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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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03660774
Recruitment Status : Completed
First Posted : September 7, 2018
Last Update Posted : November 22, 2019
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Tracking Information
First Submitted Date August 21, 2018
First Posted Date September 7, 2018
Last Update Posted Date November 22, 2019
Actual Study Start Date September 27, 2018
Actual Primary Completion Date October 11, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 6, 2018)
  • 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
  • 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
  • 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
  • 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
  • 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
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title 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)
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 Provided
Study Type Observational
Study Design Observational Model: Ecologic or Community
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Children and young adults with hemophilia A or B, all severities
Condition
  • Haemophilia A
  • Haemophilia 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.
Study Groups/Cohorts 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.
Intervention: Other: No treatment given
Publications * 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. Review.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: November 12, 2019)
563
Original Estimated Enrollment
 (submitted: September 6, 2018)
600
Actual Study Completion Date October 11, 2019
Actual Primary Completion Date October 11, 2019   (Final data collection date for primary outcome measure)
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
Sex/Gender
Sexes Eligible for Study: Male
Ages 1 Year to 21 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03660774
Other Study ID Numbers HAEM-4436
U1111-1202-3415 ( Other Identifier: World Health Organization (WHO) )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Device Product: No
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
Responsible Party Novo Nordisk A/S
Study Sponsor Novo Nordisk A/S
Collaborators Not Provided
Investigators
Study Director: Clinical Reporting Anchor and Disclosure (1452) Novo Nordisk A/S
PRS Account Novo Nordisk A/S
Verification Date November 2019