Sickle Cell DiseaseNCT01123863
The Brigance Assessment Of Individual Neurodevelopment In Young Children With Sickle Cell Disease- 2
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
A preliminary study was conducted involving 88 three-year-old children with sickle cell disease (SCD) who were followed at the St. Jude Children's Research Hospital Sickle Cell Center. Read more about this study
- Who is it for?
- Ages 3 to 4 yearsHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Brigance Preschool Screen -II
- How many people
- 248 participants
- Where
- 1 siteUnited States
- Study dates
- May 2010to May 2015
- 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 3 to 4 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: The patient group will consist of 3 year-old-children with SCD who are followed at the St. Jude Children's Research Hospital Sickle Cell Center. The control group will consist of 3-year-old children attending day care in Memphis.
Who may be able to join
- 3.0-<4.0 years of age
- African-American
- Diagnosis of sickle cell disease (HbSS, HbSC, HbSβºthalassemia, HbSβ^+thalassemia)
- Followed at St. Jude Children's Research Hospital Sickle Cell Center
- 3.0-<4.0 years of age
- African-American
- Attendee of day-care in Memphis area
Who cannot join
- Previous stroke
- Patients who are currently on a chronic transfusion program
- Known diagnosis associated with significant cognitive impairment (e.g. Down syndrome, mental retardation)
- Previously tested with Brigance Preschool Screen-II
- CONTROL GROUP
- Known diagnosis associated with significant cognitive impairment (e.g. stroke, Down syndrome, mental retardation)
- Known diagnosis sickle cell disease (HbSS, HbSC, HbSβºthalassemia, HbSβ^+thalassemia)
- Previously tested with Brigance Preschool Screen-II
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
A preliminary study was conducted involving 88 three-year-old children with sickle cell disease (SCD) who were followed at the St. Jude Children's Research Hospital Sickle Cell Center. They were offered developmental screening with the Brigance Preschool Screen-II test during their regular clinic visits from January 2006 to August 2008. Data from this work showed that 50% of 3 year old children with SCD had low developmental screening scores. In addition, the low scores were found to be associated with less parental education and with speech deficits. However they were not associated with sickle cell genotype and hemoglobin level.
The primary goal of this study is to prospectively administer Brigance Preschool Screen -II to 3 year old children with SCD and 3 year old children without SCD who come from similar socioeconomic backgrounds and compare the results between the two groups.
Read the detailed description
The primary objective of this study is to compare development in 3 year old children with SCD who are not on any treatment to age matched healthy controls using pass/fail rate for the Brigance Preschool Screen II.
Secondary objectives:
- To compare the raw scores of the Brigance Preschool Screen II between SCD and control groups.
- To compare the pass/fail rate and the raw scores between SCD patients who are not on any treatment and those who are being treated with hydroxyurea.
- To compare the pass/fail rate and the raw scores between SCD patients on hydroxyurea treatment and the healthy controls
- To assess the influence of medical factors on the Brigance Preschool Screen II performance in children with SCD.
- To assess the influence of socioeconomic factors on the Brigance Preschool II results in children with SCD and controls.
- Conditions
- Sickle Cell Disease
- Being tested
- Brigance Preschool Screen -II
- Keywords
- Neuro cognitive deficits;, Brigance pre-school screen;
- 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.
Brigance Preschool Screen -IIOther
Three-year-old children with SCD who are followed at the St. Jude Children's Research Hospital Sickle Cell Center will be offered developmental screening with the Brigance Preschool Screen II during their regular clinic visits. Along with the screening test, socioeconomic data will be collected using a short questionnaire. Families will be asked to participate in this study by allowing us to collect their test results, medical information and socioeconomic data to compare with data collected from the control group. Parents who decline participation in this study will still be offered the Brigance Preschool Screen II as part of their child's clinical care.
Brigance Preschool Screen -IIOther
Children in the Control Group will be given the Brigance preschool Screen-II Screening at the daycare they attend. The STARR coordinators have selected four daycare centers that have agreed to serve as control group sites. The family will be asked not to respond or aid the child during the assessment. One parent/guardian will fill out the Socioeconomic Data Collection Form while the examiner works with the child.
The screening requires the child to give both verbal and non-verbal responses. The examiner records responses on the Three-Year-Old Child Data Sheet for the Brigance Preschool Screen-II. After the screening is complete the examiner collects the Socioeconomic Data Collection Form from the parent and leaves the room to score the assessment and write recommendations. The examiner then returns to the clinic room to discuss the results with the parent/guardian.
| Study group | Role | What they receive |
|---|---|---|
| Patient Group | This study will administer Brigance Preschool Screen -II to 3 year old children with SCD followed at St. Jude Children's Research Hospital
Intervention: Brigance Preschool Screen -II Receives: Other: Brigance Preschool Screen -II | |
| control group | The control group will consist of 3-year-old children attending day care in the Memphis area and serve as a population that come from a similar socioeconomic background as the SCD patient population.
Intervention: Brigance Preschool Screen -II Receives: Other: Brigance Preschool Screen -II |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare development in 3 year old children with SCD who are not on treatment to age matched controls using pass/fail rate for the Brigance Preschool Screen II.
Measured: 4 years
Secondary outcomes (5)
Compare the raw scores of the Brigance Preschool Screen II between SCD and control groups.
Measured: 4 years
Compare the pass/fail rate and the raw scores between SCD patients who are not on any treatment and those who are being treated with hydroxyurea.
Measured: 4 years
Compare the pass/fail rate and the raw scores between SCD patients on hydroxyurea treatment and the healthy controls
Measured: 4 years
Assess the influence of medical factors on the Brigance Preschool Screen II performance in children with SCD.
Measured: 4 years
Assess the influence of socioeconomic factors on the Brigance Preschool II results in children with SCD and controls.
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2010 (estimated)
- Study startMay 2010
- Primary completionMay 2015
- Study completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 248 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jeremie Estepp, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01123863
- Sponsor's protocol ID
- BRAIN2
- Status last verified
- Jun 2015
- First submitted
- 12 May 2010
- Last updated
- 28 Jun 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Brigance Assessment Of Individual Neurodevelopment In Young Children With Sickle Cell Disease- 2
- ClinicalTrials.gov ID
- NCT01123863
- Acronym / sponsor ID
- BRAIN2
- Conditions
- Sickle Cell Disease
- Keywords
- Neuro cognitive deficits;, Brigance pre-school screen;
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 248 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- May 2010
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 14 May 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.
A preliminary study was conducted involving 88 three-year-old children with sickle cell disease (SCD) who were followed at the St. Jude Children's Research Hospital Sickle Cell Center. They were offered developmental screening with the Brigance Preschool Screen-II test during their regular clinic visits from January 2006 to August 2008. Data from this work showed that 50% of 3 year old children with SCD had low developmental screening scores. In addition, the low scores were found to be associated with less parental education and with speech deficits. However they were not associated with sickle cell genotype and hemoglobin level.
The primary goal of this study is to prospectively administer Brigance Preschool Screen -II to 3 year old children with SCD and 3 year old children without SCD who come from similar socioeconomic backgrounds and compare the results between the two groups.
Detailed description
The primary objective of this study is to compare development in 3 year old children with SCD who are not on any treatment to age matched healthy controls using pass/fail rate for the Brigance Preschool Screen II.
Secondary objectives:
- To compare the raw scores of the Brigance Preschool Screen II between SCD and control groups.
- To compare the pass/fail rate and the raw scores between SCD patients who are not on any treatment and those who are being treated with hydroxyurea.
- To compare the pass/fail rate and the raw scores between SCD patients on hydroxyurea treatment and the healthy controls
- To assess the influence of medical factors on the Brigance Preschool Screen II performance in children with SCD.
- To assess the influence of socioeconomic factors on the Brigance Preschool II results in children with SCD and controls.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patient Group | – | Other: Brigance Preschool Screen -IIThis study will administer Brigance Preschool Screen -II to 3 year old children with SCD followed at St. Jude Children's Research Hospital Intervention: Brigance Preschool Screen -II |
| control group | – | Other: Brigance Preschool Screen -IIThe control group will consist of 3-year-old children attending day care in the Memphis area and serve as a population that come from a similar socioeconomic background as the SCD patient population. Intervention: Brigance Preschool Screen -II |
Who may qualify
- Ages
- 3 to 4 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- The patient group will consist of 3 year-old-children with SCD who are followed at the St. Jude Children's Research Hospital Sickle Cell Center. The control group will consist of 3-year-old children attending day care in Memphis.
Key inclusion criteria (7 listed)
- 3.0-<4.0 years of age
- African-American
- Diagnosis of sickle cell disease (HbSS, HbSC, HbSβºthalassemia, HbSβ^+thalassemia)
- Followed at St. Jude Children's Research Hospital Sickle Cell Center
- 3.0-<4.0 years of age
Key exclusion criteria (8 listed)
- Previous stroke
- Patients who are currently on a chronic transfusion program
- Known diagnosis associated with significant cognitive impairment (e.g. Down syndrome, mental retardation)
- Previously tested with Brigance Preschool Screen-II
- CONTROL GROUP
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Brigance Preschool Screen -IIThree-year-old children with SCD who are followed at the St. Jude Children's Research Hospital Sickle Cell Center will be offered developmental screening with the Brigance Preschool Screen II during their regular clinic visits. Along with the screening test, socioeconomic data will be collected using a short questionnaire. Families will be asked to participate in this study by allowing us to collect their test results, medical information and socioeconomic data to compare with data collected from the control group. Parents who decline participation in this study will still be offered the Brigance Preschool Screen II as part of their child's clinical care. | Other | Patient Group |
| Brigance Preschool Screen -IIChildren in the Control Group will be given the Brigance preschool Screen-II Screening at the daycare they attend. The STARR coordinators have selected four daycare centers that have agreed to serve as control group sites. The family will be asked not to respond or aid the child during the assessment. One parent/guardian will fill out the Socioeconomic Data Collection Form while the examiner works with the child. The screening requires the child to give both verbal and non-verbal responses. The examiner records responses on the Three-Year-Old Child Data Sheet for the Brigance Preschool Screen-II. After the screening is complete the examiner collects the Socioeconomic Data Collection Form from the parent and leaves the room to score the assessment and write recommendations. The examiner then returns to the clinic room to discuss the results with the parent/guardian. | Other | control group |
Primary outcome
Compare development in 3 year old children with SCD who are not on treatment to age matched controls using pass/fail rate for the Brigance Preschool Screen II.
Time frame: 4 years
5 secondary outcomes
Compare the raw scores of the Brigance Preschool Screen II between SCD and control groups.
Time frame: 4 years
Compare the pass/fail rate and the raw scores between SCD patients who are not on any treatment and those who are being treated with hydroxyurea.
Time frame: 4 years
Compare the pass/fail rate and the raw scores between SCD patients on hydroxyurea treatment and the healthy controls
Time frame: 4 years
Assess the influence of medical factors on the Brigance Preschool Screen II performance in children with SCD.
Time frame: 4 years
Assess the influence of socioeconomic factors on the Brigance Preschool II results in children with SCD and controls.
Time frame: 4 years
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 28 Jun 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.
Jeremie Estepp, MD 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 Disease, 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 7 inclusion and 8 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 28 Jun 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?
Compare development in 3 year old children with SCD who are not on treatment to age matched controls using pass/fail rate for the Brigance Preschool Screen II. (time frame: 4 years). 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 28 Jun 2018.