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State Of The Art Functional Imaging In Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT01137721
Recruitment Status : Completed
First Posted : June 4, 2010
Last Update Posted : August 2, 2016
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

June 3, 2010
June 4, 2010
August 2, 2016
September 2010
June 2016   (Final data collection date for primary outcome measure)
Change in cerebral blood flow [ Time Frame: from baseline to 12 +/- 3 months ]
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.
To compare the research participant's GM CBF by ASL techniques before and after reaching a stable hydroxyurea MTD (12±3 months after starting hydroxyurea). [ Time Frame: 3 years ]
Complete list of historical versions of study NCT01137721 on ClinicalTrials.gov Archive Site
Change in cerebral blood flow by territory [ Time Frame: From baseline to 12 +/- 3 months ]
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.
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State Of The Art Functional Imaging In Sickle Cell Disease
State Of The Art Functional Imaging In Sickle Cell Disease
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.

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.

Observational
Observational Model: Case Control
Time Perspective: Prospective
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Non-Probability Sample
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.
Sickle Cell Anemia
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  • 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.
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
38
79
June 2016
June 2016   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
5 Years to 19 Years   (Child, Adult)
Yes
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT01137721
SCDMR4
No
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St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
Not Provided
Principal Investigator: Kathleen J Helton, M.D St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
August 2016