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

August 3, 2026 updated by: BlueRock Therapeutics

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

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

102

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • New South Wales
      • Randwick, New South Wales, Australia, 2031
        • Recruiting
        • NeuRA (Neuroscience Research Australia)
        • Contact:
    • Queensland
      • Southport, Queensland, Australia, 4215
      • Woolloongabba, Queensland, Australia, 4102
    • Victoria
      • Clayton, Victoria, Australia, 3168
      • Melbourne, Victoria, Australia, 3004
      • Parkville, Victoria, Australia, 3050
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V6T 1Z3
    • Ontario
      • Toronto, Ontario, Canada, M5T 2S8
        • Recruiting
        • University Health Network - Toronto Western Hospital
        • Contact:
    • Quebec
      • Montreal, Quebec, Canada, H2X 0C1
        • Recruiting
        • CHUM - Centre Hospitalier de l'Universite de Montreal
        • Contact:
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259
      • Sun City, Arizona, United States, 85351
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Recruiting
        • University of Arkansas for Medical Sciences
        • Contact:
    • California
      • Los Angeles, California, United States, 90095
        • Recruiting
        • UCLA NeuroTranslational Research Center
        • Contact:
      • Orange, California, United States, 92868
      • Palo Alto, California, United States, 94304
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado Hospital - Neurology Clinic
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20007
        • Recruiting
        • MedStar Georgetown University Hospital
        • Contact:
    • Florida
      • Jacksonville, Florida, United States, 32224
      • Miami, Florida, United States, 33136
        • Recruiting
        • University of Miami Health System - Neurology
        • Contact:
      • Tampa, Florida, United States, 33613
        • Recruiting
        • USF Parkinson's Disease and Movement Disorders Center
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Parkinson's Disease and Movement Disorders Center at Northwestern Memorial Hospital
        • Contact:
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • Rush University Medical Center - Neurology
        • Contact:
    • Kansas
      • Kansas City, Kansas, United States, 66160-8500
    • Kentucky
      • Louisville, Kentucky, United States, 40202
    • Maryland
      • Baltimore, Maryland, United States, 21287
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
      • Boston, Massachusetts, United States, 02111
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan Clinical Research Unit - Neurology
        • Contact:
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • Michigan Clinical Research Unit - Neurology
        • Contact:
      • Farmington Hills, Michigan, United States, 48334
    • New York
      • Buffalo, New York, United States, 14221
      • New York, New York, United States, 10065
        • Recruiting
        • New York Presbyterian/Weill Cornell Medical Center
        • Contact:
      • New York, New York, United States, 10019
    • North Carolina
      • Durham, North Carolina, United States, 27710
    • Ohio
      • Toledo, Ohio, United States, 43614
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • OHSU Neurology Clinic, South Waterfront
        • Contact:
    • Rhode Island
      • Providence, Rhode Island, United States, 02903
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina, Movement Disorders Dept.
        • Contact:
    • Tennessee
      • Nashville, Tennessee, United States, 37232
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • UTHealth Houston Neurosciences - Texas Medical Center
        • Contact:
    • Vermont
      • Burlington, Vermont, United States, 05401
    • Virginia
      • Richmond, Virginia, United States, 23284
    • Washington
      • Kirkland, Washington, United States, 98034

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Group A
Bemdaneprocel will be administered on Day 0
Biological: bemdaneprocel
Investigational cell therapy comprising midbrain dopaminergic neuron progenitors derived from human embryonic stem cells
Other Names:
  • MSK-DA01
  • BRT-DA01
Sham Comparator: Group B
Sham surgery will be performed on Day 0
Procedure: Sham surgery
Sham surgery will be performed on Day 0

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day
Time Frame: From baseline to Week 78
From baseline to Week 78

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in PD diary measure of OFF-time, adjusted for a 16-hour waking day
Time Frame: From baseline to Week 78
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.
Time Frame: From baseline to Week 78
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.
Time Frame: From baseline to Week 78
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.
Time Frame: From baseline to Week 78
From baseline to Week 78
Incidence and severity of treatment-emergent adverse events
Time Frame: From baseline to month 60
From baseline to month 60

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BlueRock Therapeutics

Collaborators

Bayer

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 17, 2025

Primary Completion (Estimated)

March 1, 2027

Study Completion (Estimated)

March 1, 2032

Study Registration Dates

First Submitted

April 7, 2025

First Submitted That Met QC Criteria

April 17, 2025

First Posted (Actual)

April 25, 2025

Study Record Updates

Last Update Posted (Actual)

August 4, 2026

Last Update Submitted That Met QC Criteria

August 3, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BRT-DA01-301

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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