Natural History of Sickle Cell Disease and Other Hemolytic Disorders
|First Received Date ICMJE||April 15, 2004|
|Last Updated Date||December 6, 2013|
|Start Date ICMJE||April 2004|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE||Not Provided|
|Original Primary Outcome Measures ICMJE||Not Provided|
|Change History||Complete list of historical versions of study NCT00081523 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE||Not Provided|
|Original Secondary Outcome Measures ICMJE||Not Provided|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||Natural History of Sickle Cell Disease and Other Hemolytic Disorders|
|Official Title ICMJE||Studies of the Natural History of Sickle Cell Disease and Other Hemolytic Disorders|
Under this study, NIH physicians will evaluate and treat patients with sickle cell disease and other hemolytic disorders (diseases involving damage to red blood cells) in order to learn more about the diseases and the painful attacks and lung complications that are often associated with them. Patients in this study will not receive experimental therapy, but will be given standard medical care. Patients who meet the medical criteria for another NIH protocol may be offered participation in that study, but are not obligated to enroll in it.
Patients 5 years of age and older with known or suspected sickle cell disease, sickle cell trait or another anemia or red blood cell disorder such as thalassemia may be eligible for this study. Participants will be admitted to the NIH Clinical Center as an inpatient or outpatient for evaluation and treatment. In addition to a physical examination, evaluation may include the following types of tests and procedures:
Patients are offered treatment for high blood pressure in the lungs. The medicines used are approved by the Food and Drug Administration for patients with pulmonary hypertension.
This protocol will permit the evaluation and treatment of subjects with hereditary and acquired hemolytic diseases, to facilitate understanding of the pathogenesis and natural history of vaso-occlusive painful crisis and pulmonary complications of sickle cell disease and related cardiopulmonary complications of other hereditary, acquired and iatrogenic hemolytic disorders. Patients will be evaluated with a medical history and physical examination and routine laboratory studies will be obtained as needed to assess diagnosis, disease activity, and disease complications and to monitor for treatment-related responses and toxicities. Blood can be obtained, with subject's consent, for research studies evaluating gene/protein expression and to evaluate the role of vasodialators, vasoconstrictors, inflammatory and redox stress mediators in this population. Patients identified with pulmonary hypertension will have the option to undergo invasive hemodynamic evaluation and treatment with FDA approved drugs, according to current standards of medical practice, with signed informed consent for all offered procedures. Patients eligible for other research protocols will be offered an opportunity to participate in theses studies by signed informed consent. Apart from such protocols, any medical care recommended or provided to the patient will be consistent with routine standards of practice and will be provided in consultation with the patient's referring physician. The establishment of this protocol will provide a means to evaluate and treat patients, and to generate hypotheses and protocols based on this clinical experience in sickle cell disease and in other acquired or inherited hemolytic anemias.
|Study Type ICMJE||Observational|
|Study Design ICMJE||Not Provided|
|Target Follow-Up Duration||Not Provided|
|Sampling Method||Not Provided|
|Study Population||Not Provided|
|Intervention ICMJE||Not Provided|
|Study Group/Cohort (s)||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Recruiting|
|Estimated Enrollment ICMJE||99999999|
|Completion Date||Not Provided|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
Patients with sickle cell disease and other hemolytic disorders will be evaluated at the NIH CCMD/NHLBI/NIDDK sickle cell clinic or day hospital. Patients will not be recruited or selected with reference to age, race or gender. The patient population is expected, however, to be predominantly minority in composition (mainly African-American and Latino, with some Asian, European and Native American patients as well) as function of demographics and genetics of these diseases. Children may be evaluated or treated in this protocol with parental informed consent and assent of the child when appropriate.
-Any concurrent condition (medical, social, behavioral) which in the opinion of the research team would preclude or confound acquisition/interpretation of data or delivery of clinical care.
|Ages||5 Years and older|
|Accepts Healthy Volunteers||No|
|Location Countries ICMJE||United States|
|NCT Number ICMJE||NCT00081523|
|Other Study ID Numbers ICMJE||040161, 04-H-0161|
|Has Data Monitoring Committee||Not Provided|
|Responsible Party||Not Provided|
|Study Sponsor ICMJE||National Heart, Lung, and Blood Institute (NHLBI)|
|Collaborators ICMJE||Not Provided|
|Information Provided By||National Institutes of Health Clinical Center (CC)|
|Verification Date||November 2013|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP