Natural History of Sickle Cell Disease

Studies of the Natural History of Sickle Cell Disease


Lead Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Source National Institutes of Health Clinical Center (CC)
Brief Summary

This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated. Subjects will receive their general medical care outside the NIH and will be seen at our clinic or at CNHS with varying frequency. Subjects may be seen for multiple visits. Subjects may be asked to return for additional testing as needed. Clinical care for patients with sickle cell disease will be provided as appropriate through the Sickle Cell Clinic and the inpatient clinical center.

Detailed Description

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.

Overall Status Recruiting
Start Date April 29, 2004
Study Type Observational
Primary Outcome
Measure Time Frame
gather through clinical experience information regarding the natural history, co-morbid conditions and outcomes, and complicationsrelating to sickle cell disease and other hemolytic disorders in minority/ethnic patients Ongoing
Enrollment 3500

Sampling Method: Non-Probability Sample



- 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).


- Patient and/or guardian unable/unwilling to give informed consent or assent.

- Patients less than 2 years of age.

Gender: All

Minimum Age: 2 Years

Maximum Age: 99 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Swee Lay Thein, M.D. Principal Investigator National Heart, Lung, and Blood Institute (NHLBI)
Overall Contact

Last Name: Nancy A Asomaning

Phone: (301) 402-2105

Email: [email protected]

Facility: Status: Contact:
Childrens National Medical Center | Washington, District of Columbia, United States Recruiting Joshua Coons 202-476-2281
National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda, Maryland, 20892, United States Recruiting For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 TTY8664111010 [email protected]
Location Countries

United States

Verification Date

July 7, 2020

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Arm Group

Label: Patients

Description: Individuals with known or suspected sickle cell disease

Study Design Info

Observational Model: Case-Only

Time Perspective: Prospective