A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
This study is looking into a new treatment called PM359 for a rare inherited condition called Chronic Granulomatous Disease (CGD). CGD affects the body's immune system, making it harder to fight off infections and leading to inflammation. The treatment involves taking a person's own blood stem cells, using a new technology called Prime Editing to fix the genetic mistake causing CGD, and then giving these corrected cells back to the person. This is done after a short preparation to make space for the new cells. The main goal is to find out how safe and well this new approach works. It's a phase 1/2 study, meaning it's in its earlier stages of testing in people. Initially, it's for adults, with plans to include teenagers and younger children later. You can contact cgdtrial@primemedicine.com to learn more.
At a glance
What is this study about?
Chronic Granulomatous Disease (CGD) is a rare condition that's passed down through families. It affects some of your white blood cells, which are part of your immune system and help fight off infections. In people with CGD, these cells don't work properly, making them more likely to get serious infections and have problems with inflammation.
This study is trying a new treatment called PM359. It uses a new method called 'Prime Editing' to correct a specific genetic mistake, often called "delGT", in a gene called NCF1. This mistake is what causes CGD in about 20-25% of people with the condition. The idea is to take some of your own stem cells (special cells that can turn into different types of blood cells), correct the faulty gene in them outside your body, and then put these corrected cells back into you. The hope is that these new, fixed cells will then produce healthy white blood cells that can fight infections properly.
This is an early-stage study (Phase 1/2), which means it's one of the first times this treatment is being tested in people. The main aims are to see if the treatment is safe and if it helps to improve the symptoms of CGD. The study will start with adults and then later include teenagers (aged 12-17) and younger children (aged 6-11). It's important to remember that this is a new treatment, and researchers are still learning about how it works.
Key takeaways
- This study is for a new gene editing treatment for a specific type of Chronic Granulomatous Disease (CGD).
- It aims to correct the genetic mistake causing CGD in your own blood stem cells.
- The study is in its early stages (Phase 1/2) to check for safety and effectiveness.
- It involves collecting your cells, modifying them, and then returning them to your body after a preparation step.
- Long-term follow-up for 15 years is required to monitor the treatment's effects.
Who may be eligible?
To be considered for this study, you would need to have a specific type of Chronic Granulomatous Disease (CGD) caused by the "delGT" genetic change in the NCF1 gene. You should also have been receiving care for your CGD at a specialist centre for at least the last two years. Also, you must have had at least one serious CGD-related infection in the past, or currently have one that needs treatment, or have an inflammatory condition related to CGD that is active or needs treatment to keep it under control.
There are also some reasons why you might not be able to join. For example, if you are under 16 and have a very good match for a bone marrow donor in your family, you would likely not be eligible. You also can't have certain ongoing infections (like specific bacterial or fungal ones), or severe inflammation that isn't controlled by high-dose steroids. Other reasons include certain serious medical conditions, problems with your organs, or if you've had certain types of cancer. The medical team will check carefully to make sure the treatment is suitable and safe for you.
Finally, you must be willing to take part in this study and a follow-up study for a total of 15 years. This is so researchers can understand the long-term effects of the treatment.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Do I have CGD caused by the 'delGT' mistake in the NCF1 gene?
- Have I been treated for CGD at a specialist centre for at least the past two years?
- Have I had a serious CGD-related infection or an active CGD-related inflammation?
- Am I willing to participate in this study and a long-term follow-up study for a total of 15 years?
- Do I have any other serious medical conditions that might prevent me from joining?
What does participation involve?
If you decide to take part, the study will involve several steps. First, some of your stem cells will be collected from your blood. This is done after you've received some medicine to encourage these cells to move into your bloodstream, and then they're collected using a process similar to donating blood. These cells are then sent to a special facility to be 'corrected' using the Prime Editing technology (PM359).
Before the corrected cells can be given back to you, you'll have a preparation known as 'conditioning'. This typically involves receiving certain medicines, which help to make space in your bone marrow for the new, corrected cells. After this, the corrected cells (PM359) will be given back to you through a drip. You will then have regular check-ups and tests over a long period to monitor your health, how well the new cells are working, and to ensure your safety. The total time you would be involved in the study, including long-term follow-up, is 15 years.
Potential risks and benefits
Locations (5)
- University of California Los Angeles Medical CenterVerified postcodeLos Angeles, United States
- NIH Clinical CenterVerified postcodeBethesda, United States
- The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood CancersVerified postcodeNashville, United States
- CHU - Sainte Justine HospitalVerified postcodeMontreal, Canada
- University College of London HospitalVerified postcodeLondon, United Kingdom
Common questions
What is CGD?
CGD is a rare condition where certain white blood cells don't work properly, making it hard to fight off infections and leading to inflammation.
What is Prime Editing?
Prime Editing is a new technology used in this study to correct the specific genetic mistake that causes CGD in your own cells.
Will I need to stay in hospital?
You will likely need to be in hospital for the conditioning and to receive the new cells, and for close monitoring afterwards.
How long does the study last?
Your involvement, including follow-up, will be for a total of 15 years to track the long-term effects of the treatment.
Can children join this study?
Initially, the study is for adults. Plans are in place to include teenagers (12-17) and then younger children (6-11) later on.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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