Clinical trial NCT01123863: The Brigance Assessment Of Individual Neurodevelopment In Young Children With Sickle Cell Disease- 2
Official title: The Brigance Assessment Of Individual Neurodevelopment In Young Children With Sickle Cell Disease- 2
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Keywords: Neuro cognitive deficits;
Brigance pre-school screen;
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
First posted: 2010-05-14
Enrollment: 248
Sex: ALL
Minimum age (years): 3
Maximum age (years): 4
Standard ages: CHILD
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

Brief 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. 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:

1. To compare the raw scores of the Brigance Preschool Screen II between SCD and control groups.
2. 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.
3. To compare the pass/fail rate and the raw scores between SCD patients on hydroxyurea treatment and the healthy controls
4. To assess the influence of medical factors on the Brigance Preschool Screen II performance in children with SCD.
5. To assess the influence of socioeconomic factors on the Brigance Preschool II results in children with SCD and controls.

Eligibility criteria: PATIENT GROUP

Inclusion criteria:

* 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

Exclusion criteria:

* 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

Inclusion criteria:

* 3.0-\<4.0 years of age
* African-American
* Attendee of day-care in Memphis area

Exclusion criteria:

1. Known diagnosis associated with significant cognitive impairment (e.g. stroke, Down syndrome, mental retardation)
2. Known diagnosis sickle cell disease (HbSS, HbSC, HbSβºthalassemia, HbSβ\^+thalassemia)
3. Previously tested with Brigance Preschool Screen-II

Primary outcomes:
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.
timeFrame: 4 years
Secondary outcomes:
measure: Compare the raw scores of the Brigance Preschool Screen II between SCD and control groups.
timeFrame: 4 years
measure: 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.
timeFrame: 4 years
measure: Compare the pass/fail rate and the raw scores between SCD patients on hydroxyurea treatment and the healthy controls
timeFrame: 4 years
measure: Assess the influence of medical factors on the Brigance Preschool Screen II performance in children with SCD.
timeFrame: 4 years
measure: Assess the influence of socioeconomic factors on the Brigance Preschool II results in children with SCD and controls.
timeFrame: 4 years
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Jeremie Estepp, MD
role: PRINCIPAL_INVESTIGATOR
References: