A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease (exPDite-2)

BlueRock Therapeutics

Status and phase: Not yet enrolling; Phase 3

Conditions: Parkinsons Disease (PD)

Treatments: Procedure: Sham surgery; Biological: bemdaneprocel

Study type: Interventional

Funder types: Industry

Identifiers

NCT06944522

BRT-DA01-301

Details and patient eligibility

About

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).

Full 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.

Enrollment: 102 estimated patients

Sex: All

Ages: 45 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. 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

1. 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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102 participants in 2 patient groups

Group A

Active Comparator group

Description:

Bemdaneprocel will be administered on Day 0

Treatment:

Biological: bemdaneprocel

Group B

Sham Comparator group

Description:

Sham surgery will be performed on Day 0

Treatment:

Procedure: Sham surgery