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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

102 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-17

Study Completion Date

2032-03-31

Brief Summary

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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

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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.

Conditions

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Parkinsons Disease (PD)

Keywords

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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.

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Outcome Assessors

Study Groups

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Group A

Bemdaneprocel will be administered on Day 0

Group Type ACTIVE_COMPARATOR

bemdaneprocel

Intervention Type BIOLOGICAL

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

Group B

Sham surgery will be performed on Day 0

Group Type SHAM_COMPARATOR

Sham surgery

Intervention Type PROCEDURE

Sham surgery will be performed on Day 0

Interventions

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bemdaneprocel

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

Intervention Type BIOLOGICAL

Sham surgery

Sham surgery will be performed on Day 0

Intervention Type PROCEDURE

Other Intervention Names

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MSK-DA01 BRT-DA01

Eligibility Criteria

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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

Minimum Eligible Age

45 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

Group A

bemdaneprocel

Group B

Sham surgery

Interventions

bemdaneprocel

Sham surgery

Other Intervention Names

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Bayer

BlueRock Therapeutics

Responsible Party

Locations

University of Colorado Hospital - Neurology Clinic

MedStar Georgetown University Hospital

University of Miami Health System - Neurology

USF Parkinson's Disease and Movement Disorders Center

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

University of Kansas Medical Center

Tufts Medical Center - Neurology

University of Michigan Clinical Research Unit - Neurology

Quest Research Institute

UBMD Neurology

New York Presbyterian/Weill Cornell Medical Center

Rhode Island Hospital - Neurology

Vanderbilt University Medical Center

Virginia Commonwealth University

Countries

Central Contacts

Patient Engagement

Facility Contacts

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Patient Engagement

Other Identifiers

BRT-DA01-301