Abstract
Objective
- To describe the design of the Phase 3
exPDite-2trial (NCT06944522), a multicenter, multisite, randomized, sham surgery-controlled, double-blind trial to evaluate the efficacy and safety of bemdaneprocel in people with PD.
Methods
exPDite-2will enroll participants (US, Canada, and Australia) with PD aged 45 to 75 years with motor symptoms inadequately controlled by standard treatments.- Bemdaneprocel will be administered stereotactically into the posterior putamen bilaterally through a single burr hole on each side in a single session (Figure 1).
- A partial burr hole that does not penetrate the inner table of the skull will be created over each hemisphere.
- Basiliximab (20 mg, intravenous [IV]) intraoperatively and on postoperative day 4.
- Methylprednisolone (500 mg, IV) perioperatively followed by prednisone taper to 5 mg, oral administration (PO) once daily (QD) and continued for 1 year.
- Tacrolimus (PO) initiated on postoperative day 1 and adjusted to a target trough blood level of 4 to 7 ng/mL twice daily (BID) for 1 year.
Assessments
- To evaluate the efficacy of bemdaneprocel on motor symptoms in participants with PD compared with participants who undergo sham surgery | Change from baseline to Week 78 in PD diary measure of ON time without troublesome dyskinesiaa-c
- To evaluate the effects of bemdaneprocel on motor function, quality of life, non-motor symptoms, disease severity, and use of PD medications or therapies | Key Secondary: Change from baseline to Week 78: PD diary OFF time; MDS-UPDRS Part III OFF score; MDS-UPDRS Part II score; PDQ-39 summary index
- To evaluate the safety of bemdaneprocel | Incidence and severity of TEAEs; AEs related to cellular drug product; AEs related to surgical procedure; AEs related to immunosuppression regimen; change from baseline over time in UDysRS score and C-SSRS score
- A total sample size of
N=102randomized participants will provide 90% power (2-sidedalpha=0.05) to detect a difference of 1.85 hours between bemdaneprocel and sham surgery on the primary endpoint of ON time without troublesome dyskinesia.
Conclusions
- Bemdaneprocel demonstrated a favorable safety profile and maintained improvements or stability across clinical assessments through 3 years, supporting the ongoing development of bemdaneprocel for the potential treatment of PD.5
- The
exPDite-2trial is a Phase 3, randomized, sham surgery-controlled, double-blind trial that will evaluate the efficacy and safety of bemdaneprocel on motor symptoms and safety in participants with Parkinson’s disease. exPDite-2will be the first large-scale, potentially registrational trial of stem cell-derived midbrain dopaminergic neuron progenitors in people with Parkinson’s disease.- The first participant was randomized and underwent surgery in August 2025.
View references
- Kim TW, et al. Cell Stem Cell. 2021;28(2):343-55.e5.
- Piao J, et al. Cell Stem Cell. 2021;28(2):217-29.e7.
- Kriks S, et al. Nature. 2011;480(7378):547-551.
- Henchcliffe C, et al. Presented at: International Congress of Parkinson’s Disease and Movement Disorders; August 27-31, 2023; Copenhagen, Denmark.
- Sarva H, et al. Presented at: Annual Meeting of the International Congress of Parkinson’s Disease and Movement Disorders; October 5-9, 2025; Honolulu, HI.
