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A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease
NCT06944522 · BlueRock Therapeutics
In plain English
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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
About this study
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:
* Diagnosis of clinically established PD as defined by the International Parkinson and Movement Disorders Society
* Individual of any sex ≥45 to ≤75 years of age at informed consent
* Robust and clear response to DA therapy as defined by MDS-UPDRS Part III
* ≥4 and \<12 years from time of PD diagnosis at informed consent
* Must demonstrate responsiveness to levodopa therapy
* Receiving medical therapy for the treatment of PD symptoms
* ≥2.5 hours of daily OFF-time
* Vaccinated per current national guidelines or local practice for patients with altered immunocompetence
Exclusion Criteria:
* PD presenting with recurrent falls
* 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
* 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
* History of gene therapy or cell therapy
* Prior treatment with intrajejunal or subcutaneous infusion therapies for PD
* Prior surgical or radiation therapy to the brain, including deep brain stimulation (DBS) and lesion therapy, or prior history of intradural spinal cord surgery
* Contraindication to surgery, general anesthesia, cell therapy, immunosuppression, or other required drugs, or anything that prevents use of PET or MRI
* Any active infection (including but not limited to HIV, HCV, HBV, CMV, syphilis, or tuberculosis) or condition that, in the opinion of the investigator could put the participant at significant risk from immunosuppression or impact the participant's ability to perform study assessments
* Current or previously active malignant disease within the past 5 years
* Chronic immunosuppressive therapy
* Receipt of another investigational therapy within 5 half-lives of the active treatment
* Pregnancy or breastfeeding
Study design
Enrollment target: 102 participants
Allocation: randomized
Masking: triple
Age groups: adult, older_adult
Timeline
Starts: 2025-06-17
Estimated completion: 2032-03
Last updated: 2026-04-01
Interventions
Biological: bemdaneprocelProcedure: Sham surgery
Primary outcomes
- • Change from baseline in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day (From baseline to Week 78)
Sponsor
BlueRock Therapeutics · industry
With: Bayer
Contacts & investigators
ContactPatient Engagement · contact · clinicaltrials@bluerocktx.com · 1-877-380-3967
All locations (37)
Banner Sun Health Research InstituteRecruiting
Sun City, Arizona, United States
University of Arkansas for Medical SciencesRecruiting
Little Rock, Arkansas, United States
UCLA NeuroTranslational Research CenterRecruiting
Los Angeles, California, United States
University of Colorado Hospital - Neurology ClinicRecruiting
Aurora, Colorado, United States
MedStar Georgetown University HospitalRecruiting
Washington D.C., District of Columbia, United States
University of Miami Health System - NeurologyRecruiting
Miami, Florida, United States
USF Parkinson's Disease and Movement Disorders CenterRecruiting
Tampa, Florida, United States
Parkinson's Disease and Movement Disorders Center at Northwestern Memorial HospitalRecruiting
Chicago, Illinois, United States
University of Kansas Medical CenterRecruiting
Kansas City, Kansas, United States
University of Louisville HospitalRecruiting
Louisville, Kentucky, United States
Johns Hopkins Hospital - NeurologyRecruiting
Baltimore, Maryland, United States
Tufts Medical Center - NeurologyRecruiting
Boston, Massachusetts, United States
Beth Israel Deaconess Medical CenterRecruiting
Boston, Massachusetts, United States
Michigan Clinical Research Unit - NeurologyRecruiting
Ann Arbor, Michigan, United States
University of Michigan Clinical Research Unit - NeurologyRecruiting
Ann Arbor, Michigan, United States
Quest Research InstituteRecruiting
Farmington Hills, Michigan, United States
UBMD NeurologyRecruiting
Buffalo, New York, United States
Mount Sinai West - NeurologyRecruiting
New York, New York, United States
New York Presbyterian/Weill Cornell Medical CenterRecruiting
New York, New York, United States
University of Toledo Medical CenterRecruiting
Toledo, Ohio, United States
OHSU Neurology Clinic, South WaterfrontRecruiting
Portland, Oregon, United States
Rhode Island Hospital - NeurologyRecruiting
Providence, Rhode Island, United States
Medical University of South Carolina, Movement Disorders Dept.Recruiting
Charleston, South Carolina, United States
Vanderbilt University Medical CenterRecruiting
Nashville, Tennessee, United States
UTHealth Houston Neurosciences - Texas Medical CenterRecruiting
Houston, Texas, United States
University of Vermont Medical CenterRecruiting
Burlington, Vermont, United States
Virginia Commonwealth UniversityRecruiting
Richmond, Virginia, United States
EvergreenHealth - Research DepartmentRecruiting
Kirkland, Washington, United States
NeuRA (Neuroscience Research Australia)Recruiting
Randwick, New South Wales, Australia
Gold Coast Hospital & Health ServiceRecruiting
Southport, Queensland, Australia
Princess Alexandra HospitalRecruiting
Woolloongabba, Queensland, Australia
Monash Medical CentreRecruiting
Clayton, Victoria, Australia
The Alfred HospitalRecruiting
Melbourne, Victoria, Australia
The Royal Melbourne HospitalRecruiting
Parkville, Victoria, Australia
The University of British ColumbiaRecruiting
Vancouver, British Columbia, Canada
University Health Network - Toronto Western HospitalRecruiting
Toronto, Ontario, Canada
CHUM - Centre hospitalier de l'Université de MontréalRecruiting
Montreal, Quebec, Canada