arm. Patients will be stratified according to the Tanner stage of sexual development. Hepatitis C Study: The randomized trial will evaluate the efficacy of treatment with PEGylated interferon-alpha (IFN-alpha) versus PEGylated IFN-alpha and ribavirin in producing a sustained virological and clinical response (changes in liver histology) in beta-thalassemia major patients with hepatitis C infection. Hemoglobin F Induction Study: In this two-armed, cross-over study patients will be randomized to start SPB + HU or AraC, based on age. Twenty five patients will be randomized to start each arm. There will be early stopping with one interim look after the first 15 patients complete each arm. ICL670 Study: This two-armed, randomized, open label study compares the efficacy and safety of long-term treatment with ICL670 with deferoxamine in beta-thalassemia patients with transfusional hemosiderosis. Pilot DFO/L1 Cardiac Clinical Trial: This is a multicenter, randomized, double-blind, placebo-controlled trial comparing deferoxamine (DFO) with or without oral deferiprone (L1) on left ventricular function assessed by magnetic resonance imaging (MRI) in adults with transfusion-dependent beta-thalassemia. The trial is under development.
Eligibility:
Study Type: Interventional, Treatment, Randomized
Minimum Age/Maximum Age: 1 Year/75 Years
Genders: Both
Protocol Entry Criteria: Thalassemic patients of both sexes, including children.
Total Enrollment:
Location and Contact Information:
Overall Study Official:
AlanCohen, , Children's Hospital of Philadelphia
Children's Hospital Oakland *Recruiting*
Oakland, California, 94609
United States
Recruiting Elliott Vichinsky 510-428-3651
Weill Medical College of Cornell University *Recruiting*
New York City, New York, 10021
United States
Recruiting Patricia Giardina 212-746-3415
Children's Hospital of Philadelphia *Recruiting*
Philadelphia, Pennsylvania, 19104
United States
Recruiting Alan Cohen 215-590-3438
Hospital for Sick Children *Recruiting*
Toronto, Ontario, M5G 2C4
Canada
Recruiting Nancy Oliverieri (416) 340-3979
Children's Hospital *Recruiting*
Boston, Massachusetts, 02115
United States
Recruiting Ellis Neufeld 617-355-8183
Additional Information:
Study ID Numbers: 317;
Study Start Date: July 2000
Record last reviewed: May 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00000623
Other Osteoporosis Studies:
1. Blood and Marrow Transplant Clinical Research Network
2. Investigation of Selected Patient Groups From The Cooperative Study of Sickle Cell Disease
3. Hepatitis C in Clinically Discordant Hemophilic Siblings
4. Pediatric Hydroxyurea in Sickle Cell Anemia (BABY HUG)
5. Cytomegalovirus Spread and Reactivation in Blood Cells
Related Studies:
Other Osteoporosis Clinical Trials
Other Massachusetts Clinical Trials
Other Boston Clinical Trials
Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)
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