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NCT Number: NCT03660774

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

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

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Key information

Conditions

Haemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Haemophilia B Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemophilia B Hemorrhagic Disorders

Age range

1 year–21 year

Sex eligibility

Male

Study type

Observational

Primary location

Novo Nordisk Investigational Site, Los Angeles, California, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

No treatment given

Other

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.

Primary outcomes

  1. Cognition composite score according to the BAYLEY-III instrument

    Time frame: Day 1

    Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average

  2. Behavioural Symptoms Index according to Behavior Assessment System for Children - Third Edition (BASC-3) parent rating scale

    Time frame: Day 1

    Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average

  3. Adaptive behaviour domain score according to Adaptive Behavior Assessment System, Third Edition (ABAS-3) rating scale

    Time frame: Day 1

    Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average

  4. Global executive composite index according to Behavior Rating Inventory of Executive Function (BRIEF)

    Time frame: Day 1

    Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average

  5. 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

    Time frame: Day 1

    Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study title

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

Acronym: eTHINK

Important dates

Study start
2018
Primary completion
2019
Study completion
2019
First posted
Sep 7, 2018
Registry last updated
Nov 22, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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