State Of The Art Functional Imaging In Sickle Cell Disease

NCT01137721 | Completed

• 1 other identifier: SCDMR4
• Study Type: observational
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

Sickle cell anemia (SCA) is a serious blood disease with blood vessel changes leading to brain injury and stroke. Studies show about 11% of patients with SCA will develop obvious stroke before age 20 years, with children less than 10 years of age especially vulnerable. The main objective of the SCDMR4\[State Of The Art Functional Imaging In Sickle Cell Disease\] trial is to compare the gray matter cerebral blood flow, measured by MRI,\[magnetic resonance imaging\] ASL \[Arterial Spin Labeling\] perfusion before treatment begins and after the appropriate hydroxyurea dosage is reached (\~ one year). Other important objectives of the SCDMR4 trial include describing the effect of hydroxyurea therapy and transfusion therapy on the functional MRI response, diffusion tensor imaging of white matter, brain function, and transcranial Doppler blood velocities.

Conditions

Sickle Cell Anemia

Keywords

magnetic resonance imaging; sickle cell disease; hydroxyurea therapy

Outcome Measures

Primary Outcomes (1)

• Change in cerebral blood flow

  Change in gray matter cerebral blood flow measured by arterial spin labeling techniques from before (baseline) to after reaching a stable hydroxyurea maximum tolerated dose.

  from baseline to 12 +/- 3 months

Secondary Outcomes (1)

• Change in cerebral blood flow by territory

  From baseline to 12 +/- 3 months

Study Arms (4)

Pre-Hydroxyurea - subjects with SCD

Patients with a diagnosis of HbSS (sickle cell anemia) or HbS/ß0-thalassemia (beta thalassemia) who will be treated with hydroxyurea therapy.

Sibling control

Sibling control with no diagnosis of HbSS or HbS/ß0-thalassemia.

Observational - subjects with SCD

Patients with a diagnosis of HbSS or HbS/ß0-thalassemia.

Pre-transfusion - subjects with SCD

Patients with a diagnosis of HbSS or HbS/ß0-thalassemia who will be treated with transfusion therapy.

Eligibility Criteria

• Age: 5 Years - 19 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

The potential research participants will be recruited, screened and consented from the School-Age and Teen Sickle Cell Clinics by the referring St. Jude hematology co-investigators. There will be no advertisement per se. Affiliate hematology co-investigators will contact St. Jude hematology co-investigators about potentially eligible research participants. Affiliate potential research participants will be screened, and if appropriate, consented and tested at St. Jude institution.

You may qualify if:

• The diagnosis of HbSS or HbS/ß0-thalassemia
• Age: 8.0 -- \<19 years old
• The diagnosis of HbSS or HbS/ß0-thalassemia
• Age: 8.0 -- \<19 years old
• No diagnosis of HbSS or HbS/ß0-thalassemia
• Age: 8.0 -- \<19 years old

You may not qualify if:

• Unable to tolerate the anatomical or fMRI \[functional magnetic resonance imaging\] without sedation or anesthesia
• Currently receiving hydroxyurea therapy or transfusion therapy
• Previous stem cell transplant or other myelosuppressive therapy
• History of clinical stroke
• Inability or unwillingness of research participant or legal guardian/representative to give written informed consent/assent.
• Unable to tolerate anatomical or fMRI components without sedation or anesthesia
• Currently receiving hydroxyurea or transfusion therapy
• Previous stem cell transplant or other myelosuppressive therapy
• History of clinical stroke
• Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
• Unable to tolerate anatomical or fMRI components without sedation or anesthesia
• Currently receiving hydroxyurea or transfusion therapy
• Previous stem cell transplant or other myelosuppressive therapy
• History of clinical stroke
• Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Robert Ogg, M.D

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 3, 2010
• First Posted: June 4, 2010
• Study Start: September 1, 2010
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2016
• Last Updated: August 23, 2018

Record last verified: 2016-08

Locations

US (1)