Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Natural History of Sickle Cell Disease and Other Hemolytic Disorders

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2016 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier:
NCT00081523
First received: April 15, 2004
Last updated: September 3, 2016
Last verified: September 2016

April 15, 2004
September 3, 2016
April 2004
Not Provided
Not Provided
Not Provided
Complete list of historical versions of study NCT00081523 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Natural History of Sickle Cell Disease and Other Hemolytic Disorders
Studies of the Natural History of Sickle Cell Disease

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:

  • Routine blood and urine tests, including a 24-hour urine collection
  • Blood test for genetic analysis to look for genes that may be relevant to the development of sickle cell anemia or other hemolytic disorders
  • X-rays, if needed, to evaluate the patient's condition
  • Computerized tomography (CT) scans or magnetic resonance imaging (MRI), or both, as needed to examine the lungs, brain, or heart and to measure blood flow in these organs.
  • Echocardiogram (heart ultrasound) to determine if there is high blood pressure in the lungs
  • Pulmonary (lung) function tests and cardiopulmonary (heart and lung) exercise test to evaluate breathing and exercise capacity to determine if there are lung or heart problems associated with the patient's condition.
  • Heart catheterization to measure blood pressures in the heart and lung blood vessels will be offered if the results of clinical evaluation or echocardiogram suggest the presence of high blood pressure in the lungs. For this procedure, the patient is given a local anesthetic, and a catheter (plastic tube) is inserted into a vein in the arm, thigh, chest, or neck until it passes through the right side of the heart. When the catheter is in place, a tiny balloon at the end of the catheter is inflated, temporarily blocking the blood flow and allowing the physician to measure pressure in the lung artery system.
  • Ventilation-perfusion scan to measure breathing and circulation in all areas of the lungs. For the perfusion scan, radioactive albumin is injected into a vein and the patient is placed on a table that is positioned under the scanner. The lungs are scanned to detect the location of the radioactive particles as blood flows through the lungs. The ventilation scan is performed by scanning the lungs while the patient inhales a radioactive gas. A mask is placed over the nose and mouth, and the patient breathes the gas while sitting or lying on the table beneath the arm of the scanner. The gas is breathed out entirely in a matter of minutes and is removed from the room by a special exhaust system.

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 establishment of a repository of biospecimens from individuals affected with sickle cell disease to identify and evaluate underlying disease mechanism(s), and to facilitate understanding of the pathogenesis and natural history of vaso-occlusive painful crisis and other complications of the disease. 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. This protocol may also provide for the continued storage and analysis of clinical and research data, and biospecimens previously obtained from other NIH Institutional Review Board (IRB)-approved protocols with subject consent.

Patients eligible for other research protocols will be offered an opportunity to participate in these studies after signed informed consent has been obtained. Apart from such protocols, any medical care or additional tests/investigations 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

Observational
Not Provided
Not Provided
Not Provided
Not Provided
Not Provided
  • Hemoglobin SC Disease
  • Hematologic Diseases
  • Sickle Cell Anemia
  • Hemolytic Anemia
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
99999999
Not Provided
Not Provided
  • INCLUSION CRITERIA:
  • Individuals with known or suspected sickle cell disease
  • 2 years of age and older
  • Willingness and capacity to provide informed consent or appropriate informed consent from parent or legal guardian
  • Patients seen at sickle outpatient clinics at any one of the participating centers (CNHS or NIH).

EXCLUSION CRITERIA:

  • Patient and/or guardian unable/unwilling to give informed consent or assent.
  • Patients less than 2 years of age.
Both
5 Years and older   (Child, Adult, Senior)
No
Contact: James Nichols, R.N. (301) 435-2345 jnichols@mail.nih.gov
Contact: Swee Lay Thein, M.D. (301) 402-6699 sweelay.thein@nih.gov
United States
 
NCT00081523
040161, 04-H-0161
Not Provided
Not Provided
Not Provided
National Heart, Lung, and Blood Institute (NHLBI)
National Heart, Lung, and Blood Institute (NHLBI)
Not Provided
Principal Investigator: Swee Lay Thein, M.D. National Heart, Lung, and Blood Institute (NHLBI)
National Institutes of Health Clinical Center (CC)
September 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP