Clinical trial NCT03660774: A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia
Official title: Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence and Other Cognitive Functions (eTHINK)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Haemophilia A
Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2018-09-07
Enrollment: 563
Sex: MALE
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: ECOLOGIC_OR_COMMUNITY
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children and young adults with hemophilia A or B, all severities

Brief summary: The Hemophilia Growth and Development Study (HGDS) nearly 25 years ago showed haemophilia and HIV impacted brain development, thinking, and behaviour in children and adolescents. The eTHINK study is designed to understand whether advances in hemophilia treatment have removed any impact of hemophilia. If there still is an impact of hemophilia, the eTHINK study will help to identify which children or adolescents are still at risk, and how to screen other children. Participants and their child will participate in a comprehensive assessment of child's brain development, thinking and behavior through completing tests and questionnaires. There is only one study visit of 1 to 1.5 hours with no follow-up required


Eligibility criteria: Inclusion Criteria: - Male child or young adult (ages 1:0-3:6 and 4:0-21:11 years with diagnosis of congenital hemophilia A or B, any severity, with or without inhibitors - Parent able and willing to provide consent, and young adult with hemophilia (age 18-21) able to provide consent; children (ages 7-14) and adolescents/young adults (ages 14-17) able and willing to provide assent based upon local institutional policies - Parent able and willing to complete neurodevelopment and cognitive parent reported scales during comprehensive or other Hemophilia Treatment Center visit for about 45-60 minutes in English or Spanish depending on age group) - Children, adolescents and young adults with hemophilia (ages 1-21) able to participate in assessments of development and intelligence for about 40-45 minutes and adolescents and young adults (age 11-21) with hemophilia able and willing to complete additional self-assessment scales for about 30-45 minutes during comprehensive or other elective visit for in English or Spanish (depending on age group) - Provision of informed consent before the start of any study-related activities Exclusion Criteria: - Patient aged 3:7-3:11 years (43-47 months) - Prior participation in the study - Prior neurocognitive screening with the same or related instruments in the prior 6 months - Patients with hemophilia or their caregivers for whom test measures may be culturally inappropriate - Patients with hemophilia with known history of alcohol or substance abuse - Patients with hemophilia who have used opiates in the past 24 hours or other illicit drugs within the past 48 hours - Patients with hemophilia currently experiencing an acute bleed that has not resolved - Patients with hemophilia with recent head injury or concussion within the past 4 weeks - Concurrent diagnosis of another bleeding or thrombotic disorder - Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation

Primary outcomes:
measure: Cognition composite score according to the BAYLEY-III instrument
timeFrame: Day 1
measure: Behavioural Symptoms Index according to Behavior Assessment System for Children - Third Edition (BASC-3) parent rating scale
timeFrame: Day 1
measure: Adaptive behaviour domain score according to Adaptive Behavior Assessment System, Third Edition (ABAS-3) rating scale
timeFrame: Day 1
measure: Global executive composite index according to Behavior Rating Inventory of Executive Function (BRIEF)
timeFrame: Day 1
measure: Attention and processing speed (full scale IQ) according to Wechsler Preschool and Primary Scale of Intelligence-4th Edition (WPPSI-IV) for age group 4-6 years or Wechsler Abbreviated Scale of Intelligence, 2nd Edition (WASI-II) for age group 7-22 years
timeFrame: Day 1
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Orange
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: San Diego
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Aurora
country: United States
facility: Novo Nordisk Investigational Site
state: Colorado
city: Washington
country: United States
facility: Novo Nordisk Investigational Site
state: District of Columbia
city: Tampa
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Atlanta
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
city: Chicago
country: United States
facility: Novo Nordisk Investigational Site
state: Illinois
city: Indianapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Indiana
city: New Orleans
country: United States
facility: Novo Nordisk Investigational Site
state: Louisiana
city: Boston
country: United States
facility: Novo Nordisk Investigational Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Novo Nordisk Investigational Site
state: Michigan
city: Detroit
country: United States
facility: Novo Nordisk Investigational Site
state: Michigan
city: Minneapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Minnesota
city: Omaha
country: United States
facility: Novo Nordisk Investigational Site
state: Nebraska
city: Charlotte
country: United States
facility: Novo Nordisk Investigational Site
state: North Carolina
city: Cleveland
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Oklahoma City
country: United States
facility: Novo Nordisk Investigational Site
state: Oklahoma
city: Philadelphia
country: United States
facility: Novo Nordisk Investigational Site
state: Pennsylvania
city: Nashville
country: United States
facility: Novo Nordisk Investigational Site
state: Tennessee
city: Salt Lake City
country: United States
facility: Novo Nordisk Investigational Site
state: Utah
city: Seattle
country: United States
facility: Novo Nordisk Investigational Site
state: Washington
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Reporting Anchor and Disclosure (1452)
role: STUDY_DIRECTOR
References:
citation: Buranahirun C, Walsh KS, Mrakotsky C, Croteau SE, Rajpurkar M, Kearney S, Hannemann C, Wilkening GN, Shapiro KA, Cooper DL. Neuropsychological function in children with hemophilia: A review of the Hemophilia Growth and Development Study and introduction of the current eTHINK study. Pediatr Blood Cancer. 2020 Jan;67(1):e28004. doi: 10.1002/pbc.28004. Epub 2019 Oct 8.