A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease

NCT06944522 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: BRT-DA01-301
• Study Type: interventional
• Target: 102
• Locations: 3 countries
• Sites: 39

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).

Conditions

Parkinsons Disease (PD)

Keywords

exPDite-2; Cell Therapy; Cellular Therapy; Dopaminergic Neuronal Cell Therapy; Parkinsons Disease

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• 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

Study Arms (2)

Group A

ACTIVE COMPARATOR

Bemdaneprocel will be administered on Day 0

Biological: bemdaneprocel

Group B

SHAM COMPARATOR

Sham surgery will be performed on Day 0

Procedure: Sham surgery

Interventions

bemdaneprocel BIOLOGICAL

Investigational cell therapy comprising midbrain dopaminergic neuron progenitors derived from human embryonic stem cells

Also known as: MSK-DA01, BRT-DA01

Group A

Sham surgery PROCEDURE

Sham surgery will be performed on Day 0

Group B

Eligibility Criteria

• Age: 45 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• BlueRock Therapeutics: lead
• Bayer: collaborator

Study Sites (39)

Banner Sun Health Research Institute

Sun City, Arizona, 85351, United States

RECRUITING

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

RECRUITING

UCLA NeuroTranslational Research Center

Los Angeles, California, 90095, United States

RECRUITING

UCI Medical Center - Neurology

Orange, California, 92868, United States

RECRUITING

University of Colorado Hospital - Neurology Clinic

Aurora, Colorado, 80045, United States

RECRUITING

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

RECRUITING

University of Miami Health System - Neurology

Miami, Florida, 33136, United States

RECRUITING

USF Parkinson's Disease and Movement Disorders Center

Tampa, Florida, 33613, United States

RECRUITING

Parkinson's Disease and Movement Disorders Center at Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160-8500, United States

RECRUITING

University of Louisville Hospital

Louisville, Kentucky, 40202, United States

RECRUITING

Johns Hopkins Hospital - Neurology

Baltimore, Maryland, 21287, United States

RECRUITING

Tufts Medical Center - Neurology

Boston, Massachusetts, 02111, United States

RECRUITING

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

RECRUITING

Michigan Clinical Research Unit - Neurology

Ann Arbor, Michigan, 48109, United States

RECRUITING

University of Michigan Clinical Research Unit - Neurology

Ann Arbor, Michigan, 48109, United States

RECRUITING

Quest Research Institute

Farmington Hills, Michigan, 48334, United States

RECRUITING

UBMD Neurology

Buffalo, New York, 14221, United States

RECRUITING

Mount Sinai West - Neurology

New York, New York, 10019, United States

RECRUITING

New York Presbyterian/Weill Cornell Medical Center

New York, New York, 10065, United States

RECRUITING

Duke Neurological Disorders Clinic

Durham, North Carolina, 27710, United States

RECRUITING

University of Toledo Medical Center

Toledo, Ohio, 43614, United States

RECRUITING

OHSU Neurology Clinic, South Waterfront

Portland, Oregon, 97239, United States

RECRUITING

Rhode Island Hospital - Neurology

Providence, Rhode Island, 02903, United States

RECRUITING

Medical University of South Carolina, Movement Disorders Dept.

Charleston, South Carolina, 29425, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

UTHealth Houston Neurosciences - Texas Medical Center

Houston, Texas, 77030, United States

RECRUITING

University of Vermont Medical Center

Burlington, Vermont, 05401, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23284, United States

RECRUITING

EvergreenHealth - Research Department

Kirkland, Washington, 98034, United States

RECRUITING

NeuRA (Neuroscience Research Australia)

Randwick, New South Wales, 2031, Australia

RECRUITING

Gold Coast Hospital & Health Service

Southport, Queensland, 4215, Australia

RECRUITING

Princess Alexandra Hospital

Woolloongabba, Queensland, 4102, Australia

RECRUITING

Monash Medical Centre

Clayton, Victoria, 3168, Australia

RECRUITING

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

RECRUITING

The Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

RECRUITING

The University of British Columbia

Vancouver, British Columbia, V6T 1Z3, Canada

RECRUITING

University Health Network - Toronto Western Hospital

Toronto, Ontario, M5T 2S8, Canada

RECRUITING

CHUM - Centre hospitalier de l'Université de Montréal

Montreal, Quebec, H2X 0C1, Canada

RECRUITING

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Central Study Contacts

Patient Engagement

CONTACT

1-877-380-3967; clinicaltrials@bluerocktx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: April 7, 2025
• First Posted: April 25, 2025
• Study Start: June 17, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2032
• Last Updated: May 1, 2026

Record last verified: 2026-04

Locations

US (30); CA (3); AU (6)