Clinical Trial Protocol: TRANSCEND 1 – an Observational Study of Patients with Moderate Parkinson’s Disease (P2-11.002)

Objective: An observational study ahead of the proof-of-concept transplant trial may reduce some of the confounding factors associated with investigating therapies in Parkinson’s disease. TRANSCEND 1 will follow a cohort of patients with Parkinson’s disease receiving a local standard of care (SoC) to monitor fluctuations in individual performance and disease characteristics over time and to facilitate a reliable baseline of disease severity before a subsequent proof-of-concept trial with a novel stem-cell based treatment in some patients from this cohort. Background: Novo Nordisk is developing a cell therapy for Parkinson’s disease in collaboration with groups at Lund University (Sweden) and the University of Cambridge (UK). The therapy comprises dopaminergic progenitor cells derived from human embryonic stem cells; the clinical development programme currently includes this observational study (TRANSCEND 1) and a planned phase 1/2, proof-of-concept transplant trial (TRANSCEND 2). Design/Methods: Patients (n=96) with moderate Parkinson’s disease (50–68 years old), with fluctuating disease and a disease duration of >5 years, will be followed for up to 24 months. Patients will be examined every 3 months using standard motor (MDS-UPDRS III), cognitive (MoCA), psychiatric (MADRS) and other clinical measures. Patients will receive SoC which may lead to changes in their medication regimen. However, the participants are required to have ≥2 rounds of assessments and 3 months of observation without medication adjustments prior to being invited to be screened for participation in the proof-of-concept transplant trial. Results: The study will start recruiting in February 2023. Conclusions: Results are expected in 2025. Disclosure: Prof. Gesine has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NovoNordisk. Prof. Gesine has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for NovoNordisk. The institution of Prof. Gesine has received research support from Swedish Research Council. The institution of Prof. Widner has received research support from Academic Learing Fund, ALF. The institution of Prof. Widner has received research support from Horizon 2020, European Union. Prof. Widner has a non-compensated relationship as a Co-chair/Chair with Swedish Movement Disorders Society that is relevant to AAN interests or activities. Dr. Bergholdt has received personal compensation for serving as an employee of Novo Nordisk A/S. Dr. Bergholdt has stock in Novo Nordisk A/S. Ms. Frimer-Larsen has received personal compensation for serving as an employee of Novo Nordisk A/S. Ms. Frimer-Larsen has stock in Novo Nordisk A/S. Ms. Mathiasen has received personal compensation for serving as an employee of Novo Nordisk A/S. Ms. Mathiasen has stock in Novo Nordisk A/S. Dr. Nielsen has received personal compensation for serving as an employee of Novo Nordisk. Dr. Nielsen has stock in Novo Nordisk. Roger Barker, PhD has nothing to disclose.