State Of The Art Functional Imaging In Sickle Cell Disease

Sickle Cell Anemia Clinical Trial

Official title:

State Of The Art Functional Imaging In Sickle Cell Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01137721
• Other study ID #: SCDMR4
• Status: Completed
• Start date: September 2010
• Est. completion date: June 2016

Study information

• Verified date: August 2016
• Source: St. Jude Children's Research Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

The Primary Objective of the study is to compare the research participant's GM [Gray Matter] CBF [Cerebral Blood Flow] by ASL [Arterial Spin Labeling] techniques before and after reaching a stable hydroxyurea MTD [Maximum Tolerated Dose] (12±3 months after starting hydroxyurea).

This is an observational study. Participants receive hydroxyurea as part of their standard of care treatment. This study will observe the above measures prior to beginning hydroxyurea and after participants reach the maximum tolerated dose in order to describe the effect of therapy on the participants' functional response.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 38
• Est. completion date: June 2016
• Est. primary completion date: June 2016
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 5 Years to 19 Years

Eligibility

Inclusion Criteria for Pre-Hydroxyurea or Pre-Transfusion Therapy Study Participants:

1. The diagnosis of HbSS or HbS/ß0-thalassemia

2. Age: 8.0 -- <19 years old

Inclusion Criteria for Study Participants for Observation:

1. The diagnosis of HbSS or HbS/ß0-thalassemia

2. Age: 8.0 -- <19 years old

Inclusion Criteria for Study Participants for Family Related Controls:

1. No diagnosis of HbSS or HbS/ß0-thalassemia

2. Age: 8.0 -- <19 years old

Exclusion Criteria for Pre-Hydroxyurea or Pre-Transfusion Therapy Study Participants:

1. Unable to tolerate the anatomical or fMRI [functional magnetic resonance imaging] without sedation or anesthesia

2. Currently receiving hydroxyurea therapy or transfusion therapy

3. Previous stem cell transplant or other myelosuppressive therapy

4. History of clinical stroke

5. Inability or unwillingness of research participant or legal guardian/representative to give written informed consent/assent.

Exclusion Criteria for Study Participants for Observation:

1. Unable to tolerate anatomical or fMRI components without sedation or anesthesia

2. Currently receiving hydroxyurea or transfusion therapy

3. Previous stem cell transplant or other myelosuppressive therapy

4. History of clinical stroke

5. Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Exclusion Criteria for Study Participants for Family Related Controls:

1. Unable to tolerate anatomical or fMRI components without sedation or anesthesia

2. Currently receiving hydroxyurea or transfusion therapy

3. Previous stem cell transplant or other myelosuppressive therapy

4. History of clinical stroke

5. Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Anemia

Locations

Country	Name	City	State
United States	St. Jude Children's Research Hospital	Memphis	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
St. Jude Children's Research Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change in cerebral blood flow by territory	Change in gray matter cerebral blood flow in individual anterior cerebral artery, middle cerebral artery, and posterior cerebral artery territories, and hemispheric gray matter measured by arterial spin labeling techniques from before (baseline) to after reaching a stable hydroxyurea maximum tolerated dose.	From baseline to 12 +/- 3 months

External Links

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