A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease
- Conditions
- Parkinsons Disease (PD)
- Interventions
- Procedure: Sham surgery
- Registration Number
- NCT06944522
- Lead Sponsor
- BlueRock Therapeutics
- 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.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 102
- Individual of any sex ≥45 to ≤75 years of age at informed consent
- Diagnosis of clinically established PD
- ≥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
- 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 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
- Current or previously active malignant disease within the past 5 years
- Chronic immunosuppressive therapy
- Receipt of another investigational therapy
- Pregnancy or breastfeeding
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Group A bemdaneprocel Bemdaneprocel will be administered on Day 0 Group B Sham surgery Sham surgery will be performed on Day 0
- Primary Outcome Measures
Name Time Method 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
- Secondary Outcome Measures
Name Time Method Change from baseline in PD diary measure of OFF-time, adjusted for a 16-hour waking day From baseline to Week 78 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. From baseline to Week 78 Incidence and severity of treatment-emergent adverse events From baseline to month 60 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. From baseline to Week 78 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. From baseline to Week 78
Trial Locations
- Locations (7)
Parkinson's Disease and Movement Disorders Center of Boca Raton
🇺🇸Boca Raton, Florida, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Quest Research Institute
🇺🇸Farmington Hills, Michigan, United States
New York Presbyterian/Weill Cornell Medical Center
🇺🇸New York, New York, United States
Rhode Island Hospital
🇺🇸Providence, Rhode Island, United States