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NCT Number: NCT01137721

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.

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Key information

Conditions

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

Age range

5 year–19 year

Sex eligibility

All sexes

Study type

Observational

Primary location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

  • The diagnosis of HbSS or HbS/ß0-thalassemia
  • Age: 8.0 -- <19 years old

Inclusion criteria

for Study Participants for Observation:

  • The diagnosis of HbSS or HbS/ß0-thalassemia
  • Age: 8.0 -- <19 years old

Inclusion criteria

for Study Participants for Family Related Controls:

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

Exclusion criteria

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

  • 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.

Exclusion criteria

for Study Participants for Observation:

  • 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.

Exclusion criteria

for Study Participants for Family Related Controls:

  • 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.

Treatment and study plan

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

Sponsors and collaborators

Lead sponsor

St. Jude Children's Research Hospital

Other

Registry information

Important dates

Study start
2010
Primary completion
2016
Study completion
2016
First posted
Jun 4, 2010
Registry last updated
Aug 23, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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