Haemophilia AeTHINKNCT03660774
A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia
An observational study of Haemophilia A and Haemophilia B, run by Novo Nordisk A/S at 22 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 21 years, men only
- Conditions the study is for
- Haemophilia A, Haemophilia B
- What is being tested?
- No treatment given
- How many people
- 563 participants
- Where
- 22 sitesUnited States
- Study dates
- 27 Sep 2018to 11 Oct 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 21 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Children and young adults with hemophilia A or B, all severities
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
Trial locations and contacts
22 sites in 1 country.
22 sites in 1 country
United States22 sites
Novo Nordisk Investigational Site
Ann Arbor, Michigan, 48105
Novo Nordisk Investigational Site
Atlanta, Georgia, 30322
Novo Nordisk Investigational Site
Aurora, Colorado, 80045
Novo Nordisk Investigational Site
Boston, Massachusetts, 02215
Novo Nordisk Investigational Site
Charlotte, North Carolina, 28204
Novo Nordisk Investigational Site
Chicago, Illinois, 60612
Novo Nordisk Investigational Site
Cleveland, Ohio, 44106
Novo Nordisk Investigational Site
Detroit, Michigan, 48201
Novo Nordisk Investigational Site
Indianapolis, Indiana, 46260
Novo Nordisk Investigational Site
Los Angeles, California, 90027
Novo Nordisk Investigational Site
Minneapolis, Minnesota, 55404
Novo Nordisk Investigational Site
Nashville, Tennessee, 37212
Novo Nordisk Investigational Site
New Orleans, Louisiana, 70112
Novo Nordisk Investigational Site
Oklahoma City, Oklahoma, 73104
Novo Nordisk Investigational Site
Omaha, Nebraska, 68198-6828
Novo Nordisk Investigational Site
Orange, California, 92868
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, 19134
Novo Nordisk Investigational Site
Salt Lake City, Utah, 84113
Novo Nordisk Investigational Site
San Diego, California, 92123
Novo Nordisk Investigational Site
Seattle, Washington, 98105
Novo Nordisk Investigational Site
Tampa, Florida, 33607
Novo Nordisk Investigational Site
Washington D.C., District of Columbia, 20010-2978
About this study
Official title: Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence and Other Cognitive Functions (eTHINK)
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
- Conditions
- Haemophilia A, Haemophilia B
- Being tested
- No treatment given
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: No treatment given |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Sep 2018
- Study start27 Sep 2018
- Primary completion11 Oct 2019
- Study completion11 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Haemophilia AStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Ecologic or community
- Time perspective
- Cross-sectional
- Enrolment
- 563 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Reporting Anchor and Disclosure (1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03660774
- Sponsor's protocol ID
- HAEM-4436
- Other IDs
- U1111-1202-3415
- Acronym
- eTHINK
- Status last verified
- Nov 2019
- First submitted
- 21 Aug 2018
- Last updated
- 22 Nov 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Publications
Mrakotsky C, Walsh KS, Buranahirun Burns C, Croteau SE, Markert A, Geybels M, Hannemann C, Rajpurkar M, Shapiro KA, Wilkening GN, Ventola P, Cooper DL; eTHINK Study Group. The eTHINK Study: Cognitive and Behavioral Outcomes in Children with Hemophilia. J Pediatr. 2024 Sep;272:114089. doi: 10.1016/j.jpeds.2024.114089. Epub 2024 May 9.
PubMed 38734133Buranahirun 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.
PubMed 31595670
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence and Other Cognitive Functions (eTHINK)
- ClinicalTrials.gov ID
- NCT03660774
- Acronym / sponsor ID
- eTHINK / HAEM-4436
- Conditions
- Haemophilia A, Haemophilia B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 563 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 27 Sep 2018 (actual)
- Primary completion
- 11 Oct 2019 (actual)
- Study completion
- 11 Oct 2019 (actual)
- First posted
- 7 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Observational model
- Ecologic or community
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with hemophilia | – | Other: No treatment givenChildren 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. |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children and young adults with hemophilia A or B, all severities
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No treatment givenThe 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. | Other | Patients with hemophilia |
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2019.
- Derived from this study: PubMed 38734133Mrakotsky C, Walsh KS, Buranahirun Burns C, Croteau SE, Markert A, Geybels M, Hannemann C, Rajpurkar M, Shapiro KA, Wilkening GN, Ventola P, Cooper DL; eTHINK Study Group. The eTHINK Study: Cognitive and Behavioral Outcomes in Children with Hemophilia. J Pediatr. 2024 Sep;272:114089. doi: 10.1016/j.jpeds.2024.114089. Epub 2024 May 9.
- Derived from this study: PubMed 31595670Buranahirun 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.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Reporting Anchor and Disclosure (1452) study director
Novo Nordisk A/S
22 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haemophilia A and Haemophilia B, run by Novo Nordisk A/S at 22 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cognition composite score according to the BAYLEY-III instrument (time frame: Day 1). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03660774, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2019.