State Of The Art Functional Imaging In Sickle Cell Disease

August 21, 2018 updated by: St. Jude Children's Research Hospital
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.

Study Overview

Status

Completed

Conditions

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).

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.

Study Type

Observational

Enrollment (Actual)

38

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • St. Jude Children's Research Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 19 years (Child, Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
from baseline to 12 +/- 3 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
From baseline to 12 +/- 3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

St. Jude Children's Research Hospital

Investigators

  • Principal Investigator: Robert Ogg, M.D, St. Jude Children's Research Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2010

Primary Completion (Actual)

June 1, 2016

Study Completion (Actual)

June 1, 2016

Study Registration Dates

First Submitted

June 3, 2010

First Submitted That Met QC Criteria

June 3, 2010

First Posted (Estimate)

June 4, 2010

Study Record Updates

Last Update Posted (Actual)

August 23, 2018

Last Update Submitted That Met QC Criteria

August 21, 2018

Last Verified

August 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SCDMR4

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Sickle Cell Anemia

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Hungary

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe