Sickle Cell AnemiaNCT01137721
State Of The Art Functional Imaging In Sickle Cell Disease
An observational study of Sickle Cell Anemia, run by St. Jude Children's Research Hospital at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 5 to 19 yearsHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell Anemia
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2010to Jun 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 19 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
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.
Who may be able to join
- 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:
Show all 11Show fewer
- 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.
Who cannot join
- 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:
Show all 11Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
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.
Read the 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.
- Conditions
- Sickle Cell Anemia
- Keywords
- magnetic resonance imaging, sickle cell disease, hydroxyurea therapy
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: from baseline to 12 +/- 3 months
Secondary outcomes (1)
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.
Measured: From baseline to 12 +/- 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2010 (estimated)
- Study startSep 2010
- Primary completionJun 2016
- Study completionJun 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Robert Ogg, M.D, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01137721
- Sponsor's protocol ID
- SCDMR4
- Status last verified
- Aug 2016
- First submitted
- 3 Jun 2010
- Last updated
- 23 Aug 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- State Of The Art Functional Imaging In Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01137721
- Acronym / sponsor ID
- SCDMR4
- Conditions
- Sickle Cell Anemia
- Keywords
- magnetic resonance imaging, sickle cell disease, hydroxyurea therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Sep 2010
- Primary completion
- Jun 2016 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 4 Jun 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 5 to 19 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (11 listed)
- The diagnosis of HbSS or HbS/ß0-thalassemia
- Age: 8.0 -- <19 years old
- No diagnosis of HbSS or HbS/ß0-thalassemia
- Age: 8.0 -- <19 years old
- Unable to tolerate the anatomical or fMRI [functional magnetic resonance imaging] without sedation or anesthesia
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
1 secondary outcome
Change in cerebral blood flow by territory
Time frame: From baseline to 12 +/- 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2018.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Ogg, M.D principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Anemia, run by St. Jude Children's Research Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Aug 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in cerebral blood flow (time frame: from baseline to 12 +/- 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Aug 2018.