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NCT06944522PHASE3Recruiting

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

BlueRock Therapeutics

Start Date

6/17/2025

Completion Date

3/1/2032

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.

Eligibility Criteria

Age Range: 45 years to 75 years

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

Interventions

BIOLOGICAL

bemdaneprocel

PROCEDURE

Sham surgery

Interested in This Trial?

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Conditions

Parkinsons Disease (PD)

Locations

Banner Sun Health Research Institute

Sun City, Arizona 85351

United States

University of Arkansas for Medical Sciences

Little Rock, Arkansas 72205

United States

UCLA NeuroTranslational Research Center

Los Angeles, California 90095

United States

UCI Medical Center - Neurology

Orange, California 92868

United States

Stanford Neuroscience Health Center

Palo Alto, California 94304

United States

University of Colorado Hospital - Neurology Clinic

Aurora, Colorado 80045

United States

MedStar Georgetown University Hospital

Washington D.C., District of Columbia 20007

United States

Mayo Clinic Jacksonville - Neurology

Jacksonville, Florida 32224

United States

University of Miami Health System - Neurology

Miami, Florida 33136

United States

USF Parkinson's Disease and Movement Disorders Center

Tampa, Florida 33613

United States

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

Chicago, Illinois 60611

United States

Rush University Medical Center - Neurology

Chicago, Illinois 60612

United States

University of Kansas Medical Center

Kansas City, Kansas 66160-8500

United States

University of Louisville Hospital

Louisville, Kentucky 40202

United States

Johns Hopkins Hospital - Neurology

Baltimore, Maryland 21287

United States

Tufts Medical Center - Neurology

Boston, Massachusetts 02111

United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts 02215

United States

Michigan Clinical Research Unit - Neurology

Ann Arbor, Michigan 48109

United States

University of Michigan Clinical Research Unit - Neurology

Ann Arbor, Michigan 48109

United States

Quest Research Institute

Farmington Hills, Michigan 48334

United States

UBMD Neurology

Buffalo, New York 14221

United States

Mount Sinai West - Neurology

New York, New York 10019

United States

New York Presbyterian/Weill Cornell Medical Center

New York, New York 10065

United States

Duke Neurological Disorders Clinic

Durham, North Carolina 27710

United States

University of Toledo Medical Center

Toledo, Ohio 43614

United States

OHSU Neurology Clinic, South Waterfront

Portland, Oregon 97239

United States

Rhode Island Hospital - Neurology

Providence, Rhode Island 02903

United States

Medical University of South Carolina, Movement Disorders Dept.

Charleston, South Carolina 29425

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232

United States

UTHealth Houston Neurosciences - Texas Medical Center

Houston, Texas 77030

United States

University of Vermont Medical Center

Burlington, Vermont 05401

United States

Virginia Commonwealth University

Richmond, Virginia 23284

United States

EvergreenHealth - Research Department

Kirkland, Washington 98034

United States

NeuRA (Neuroscience Research Australia)

Randwick, New South Wales 2031

Australia

Gold Coast Hospital & Health Service

Southport, Queensland 4215

Australia

Princess Alexandra Hospital

Woolloongabba, Queensland 4102

Australia

Monash Medical Centre

Clayton, Victoria 3168

Australia

The Alfred Hospital

Melbourne, Victoria 3004

Australia

The Royal Melbourne Hospital

Parkville, Victoria 3050

Australia

The University of British Columbia

Vancouver, British Columbia V6T 1Z3

Canada

University Health Network - Toronto Western Hospital

Toronto, Ontario M5T 2S8

Canada

CHUM - Centre hospitalier de l'Université de Montréal

Montreal, Quebec H2X 0C1

Canada