Pediatrics & Rare Disease

Cognitive and behavioral endpoint selection and management for clinical trials.

In rare neurodevelopmental disorders, detecting even the smallest change in behavior or function is critical, and often challenging.

Adopting strategic trial designs and targeted assessment selection or development improves your ability to make informed decisions about your drug development efforts. Cogstate has a group of neuroscience experts specifically with rare disease training that you can leverage to improve signal detection, increase trial efficiencies, and be more confident about your data. Learn how our data quality programs and digital cognitive tests can assist your team.

Talk with our team about your study

Improve Strategic Study Design and Execution

Rare diseases are characterized by a wide range of severity, clinical presentation, and rate of progression, making strategic study design and execution important for yielding conclusive trial results. In particular, selecting the appropriate scales is vital and their correct administration throughout the trial is paramount.

Cogstate’s science team supports all stages of trial design and execution, from protocol development to interactions with regulatory agencies.

We help you evaluate appropriate assessments for the functioning level of the population and those with sensitivity and specificity to detect change over time.

We collaborate to develop new endpoints that match the concerns of your patient populations and better support drug development decisions.

We support the development of accurate Statistical Analysis Plans to appropriately interpret often-complex results.

Optimize Clinical Outcomes for Greater Signal Detection

Cogstate’s team of scientific and operational experts can help advise your team on efficient and effective study conduct in pediatric and rare disease trials. Working with our team gives you access to robust data quality programs and reliable computerized assessments.

Implement Robust Data Quality Programs
In rare disease trials, often the assessments are complex and eligible participants limited. With numerous and geographically dispersed trial sites, inter-rater variability can compromise data quality. Not to mention, many sites are highly specialized, and raters are participating in several similar trials simultaneously. As such, high-quality rater training and data monitoring programs are essential. Cogstate supports all aspects of data quality programs with proven methodologies and leading technologies that drive endpoint reliability.
Leverage Sensitive and Reliable Computerized Batteries
Cogstate’s computerized batteries of highly sensitive tests provide rapid and reliable measurement of distinct cognitive domains affected by rare diseases. The tests are grouped into customizable batteries for a complete and nuanced picture of a subject’s cognitive state. Cogstate tests are culture- and education-neutral and designed for repeated administration with minimal practice or learning effects, making them ideal for use in global clinical trials. Cogstate tests are available in pediatric versions.

Extensive Experience with Assessments Commonly Used in Rare Disease Trials

  • Aberrant Behavior Checklist (ABC)
  • Autism Diagnostic Observational Schedule (ADOS)
  • Bayley Scales of Infant Development, Third Edition (Bayley-III)
  • Behavior Assessment System for Children (BASC-3)
  • Behavioral Rating Inventory of Executive Function Scales (BRIEF)
  • California Verbal Learning Test (CVLT)
  • Clinical Global Impressions Scale (CGI)
  • Columbia Suicide Severity Rating Scale (C-SSRS)
  • Mullen Scales of Early Learning (Mullen)
  • Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)
  • Stanford-Binet Intelligence Scales, Fifth Edition (SB-5)
  • Vineland Adaptive Behavior Scales (VABS-II/VABS-3)
  • Weschler Scales (WPPSI-IV and WASI-II)
  • And dozens more

Indications we’ve supported include:

  • Angelman’s Syndrome
  • Autism Spectrum Disorder
  • CDKL5 Deficiency Disorder
  • Down Syndrome
  • Epilepsy
  • Epileptic Encephalopathies
  • Fragile X Syndrome
  • Hemophilia
  • Kabuki Syndrome
  • Mucopolysaccharidosis (MPS) Disorders
  • Rett Syndrome
  • Sickle Cell Disease
  • Wide range of additional rare genetic disorders

Rare Disease and Pediatric Center for Excellence

With the vital need for effective cognitive measurement in rare disease and pediatric clinical trials, Cogstate has established a Rare Disease and Pediatric Center of Excellence. The Center provides industry-leading clinical and scientific expertise focused on the selection, adaptation, optimization, validation and administration of cognitive and behavioral measures in clinical studies in rare diseases and pediatrics.

Pamela VentolaPam Ventola, PhD
VP Science, Associate Professor at the Yale Child Study Center

Dr. Ventola leads Cogstate’s Rare Disease and Pediatric Center for Excellence, providing Cogstate customers with strategic oversight and expert guidance throughout all stages of their study planning and execution – from endpoint selection, rater training and strategic monitoring to final statistical analysis. As a licensed clinical psychologist, Dr. Ventola’s primary clinical expertise is in pediatric neuropsychology and specifically developmental and genetic disorders. Dr. Ventola received her Doctor of Philosophy in Clinical Psychology from University of Connecticut and completed her clinical training and Postdoctoral Fellowship at the Yale University School of Medicine, Child Study Center.

Decentralized Clinical Trials

We help bring the trial to the patient

Traveling to-and-from testing sites can be costly and particularly burdensome for families and children with rare disease, prohibiting or limiting many from participation in clinical trials. More and more teams are realizing the potential of decentralization practices to enhance data quality and improve patient experience. Cogstate digital endpoints are enabling at-home, self-administered assessment and our remote rating services are enabling at-home clinician-administered assessment to drive quality outcomes, engagement, and retention.


Solutions for Every Stage of Your Study Development

Cogstate’s clinical trial services, coupled with innovative operational approaches, advanced analytics, and scientific consulting, help research teams draw conclusions from study data faster and with more accuracy. Learn how we can support your study:

Scientific Consulting

Digital Cognitive Assessment

Rater Training

Central Monitoring

Scale Management

Remote / At-home Testing

How can we help you optimize the measurement of brain health?

Contact Our Team