Study 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
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
Primary
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
