A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia

Haemophilia A Clinical Trial

— eTHINK  

Official title:

Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence and Other Cognitive Functions (eTHINK)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03660774
• Other study ID #: HAEM-4436
• Secondary ID: U1111-1202-3415
• Status: Completed
• Start date: September 27, 2018
• Est. completion date: October 11, 2019

Study information

• Verified date: November 2019
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 563
• Est. completion date: October 11, 2019
• Est. primary completion date: October 11, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 1 Year to 21 Years

Eligibility

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

Related Conditions & MeSH terms

• Haemophilia A
• Haemophilia B
• Hemophilia A
• Hemophilia B

Intervention

Other:
• No treatment given
The participants will undergo a brief physical examination focused on neurologic function (muscle function, sensation, coordination, walking). Parents will be asked by the study team or the psychologist about their children and will complete several commonly used standard surveys about brain development, thinking, behavior, and decision making. The participants will also engage with the psychologist to evaluate development or thinking (IQ), and depending on their age they may be asked to complete one or more commonly used standard surveys about behavior and decision making.

Locations

Country	Name	City	State
United States	Novo Nordisk Investigational Site	Ann Arbor	Michigan
United States	Novo Nordisk Investigational Site	Atlanta	Georgia
United States	Novo Nordisk Investigational Site	Aurora	Colorado
United States	Novo Nordisk Investigational Site	Boston	Massachusetts
United States	Novo Nordisk Investigational Site	Charlotte	North Carolina
United States	Novo Nordisk Investigational Site	Chicago	Illinois
United States	Novo Nordisk Investigational Site	Cleveland	Ohio
United States	Novo Nordisk Investigational Site	Detroit	Michigan
United States	Novo Nordisk Investigational Site	Indianapolis	Indiana
United States	Novo Nordisk Investigational Site	Los Angeles	California
United States	Novo Nordisk Investigational Site	Minneapolis	Minnesota
United States	Novo Nordisk Investigational Site	Nashville	Tennessee
United States	Novo Nordisk Investigational Site	New Orleans	Louisiana
United States	Novo Nordisk Investigational Site	Oklahoma City	Oklahoma
United States	Novo Nordisk Investigational Site	Omaha	Nebraska
United States	Novo Nordisk Investigational Site	Orange	California
United States	Novo Nordisk Investigational Site	Philadelphia	Pennsylvania
United States	Novo Nordisk Investigational Site	Salt Lake City	Utah
United States	Novo Nordisk Investigational Site	San Diego	California
United States	Novo Nordisk Investigational Site	Seattle	Washington
United States	Novo Nordisk Investigational Site	Tampa	Florida
United States	Novo Nordisk Investigational Site	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cognition composite score according to the BAYLEY-III instrument	Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average	Day 1
Primary	Behavioural Symptoms Index according to Behavior Assessment System for Children - Third Edition (BASC-3) parent rating scale	Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average	Day 1
Primary	Adaptive behaviour domain score according to Adaptive Behavior Assessment System, Third Edition (ABAS-3) rating scale	Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average	Day 1
Primary	Global executive composite index according to Behavior Rating Inventory of Executive Function (BRIEF)	Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average	Day 1
Primary	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	Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average	Day 1