RecruitingPhase 3NCT06944522

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

exPDite-2: A Phase 3 Study to Assess the Efficacy and Safety of Midbrain Dopaminergic Neuronal Cell Therapy (Bemdaneprocel) for Participants With Parkinson's Disease


Sponsor

BlueRock Therapeutics

Enrollment

102 participants

Start Date

Jun 17, 2025

Study Type

INTERVENTIONAL

Conditions

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).


Eligibility

Min Age: 45 YearsMax Age: 75 Years

Inclusion Criteria8

  • 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 Criteria12

  • 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

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


Locations(33)

Banner Sun Health Research Institute

Sun City, Arizona, United States

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

UCLA NeuroTranslational Research Center

Los Angeles, California, United States

University of Colorado Hospital - Neurology Clinic

Aurora, Colorado, United States

MedStar Georgetown University Hospital

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

University of Miami Health System - Neurology

Miami, Florida, United States

USF Parkinson's Disease and Movement Disorders Center

Tampa, Florida, United States

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

Chicago, Illinois, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

University of Louisville Hospital

Louisville, Kentucky, United States

Tufts Medical Center - Neurology

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Michigan Clinical Research Unit - Neurology

Ann Arbor, Michigan, United States

University of Michigan Clinical Research Unit - Neurology

Ann Arbor, Michigan, United States

Quest Research Institute

Farmington Hills, Michigan, United States

UBMD Neurology

Buffalo, New York, United States

Mount Sinai West - Neurology

New York, New York, United States

New York Presbyterian/Weill Cornell Medical Center

New York, New York, United States

University of Toledo Medical Center

Toledo, Ohio, United States

Rhode Island Hospital - Neurology

Providence, Rhode Island, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

UTHealth Houston Neurosciences - Texas Medical Center

Houston, Texas, United States

University of Vermont Medical Center

Burlington, Vermont, United States

Virginia Commonwealth University

Richmond, Virginia, United States

EvergreenHealth - Research Department

Kirkland, Washington, United States

NeuRA (Neuroscience Research Australia)

Randwick, New South Wales, Australia

Gold Coast Hospital & Health Service

Southport, Queensland, Australia

Princess Alexandra Hospital

Woolloongabba, Queensland, Australia

Monash Medical Centre

Clayton, Victoria, Australia

The Alfred Hospital

Melbourne, Victoria, Australia

The Royal Melbourne Hospital

Parkville, Victoria, Australia

University Health Network - Toronto Western Hospital

Toronto, Ontario, Canada

CHUM - Centre hospitalier de l'Université de Montréal

Montreal, Quebec, Canada

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