A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease
Purpose
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).
Condition
- Parkinsons Disease (PD)
Eligibility
- Eligible Ages
- Between 45 Years and 75 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- 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
- Masking
- Triple (Participant, Care Provider, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Active Comparator Group A |
Bemdaneprocel will be administered on Day 0 |
|
|
Sham Comparator Group B |
Sham surgery will be performed on Day 0 |
|
Recruiting Locations
Kansas City, Kansas 66160-8500
More Details
- Status
- Recruiting
- Sponsor
- BlueRock Therapeutics
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.