The Clinical Neuroimaging Unit at CHDI provides a knowledge base on the status of imaging biomarker research in HD, focusing on PET and MRI. The group has broad expertise in first-in-human validation of new radioligands, including those targeting mutant huntingtin, development of structural and functional MRI imaging measures that provide markers of disease progression, and clinical and translational research studies that support biomarker development to measure response to intervention.

The neuroimaging unit provides recommendations for observational or interventional studies, including:

• First-in-human validation of new radioligands, dosimetry and kinetic analysis techniques; research studies with established tracers in HD
• Imaging data and software requirements when adopting the HD-ISS staging system (caudate volume and putamen volume are key markers of transition from Stage 0 to Stage 1)
• Selection of optimal imaging methods and endpoints for interventional studies based on statistical power, type, and objectives of the study
• Software tools and analytical approaches for image data processing, quality control and biomarker extraction
• Conceptual and technical knowledge for imaging data federation and sharing

The Clinical Wet Biomarkers Research Unit at CHDI is available for scientific consultation.  This Unit was established to demonstrate how wet biomarkers revealed by preclinical models or other neurodegenerative disorders can be utilized in the context of Huntington disease assessment or treatment.

The Wet Biomarkers team can provide expert advice in clinical biomarker assay development and suggest strategies for progressing translational biomarkers from preclinical to clinical applications. Their expertise covers a range of types of biomarkers including nucleic acids, metabolites and proteins and a range of matrices, including blood, cerebral spinal fluid and others. This team can also provide recommendations for biosample collection including methodologies and protocols for collection, storage and quality control. 

The Statistics and Modeling Unit at CHDI is available for scientific consultation. This division is responsible for conceptualizing disease progression models that will assist in characterizing the Huntington’s disease phenotype, especially with respect to clinical trial design for therapeutic studies. Some examples of such analysis include development of CAP score, PIN score, calculating minimally clinically important difference for various clinical assessments, and analysis of comorbidities in the Enroll-HD dataset.

The Statistics and Modeling team can provide expert advice to sponsors on sample size calculations, enrichment strategies for study cohorts, correlating imaging and clinical assessments for study populations, as well suitability of clinical endpoints for the clinical trial. The team can also assist sponsors with specific data analysis pertaining to their study design.

The Clinical Outcomes Unit at CHDI is available for scientific consultation. This Unit collaborates with academics, industry, and regulatory agencies to develop assessments that measure the impact of disease on people with Huntington’s disease and their families. Their aim is to address unmet needs across multiple domains using a range of assessment methods and modalities including patient reported outcomes, clinician reported outcomes, observer reported outcomes, and performance-based measures. Their goal is to provide reliable and valid assessments to the HD community to investigate and understand disease pathophysiology and the effects of interventions on disease progression.

Areas of expertise within the Clinical Outcomes Unit include HD assessments of function, cognition, motor, and behavior; digital measures; health economics and outcomes research (HEOR); and insight/anosognosia. 

Sponsors may consult with highly experienced HD clinicians and researchers through all stages of protocol development.

The CHDI clinical group can provide advice on the optimal participant population, clinical and biomarker outcomes during protocol development, and the Enroll-HD Clinical Trial Committee (CTC) provides advisory review of more advanced protocols. The CTC protocol review committee is comprised of experts in the HD field with clinical and research backgrounds in neurology, movement disorders, and neurosurgery.