Blood Sampling for Research Related to Sickle Cell Disease

ID: NCT00542230
Status: Enrolling by invitation
Phase: N/A
Start Date: October 09, 2007
First Submitted: October 10, 2007
Last Updated: February 22, 2018
Results: N/A
Sponsors & Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Location: United States
Conditions: Sickle Cell Trait, Sickle Cell Disease, Sickle Cell Anemia
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Study Description

Brief Summary

This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body. The purpose of the study is to collect a variety of blood samples that may then be used to investigate advances and potential new drug treatments for sickle cell disease.

Volunteers must be at least 8 years of old. Samples will be taken both from healthy volunteers and from volunteers who have unique red blood cell features that are related to sickle cell disease. Candidates will be screened with a medical history.

During the study, participants will undergo a one- to two-hour outpatient procedure at the National Institutes of Health Clinical Center. Once researchers have explained the study and obtained the participant s consent, participants will donate 8 cc (approximately 2 teaspoons) of blood.

Because repeat testing helps researchers validate study findings, participants who have the unique red blood cell features mentioned above may also be asked if they are willing to return and donate another 2 cc to 8 cc of blood for additional studies. The amount of blood drawn will not exceed 50 ml with any eight-week period for adults or 7 cc within any six-week period for children.

Detailed Description

The critical event leading to serious morbidity in sickle cell disease is polymerization of the abnormal hemoglobin to form fibers that stiffen and distort red blood cells and consequently, cause vaso-occlusion in the small vessels of the tissues. Currently, hydroxyurea is the only approved drug for treating sickle cell disease, but is only partially effective. This protocol seeks to identify by high sensitivity in vitro screening methods additional compounds that inhibit sickling. Subjects with sickle cell trait or disease and normal volunteers will be asked to donate blood samples for studies to verify their hemoglobinopathies and then periodically donate small volume samples of fresh blood for use in ongoing high throughput screening tests to identify potentially clinically useful anti-sickling drugs.
Condition or disease Intervention/treatment Phase

Sickle Cell Anemia

Sickle Cell Disease

Sickle Cell Trait

N/A

Tracking Information

First Submitted DateOctober 10, 2007
Last Update Posted DateFebruary 22, 2018
Start DateOctober 09, 2007
Completion DateN/A
Primary Completion DateN/A
Results First Submitted DateN/A
Received Results Disposit DateN/A

Current Primary Outcome Measures

Not Available

Original Primary Outcome Measures

Not Available

Current Secondary Outcome Measures

Not Available

Original Secondary Outcome Measures

Not Available

Study Design

Brief TitleBlood Sampling for Research Related to Sickle Cell Disease
Official TitleHigh Sensitivity Screening of Compound Libraries to Discover a Drug for the Treatment of Sickle Cell Disease
Brief Summary

This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body. The purpose of the study is to collect a variety of blood samples that may then be used to investigate advances and potential new drug treatments for sickle cell disease.

Volunteers must be at least 8 years of old. Samples will be taken both from healthy volunteers and from volunteers who have unique red blood cell features that are related to sickle cell disease. Candidates will be screened with a medical history.

During the study, participants will undergo a one- to two-hour outpatient procedure at the National Institutes of Health Clinical Center. Once researchers have explained the study and obtained the participant s consent, participants will donate 8 cc (approximately 2 teaspoons) of blood.

Because repeat testing helps researchers validate study findings, participants who have the unique red blood cell features mentioned above may also be asked if they are willing to return and donate another 2 cc to 8 cc of blood for additional studies. The amount of blood drawn will not exceed 50 ml with any eight-week period for adults or 7 cc within any six-week period for children.

Detailed Description

The critical event leading to serious morbidity in sickle cell disease is polymerization of the abnormal hemoglobin to form fibers that stiffen and distort red blood cells and consequently, cause vaso-occlusion in the small vessels of the tissues. Currently, hydroxyurea is the only approved drug for treating sickle cell disease, but is only partially effective. This protocol seeks to identify by high sensitivity in vitro screening methods additional compounds that inhibit sickling. Subjects with sickle cell trait or disease and normal volunteers will be asked to donate blood samples for studies to verify their hemoglobinopathies and then periodically donate small volume samples of fresh blood for use in ongoing high throughput screening tests to identify potentially clinically useful anti-sickling drugs.

Study TypeObservational
Study PhaseN/A
Estimated Enrollment
36
Allocation
Not Available
Interventional Model
Not Available
Masking
Not Available
Primary Purpose
Not Available
Conditions
Sickle Cell Anemia
Sickle Cell Disease
Sickle Cell Trait
Target Follow-Up Duration N/A
Biospecimen:
N/A
Sampling MethodN/A
Study PopulationN/A
Intervention
Not Available
Study Groups/Cohorts
Not Available
Study Arms
Not Available
Arm Intervention/Treatment

Recruitment Information

Recruitment Status:Enrolling by invitation
Enrollment36
Completion DateN/A
Eligibility Criteria: - INCLUSION CRITERIA:
- Patients with sickle cell trait
- Patients with known hemoglobinopathies involving one or two genes for sickle hemoglobin
- Healthy volunteers for control experiments
- Women who are pregnant or breastfeeding are eligible to enroll in this protocol.
- A pregnant or breastfeeding woman will not be enrolled of the donation of research bloos samples poses any clinical risk.
- No pregnant minors will be enrolled.
- Age range: Minors greater than or equal to 8 years of age and adults greater than or equal to 18 years of age
EXCLUSION CRITERIA:
- Subjects who are unable to comprehend the investigational nature of the laboratory research are ineligible to enroll in this protocol.
- As a safety precaution in handling the blood samples, patients with HIV, Hepatitis B or Hepatitis C will be excluded from the study. HIV, Hepatitis B or Hepatitits C testing will not be done under this study. Participants must be co-enrolled under another NIH protocol where the screening evalustion has been performed.
GenderAll
Age8 Years to N/A
Accepts Healthy VolunteersAccepts Healthy Volunteers
Contacts
Not Available
Listed Location Countries
United States

Administrative Information

NCT Number:NCT00542230
Other Study ID Numbers
080004
08-DK-0004
Has Data Monitoring CommitteeNo
U.S. FDA-regulated Product Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Device Product Not Approved or Cleared by U.S. FDA: No
IPD Sharing Statement
Not Available
Responsible Party,
Study Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborators
Not Available
Investigators
Principal Investigator
William A Eaton, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)