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

Completed

• Conditions: Haemophilia B; Haemophilia A
• Interventions: Other: No treatment given

• Registration Number: NCT03660774
• Lead Sponsor: Novo Nordisk A/S

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

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2018-08-21
• Study First Submitted QC: 2018-09-06
• Study First Posted: 2018-09-07
• Study Start: 2018-09-27
• Primary Completion: 2019-10-11
• Study Completion: 2019-10-11
• Status Verified: 2019-11
• Last Update Submitted: 2019-11-21
• Last Update Posted: 2019-11-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 563

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

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with hemophilia	No treatment given	Children and young adults with hemophilia A or B, all severities, treated in the hemophilia treatment center (comprehensive care) setting, including participation of caregivers/parents completing questionnaires designed to evaluate neurologic, neurocognitive and neurobehavioral function and development.

Primary Outcome Measures

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

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇺🇸 Seattle, Washington, United States