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exPDite-2: No Time To Waste

No Time to Waste

exPDite-2 is a double-blind, simulated surgery-controlled phase 3 study evaluating the potential efficacy and safety of an investigational cell therapy called bemdaneprocel (bem-da-nepro-cell).

Bemdaneprocel is currently being studied and is not approved for use in any country.

The sponsor of the exPDite-2 study is BlueRock Therapeutics.

About bemdaneprocel

How is bemdaneprocel created?

BlueRock has developed a way to engineer dopaminergic neurons from stem cells. Bemdaneprocel contains the early form of these dopaminergic neurons, which are implanted in an area of the brain known as the putamen.

How is bemdaneprocel thought to work?

Bemdaneprocel is a cell therapy designed to replace the dopamine-producing neurons that are lost in Parkinson’s disease with the aim of engrafting (meaning integrate) into the brain to restore dopaminergic function.

Your brain contains neurons that make an important chemical called dopamine.

Parkinson’s disease causes these neurons to start to die, resulting in less dopamine production and causing the problems with movement and balance experienced by people living with Parkinson’s disease.

When implanted into your brain in a 1-time surgery, bemdaneprocel may replace these missing or damaged neurons with the aim of integrating into the brain to produce dopamine.

exPDite-2 will evaluate whether bemdaneprocel is effective in replacing lost dopaminergic neurons in people with Parkinson’s disease to improve motor symptoms and quality of life. In the phase 1 study, bemdaneprocel was well-tolerated.

Exploring a new way: Creating bemdaneprocel

Creating a cell therapy

  • Different parts of your body are made up of different types of cells, like muscle and brain cells.
  • Stem cells are special because they can be converted into various types of cells to potentially do any type of job your body needs them to do (this is called pluripotency).
  • Cell therapy aims to replace specific cell types lost to disease.

  • Embryonic stem cells were used to create bemdaneprocel. These initial cells were collected several years ago from donated embryos not used during in vitro fertilization (meaning the embryos were never implanted in the uterus and were never fetal cells).  This is a highly regulated process requiring consent from the donor.
  • These stem cells can keep dividing and be stored in a “bank” so that a single stem cell can provide a large number of identical cells that can be used to create bemdaneprocel, without collecting additional embryonic stem cells.
Talk to us about participating in exPDite-2

Are you a good fit for exPDite-2?

Could I be considered for the exPDite-2 study?

You may be considered for the study
if you …

  • were diagnosed with Parkinson’s disease 4–12 years ago.
  • are 45–75 years old.
  • are taking levodopa-based treatment to control motor symptoms.
  • have at least 2.5 hours of OFF time each day.

You may not be able to take part if you ...

You may not be able to take part if you ...

  • have had deep-brain stimulation (DBS).
  • suffer from a condition such as multiple sclerosis, epilepsy, substance abuse, an infection, or had cancer in the last 5 years.
  • are taking certain medicines that might affect the study results.
  • have had gene or cell therapy, surgery or radiation therapy to your brain, or are unable to have a general anesthetic or surgery.
  • are pregnant or breastfeeding.

A study doctor will tell you more about these requirements.

For qualified study participants, all treatments and tests may be provided at no charge. Support with travel associated with the study and reasonable reimbursement costs for you and your care partner may be provided throughout the exPDite-2 study. The study doctor and/or their staff will discuss this with you at your first visit.

No Time to Waste

About the exPDite-2 study

The exPDite-2 study is designed to evaluate the efficacy and safety of bemdaneprocel on movement, balance, motor symptoms, and quality of life in adults with Parkinson’s disease, with input from families living with Parkinson’s.

You may tell your doctor about your participation in the study, and they may remain involved in your care.

How the study works

Part A: Screening

  • Period: 4–90 days before surgery
  • You will visit a clinical study location, where doctors will perform tests including an
    MRI
    and a
    PET
    scan to check whether you are able to take part in the study. Some of these tests may involve travelling to another center.
  • The study doctor will also make sure your current dose of Parkinson’s medication(s) is working as well as it can for your symptoms. If not, the study doctor may adjust your medications. This is called “optimization.”

  • Period: 3–10 days before surgery
    • You will meet with the study doctor for more tests and give blood and urine samples.

Part B: Surgery

  • At a surgical center, you will receive a second MRI that will help guide implantation of the dopaminergic neurons.
  • You will then receive a general anesthetic and a surgeon will perform a 1-time surgery where either bemdaneprocel will be implanted in your brain or there will be a simulated surgery. For the simulated surgery, you will have a similar procedure with no active cells implanted.
  • Two-thirds of patients will receive bemdaneprocel and one-third of patients will undergo a simulated surgery with no implantation of cells.
  • It is expected that most participants will be discharged within 24 hours after close observation.
  • You will be required to return to the hospital for 1 visit 4 days after your surgery where you will have an IV (intravenous) infusion of either a medication that helps keep your immune system from attacking the cells or a saline solution (if you did not receive bemdaneprocel). If you do not live close to the surgical center, accommodations will be provided for you to stay near the surgical center so you can return on Day 4.
  • After the Day 4 visit, all of the remainder of your study visits will be completed at the clinical study location.

Part C: Monitoring

  • Period: 18 months
    • Over the next 18 months, you will have about 17 study visits. You may have the option of having some of these visits at home, but others will require going to the clinical study location. Some visits will only involve a review of medications and collection of blood and urine samples. During this time, you will continue with your regular Parkinson’s disease medication, though you will be asked to delay your dose on the day of some visits. Your doctor will also adjust your medication if needed.
  • For the first year after surgery you will take immunosuppressive medicine (for the bemdaneprocel group) or a placebo (for the simulated surgery group).

  • Getting bemdaneprocel treatment
    • After the last patient finishes the study and, if the study shows bemdaneprocel to be safe and effective, you may have the opportunity to receive the study medication (bemdaneprocel). Please note, the study doctor must first verify you are still eligible for the study to receive bemdaneprocel. After you receive the study medication, you will have approximately one visit a month for 18 months and then be followed for approximately 5 years from your surgery.

  • Period: Approximately 5 years from surgery
  • Everyone who receives bemdaneprocel will still be seen by the study doctors for approximately 5 years after their surgery.
  • The extended evaluation period allows researchers to evaluate the results over a longer period of time and is required by regulators for advanced therapies under investigation.

The role of your care partner

  • Parkinson’s disease also affects your family and friends.
  • Your care partner will also have the opportunity to support your participation in the study by coming to your visits and completing questionnaires.

Study locations

Where are the study centers?

Planned exPDite-2 study centers are shown on the map. You and your care partner may need to travel for MRIs and surgery. Reimbursement for travel associated with the study and reasonable expenses for you and your care partner may be available. All medication and procedures may be covered as part of the study.

Where are the study centers?

All exPDite-2 study centers are shown on the map. You and your care partner may need to travel for MRIs and surgery. Reimbursement for travel associated with the study and reasonable expenses for you and your care partner may be available. All medication and procedures may be covered as part of the study.

No Time to Waste

Parkinson’s disease is a progressive neurodegenerative disorder involving a loss of dopamine-producing neurons in the brain.1

Currently available medications and therapies aim to ease symptoms and improve quality of life but do not address the underlying causes of the disease.2-4

Progression of Parkinson’s disease often requires the addition of levodopa-based treatments which can lead to increases in adverse reactions, including dyskinesia (or uncontrolled, involuntary movement) and other motor and nonmotor symptoms.5

Current treatments do not address the loss of dopaminergic neurons (an underlying cause of disease), do not affect disease progression, and lose effectiveness over time.1,6

Taking part in a clinical study

Common questions about taking part in a clinical study.

  • A clinical study tests a new treatment to find out how safe and effective it is. Clinical studies help doctors to understand how to use the treatment, what the benefits are, and whether there are any side effects. When you take part in a clinical study you are helping to bring potential new treatments to patients like you.

  • A simulated surgery-controlled study has 2 groups. One group will have a 1-time surgery with bemdaneprocel implanted in your brain. The second group will receive a simulated surgery, where you will have a similar procedure with no active cells implanted.

  • This is a study in which neither the participants nor your study physician know if they are receiving the active treatment or the simulated surgery.

  • This is the process where some study participants (by chance) are assigned to the group that will receive the active medication and some to the simulated surgery. This helps to remove bias for researchers.

  • Yes, you will receive support during the study to ensure you have all the information you need. Clinical studies do not take place until they have been approved by Health Authorities and Ethics committees, and there are strict rules to protect your safety and well-being. You will be in the care of the study doctor and team, and you may continue to see your regular doctor.

  • Treatments and tests may be provided free of charge throughout the study. You may also be reimbursed for reasonable travel costs associated with the study for you and a companion.

  • Many people get involved with clinical studies to help make new treatments available to people with Parkinson’s disease. Participating in a clinical study is a personal decision, and it may help to talk with your friends and family before you decide if it is right for you.

  • When you get involved with a clinical study you are free to withdraw at any time and for any reason.

Clinical trial phases

here are generally 4 phases of clinical trials. Successful completion of each is required to advance to the next stage.

Would you like to be part of the exPDite-2 study?

Would you like to know more about the exPDite-2 study? Our team is waiting to hear from you.

  • Call. Talk to our team +1-877-380-3967

  • Email. Complete the Contact Us form and we will get in touch.

  • Study requirements. Complete the checklist to see if the exPDite-2 study is right for your or someone you know. Our team will get in touch with you to discuss next steps.

References:  1. Ramesh S, Arachchige A. AIMS Neuro. 2023;10(3):200-231. 2. Yiannopoulou KG, Papageorgiou SG. J. Cent Nerv Syst Dis. 2020;12:1179573520907397. 3. Livingston G, et al. Lancet. 2020;396:413–446. 4. NIH-NINDS. Parkinson’s Disease: Challenges, Progress, and Promise. https://www.ninds.nih.gov/current-research/focus-disorders/parkinsons-disease-research/parkinsons-disease-challenges-progress-and-promise. Accessed March 2024. 5. Pandey S, Srivanitchapoom P. Ann Ind Acad Neurol. 2017;20(3): 190-196. 6. Tambasco N, et al. Current Neuropharmacol. 2018;16:1239-1252.