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State Of The Art Functional Imaging In Sickle Cell Disease

This study is currently recruiting participants.
Verified November 2012 by St. Jude Children's Research Hospital
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT01137721
First received: June 3, 2010
Last updated: November 13, 2012
Last verified: November 2012
History of Changes
  Purpose

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.


Condition
Sickle Cell Anemia

Study Type: Observational
Study Design: Observational Model: Case Control
Time Perspective: Prospective
Official Title: State Of The Art Functional Imaging In Sickle Cell Disease

Resource links provided by NLM:

Drug Information available for: Hydroxyurea
U.S. FDA Resources

Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
  • Change in cerebral blood flow [ Time Frame: from baseline to 12 +/- 3 months ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Change in cerebral blood flow by territory [ Time Frame: From baseline to 12 +/- 3 months ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 60
Study Start Date: September 2010
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts
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.

Detailed 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).

  Eligibility

Ages Eligible for Study:   5 Years to 19 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
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.

Criteria

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

  1. The diagnosis of HbSS or HbS/ß0-thalassemia
  2. Age: 5.0 -- <19 years old

Inclusion Criteria for Study Participants for Observation:

  1. The diagnosis of HbSS or HbS/ß0-thalassemia
  2. Age: 5.0 -- <19 years old

Inclusion Criteria for Study Participants for Family Related Controls:

  1. No diagnosis of HbSS or HbS/ß0-thalassemia
  2. Age: 5.0 -- <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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01137721

Contacts
Contact: Kathleen J Helton, M.D 1-866-278-5833 info@stjude.org

Locations
United States, Tennessee
St. Jude Children's Research Hospital Recruiting
Memphis, Tennessee, United States, 38105
Contact: Kathleen J Helton, M.D     866-278-5833     info@stjude.org    
Principal Investigator: Kathleen J Helton, M.D            
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
Principal Investigator: Kathleen J Helton, M.D St. Jude Children's Research Hospital
  More Information

Additional Information:
St. Jude Children's Research Hospital  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT01137721     History of Changes
Other Study ID Numbers: SCDMR4
Study First Received: June 3, 2010
Last Updated: November 13, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by St. Jude Children's Research Hospital:
magnetic resonance imaging
sickle cell disease
hydroxyurea therapy

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
 Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antisickling Agents
Hematologic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors

ClinicalTrials.gov processed this record on May 23, 2013