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

Sponsor

Novo Nordisk A/S (Industry)

Overall Status

Completed

CT.gov ID

NCT03660774

Collaborator

(none)

563

Enrollment

Locations

12.5

Actual Duration (Months)

25.6

Patients Per Site

2.1

Patients Per Site Per Month

Study Details

Study Description

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	Phase
Haemophilia A Haemophilia B	Other: No treatment given

Study Design

Study Type:

Observational

Actual Enrollment :

563 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 :

Sep 27, 2018

Actual Primary Completion Date :

Oct 11, 2019

Actual Study Completion Date :

Oct 11, 2019

Arms and Interventions

Arm	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.

Arm

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.

Outcome Measures

Primary Outcome Measures

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
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
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

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 21 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novo Nordisk Investigational Site	Los Angeles	California	United States	90027
2	Novo Nordisk Investigational Site	Orange	California	United States	92868
3	Novo Nordisk Investigational Site	San Diego	California	United States	92123
4	Novo Nordisk Investigational Site	Aurora	Colorado	United States	80045
5	Novo Nordisk Investigational Site	Washington	District of Columbia	United States	20010-2978
6	Novo Nordisk Investigational Site	Tampa	Florida	United States	33607
7	Novo Nordisk Investigational Site	Atlanta	Georgia	United States	30322
8	Novo Nordisk Investigational Site	Chicago	Illinois	United States	60612
9	Novo Nordisk Investigational Site	Indianapolis	Indiana	United States	46260
10	Novo Nordisk Investigational Site	New Orleans	Louisiana	United States	70112
11	Novo Nordisk Investigational Site	Boston	Massachusetts	United States	02215
12	Novo Nordisk Investigational Site	Ann Arbor	Michigan	United States	48105
13	Novo Nordisk Investigational Site	Detroit	Michigan	United States	48201
14	Novo Nordisk Investigational Site	Minneapolis	Minnesota	United States	55404
15	Novo Nordisk Investigational Site	Omaha	Nebraska	United States	68198-6828
16	Novo Nordisk Investigational Site	Charlotte	North Carolina	United States	28204
17	Novo Nordisk Investigational Site	Cleveland	Ohio	United States	44106
18	Novo Nordisk Investigational Site	Oklahoma City	Oklahoma	United States	73104
19	Novo Nordisk Investigational Site	Philadelphia	Pennsylvania	United States	19134
20	Novo Nordisk Investigational Site	Nashville	Tennessee	United States	37212
21	Novo Nordisk Investigational Site	Salt Lake City	Utah	United States	84113
22	Novo Nordisk Investigational Site	Seattle	Washington	United States	98105

Sponsors and Collaborators

Novo Nordisk A/S

Investigators

Study Director: Clinical Reporting Anchor and Disclosure (1452), Novo Nordisk A/S

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Novo Nordisk A/S

ClinicalTrials.gov Identifier:

NCT03660774

Other Study ID Numbers:

HAEM-4436
U1111-1202-3415

First Posted:

Sep 7, 2018

Last Update Posted:

Nov 22, 2019

Last Verified:

Nov 1, 2019

Individual Participant Data (IPD) Sharing Statement:

Yes

Plan to Share IPD:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Hemophilia A

Hemophilia B

Study Results

No Results Posted as of Nov 22, 2019