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

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ClinicalTrials.gov Identifier: NCT03660774
Recruitment Status : Enrolling by invitation
First Posted : September 7, 2018
Last Update Posted : April 25, 2019
Sponsor:
Information provided by (Responsible Party):
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

Condition or disease Intervention/treatment
Haemophilia A Haemophilia B Other: No treatment given

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Study Type : Observational
Estimated Enrollment : 600 participants
Observational Model: Ecologic or Community
Time Perspective: Cross-Sectional
Official Title: Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence and Other Cognitive Functions (eTHINK)
Actual Study Start Date : September 27, 2018
Estimated Primary Completion Date : July 31, 2019
Estimated Study Completion Date : July 31, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Group/Cohort Intervention/treatment
Patients with hemophilia
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.
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.




Primary Outcome Measures :
  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



Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 21 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Children and young adults with hemophilia A or B, all severities
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03660774


  Show 21 Study Locations
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
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Study Director: Clinical Reporting Anchor and Disclosure (1452) Novo Nordisk A/S

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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT03660774     History of Changes
Other Study ID Numbers: HAEM-4436
U1111-1202-3415 ( Other Identifier: World Health Organization (WHO) )
First Posted: September 7, 2018    Key Record Dates
Last Update Posted: April 25, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
URL: http://novonordisk-trials.com

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Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked