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

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

563

participants targeted

Target at P75+ for all trials

Timeline

Completed

Started Sep 2018

Geographic Reach

1 country

22 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

1 year study duration

First Submitted

Initial submission to the registry

August 21, 2018

Completed

17 days until next milestone

First Posted

Study publicly available on registry

September 7, 2018

Completed

20 days until next milestone

Study Start

First participant enrolled

September 27, 2018

Completed

1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 11, 2019

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 11, 2019

Completed

Last Updated

November 22, 2019

Status Verified

November 1, 2019

Enrollment Period

1 year

First QC Date

August 21, 2018

Last Update Submit

November 21, 2019

Conditions

Haemophilia A Haemophilia B

Outcome Measures

Primary Outcomes (5)

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

Study Arms (1)

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

Interventions

No treatment givenOTHER

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.

Patients with hemophilia

Eligibility Criteria

Age1 Year - 21 Years

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

Sampling MethodNon-Probability Sample

Study Population

Children and young adults with hemophilia A or B, all severities

Contact the study team to confirm eligibility.