Supporting Clinical Research in Rare & Orphan Disorders
Advancing Scientific Innovation in Rare & Orphan Disorders
With expertise in pediatric rare and orphan diseases, VeraSci supports clinical trials from Rett syndrome (RTT), Arginase deficiency disorder, Myasthenia Gravis, Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) to Mucopolysaccharidoses disorders (MPS). Genetic diseases, such as Mucopolysaccharidoses disorders, result in neurodegeneration and affect multiple body systems over the lifespan. These diseases require a targeted treatment strategy that focuses on symptomatic relief as well as the remittance of disease progression. Fortunately, there are several emerging treatments in various stages of clinical development.
Achieving therapeutic success in the management of these diseases requires identifying the right target, using the ideal treatment, administering the treatment at the right dose at the right time, and employing clinical endpoints that are reliable, robust, and fit for the purpose.
We bring extensive experience in clinical trial design, execution of reliable global studies including linguistics, and scientific expertise in clinical, behavioral, and cognitive measurement across a broad range of rare and orphan diseases of varying severities to ensure your clinical trial is positioned for success. VeraSci can even supply a qualified centralized assessor to sites that don’t have a qualified rater available. The assessor travels to sites, provides expert clinical and cognitive ratings, fills out required paperwork, and can enter data into the study EDC when needed.
Dependable Solutions for Global Clinical Trials
VeraSci is not just there in the beginning; we take care of your study objectives and data throughout the entire trial. As your partner, we leverage experience gained through hundreds of trials and ensure the highest quality data, all while anticipating any issues that can derail or delay your study before they arise.
We provide expert solutions in:
- Endpoint selection, trial design, and feasibility
- In-house language translation and cultural adaptation
- Alignment, calibration, and standardization of raters across sites
- Regulatory and protocol guidance
- Identifying Value Endpoints to demonstrate the pharmaeconomic value of novel therapies earlier in the development process
- Statistical considerations for small patient populations
- Expert data review & on-demand reporting through VeraSci’s Signal Analytics
- Pathway eCOA solution with clinical, functional, motor and cognitive scales
- Audio and video surveillance
Select Endpoints & Assessments for Rare & Orphan Disorders
Clinical
ABAS-III- Adaptive Behavior Assessment System: 3rd Edition
VABS-II- Vineland Adaptive Behavior Scale: 2nd Edition
VABS-III- Vineland Adaptive Behavior Scale: 3rd Edition
CBCL- Child Behavior Checklist
HINE – Hammersmith Infant Neurological Examination
Functional
ALSFRS-R- ALS Functional Rating Scale-Revised
PedsQL- Pediatric Quality of Life Inventory
PROMIS- Patient-Reported Outcomes Measurement Information System
SF-36- 36-Item Short-Form Health Survey
Motoric
PPT- Purdue Pegboard Test
NSAA- North Star Ambulatory Assessment
PDMS-2 – Peabody Developmental Motor Scales
Cognitive
BSID-III- Bayley Scales of Infant and Toddler Development: 3rd Edition
WASI-II- Wechsler Abbreviated Scale of Intelligence: 2nd Edition
WAIS-IV- Wechsler Adult Intelligence Scale: 4th Edition
WISC-V- Wechsler Intelligence Scale for Children: 5th Edition
WPPSI-IV- Wechsler Preschool and Primary Scale of Intelligence: 4th Edition
HVLT-R- Hopkins Verbal Learning Test: Revised
KABC-II- Kaufman Assessment Battery for Children: 2nd Edition
MSEL- Mullen Scales for Early Learning
RAVLT- Rey Auditory Verbal Learning Test
TOVA- Test of Variables of Attention
VP of Scientific Operations
VP, Scientific Development
Senior Director, Psychological Assessments
Clinical Scientist
Our Recent Experience Includes:
Amyotrophic Lateral Sclerosis (ALS)
Arginase 1 Deficiency
Duchenne Muscular Dystrophy (DMD)
GM1 Gangliosidosis
Guillain-Barré Syndrome
MPS I (Hurler Syndrome)
MPS II (Hunter Syndrome)
MPS III (Sanfilippo Syndrome A)
Rett Syndrome
Spinocerebellar Ataxia
Wilson’s Disease
Common Developmental and Cognitive Assessments in Pediatric Rare Disease Clinical Trials
Download this article to learn about the most commonly used assessments in pediatric rare disease trials.