Clinical trial NCT06944522: A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease
Official title: exPDite-2: A Phase 3 Study to Assess the Efficacy and Safety of Midbrain Dopaminergic Neuronal Cell Therapy (Bemdaneprocel) for Participants With Parkinson's Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Parkinsons Disease (PD)
Keywords: exPDite-2
Cell Therapy
Cellular Therapy
Dopaminergic Neuronal Cell Therapy
Parkinsons Disease
Lead sponsor: BlueRock Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: Bayer
First posted: 2025-04-25
Enrollment: 102
Sex: ALL
Minimum age (years): 45
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: In the double-blind period, participants will be randomized at a 2:1 ratio to either receive bemdaneprocel or undergo sham surgery. Participants will receive immunosuppression or placebo equivalents for approximately 12 months. Approximately 18 months after the last participant is enrolled and based on the results of the primary analysis and review by the independent data monitoring committee (DMC), eligible participants who underwent sham surgery and remain actively enrolled in the study will have the opportunity to receive bemdaneprocel in the open-label period.
Primary purpose: TREATMENT

Brief summary: Study BRT-DA01-301 is a Phase 3 multicenter, randomized, sham surgery-controlled, double-blind study to assess efficacy and safety of bemdaneprocel in approximately 102 adults with Parkinson's Disease (PD).

Detailed description: The BRT-DA01-301 study is a Phase 3, multicenter, randomized, sham surgery-controlled, double-blind study involving approximately 102 participants with Parkinson's Disease (PD). Participants will be randomized in a 2:1 ratio to either receive bemdaneprocel or undergo sham surgery. The study includes an immunosuppression regimen and placebo equivalents to maintain blinding. The primary objective is to evaluate the efficacy of bemdaneprocel on motor symptoms in participants with PD. The secondary objective is to evaluate the effects of bemdaneprocel on Motor function, Quality of life, Non-motor symptoms of PD, Disease severity, and Use of PD medications or therapies compared with participants who undergo sham surgery. Participants will be followed for at least 18 months in the double-blind period and up to five years if they receive bemdaneprocel.

Eligibility criteria: Inclusion Criteria:

1. Individual of any sex ≥45 to ≤75 years of age at informed consent
2. Diagnosis of clinically established PD
3. ≥4 and \<12 years from time of PD diagnosis at informed consent
4. Must demonstrate responsiveness to levodopa therapy
5. Receiving medical therapy for the treatment of PD symptoms
6. ≥2.5 hours of daily OFF-time

Exclusion Criteria:

1. PD presenting with recurrent falls
2. Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis, or clinical features suggestive of a neurodegenerative disease other than PD, including multiple system atrophy, progressive supranuclear palsy, corticobasal degeneration, or Lewy body dementia
3. Any current or relevant previous history of serious, severe, or unstable physical, neurological, or psychiatric illness that may interfere with study participation, participant's safety, or assessment of endpoints per investigator's judgment
4. History of gene therapy or cell therapy
5. Prior surgical or radiation therapy to the brain, including deep brain stimulation (DBS) and lesion therapy, or prior history of intradural spinal cord surgery
6. Contraindication to surgery, general anesthesia, cell therapy, immunosuppression, or other required drugs
7. Current or previously active malignant disease within the past 5 years
8. Chronic immunosuppressive therapy
9. Receipt of another investigational therapy
10. Pregnancy or breastfeeding

Primary outcomes:
measure: Change from baseline in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day
timeFrame: From baseline to Week 78
Secondary outcomes:
measure: Change from baseline in PD diary measure of OFF-time, adjusted for a 16-hour waking day
timeFrame: From baseline to Week 78
measure: Change from baseline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score in the off-medication state. Part III score can range from 0 to 132, with lower scores being better.
timeFrame: From baseline to Week 78
measure: Change from baseline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II score. Part II score can range from 0 to 52, with lower scores being better.
timeFrame: From baseline to Week 78
measure: Change from baseline in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index. Summary index score can range from 0 to 100, with lower scores being better.
timeFrame: From baseline to Week 78
measure: Incidence and severity of treatment-emergent adverse events
timeFrame: From baseline to month 60
Other outcomes:
Locations:
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Farmington Hills
country: United States
facility: Quest Research Institute
state: Michigan
city: New York
country: United States
facility: New York Presbyterian/Weill Cornell Medical Center
state: New York
city: Providence
country: United States
facility: Rhode Island Hospital
state: Rhode Island
Study officials:
References: