Parkinsons Disease (PD)
Conditions
Keywords
exPDite-2, Cell Therapy, Cellular Therapy, Dopaminergic Neuronal Cell Therapy, Parkinsons Disease
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.
Interventions
BIOLOGICALbemdaneprocel
Investigational cell therapy comprising midbrain dopaminergic neuron progenitors derived from human embryonic stem cells
PROCEDURESham surgery
Sham surgery will be performed on Day 0
Sponsors
BlueRock Therapeutics
Bayer
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Outcomes Assessor)
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.
Eligibility
Sex/Gender
ALL
Age
45 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 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 |
| 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 |
Countries
Australia, Canada, United States
Contacts
CONTACTPatient Engagement
Outcome results
None listed