An Open Label, Single Arm, Phase I/II Clinical Study of Autologous CD4+ T-Cells Edited Ex-Vivo at the CD40LG Locus by CRISPR/Cas9 and IDLV-based vector in Patients with X-linked Hyper IgM Syndrome Type 1 (HIGM1)
This study is looking for men aged 18 and over with a rare immune condition called X-linked Hyper IgM Syndrome Type 1 (HIGM1). The main goal is to test a new type of gene therapy designed to improve their immune system. This therapy involves taking some of the patient's own immune cells, changing them in a lab, and then putting them back. Researchers want to find out if this new treatment is safe and what side effects it might cause, especially at different doses. They will also look at how well the immune system recovers and if patients have fewer serious infections over two years. The study will monitor participants closely for any reactions and track their health over time.
At a glance
What is this study about?
This study is for men who have a rare genetic condition called X-linked Hyper IgM Syndrome Type 1, also known as HIGM1. This condition means their immune system doesn't work properly, making them very vulnerable to infections. The usual treatments for HIGM1 can be lifelong and have their own challenges.
The researchers are testing a new approach called gene therapy. This isn't like taking a regular medicine; instead, it involves using the patient's own immune cells. These cells are taken from the patient, carefully changed in a lab using special tools, and then given back to the patient. The idea is to correct the problem in these cells so they can help the immune system function better. This is the very first time this specific therapy is being given to people, so the main focus is on making sure it's safe and understanding any side effects it might have.
The study will also look at how well the immune system starts to recover after the treatment and if there's a reduction in serious infections. They'll compare how many infections patients had before the treatment to how many they have afterwards. This will help doctors understand if this new therapy could be an effective, long-term solution for men with HIGM1, potentially improving their quality of life by making them less prone to illness.
Key takeaways
- This is a study for men aged 18+ with X-linked Hyper IgM Syndrome Type 1.
- It's testing a new gene therapy using your own altered immune cells.
- The main goals are to check for safety and find the right dose.
- Researchers will also see if it improves immune function and reduces infections.
- Participation involves blood samples, cell infusion, and two years of follow-up.
- Standard vaccinations may be given during the study.
Who may be eligible?
This study is specifically designed for men aged 18 and older. It is crucial that participants have a confirmed diagnosis of X-linked Hyper IgM Syndrome Type 1 (HIGM1).
There will be other detailed medical criteria that you would need to meet to be eligible for this study. These are put in place to ensure your safety and that the study results are as clear as possible. These might include certain blood test results, your general health, and whether you have other medical conditions.
It's important to discuss your full medical history with the study doctor to find out if this trial is right for you. They will be able to explain all the requirements and check if you meet them.
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.
- Are you a man?
- Are you 18 years old or older?
- Do you have a diagnosis of X-linked Hyper IgM Syndrome Type 1 (HIGM1)?
- Are you willing to attend regular follow-up appointments for two years?
- Are you open to receiving standard vaccinations if required by the study?
What does participation involve?
If you decide to take part, the study will involve several steps. First, you'll have some of your blood taken to collect special immune cells. These cells will then be sent to a lab to be carefully altered. While your cells are being prepared, you might need to have some standard vaccinations, like for rabies or tick-borne encephalitis, and a booster for common childhood diseases if you haven't recently had them.
Once your altered cells are ready, they will be given back to you through an infusion, similar to a drip. After this, you will have regular hospital visits for check-ups and tests. These tests will include blood samples to monitor your immune system and check for any side effects. You will be closely monitored for 28 days after each dose and then for a longer period, with follow-up appointments at 6 months, 1 year, and 2 years after your treatment. The total duration of active follow-up for the study is two years.
Potential risks and benefits
Locations (1)
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Common questions
What is X-linked Hyper IgM Syndrome Type 1 (HIGM1)?
It's a rare genetic condition where the immune system doesn't work properly, making people very prone to infections.
What is 'gene therapy' in simple terms?
It's a treatment that involves changing a person's own cells in a lab to fix a problem in their genes, then giving them back to the person.
Will I get other medicines during the study?
You might receive some standard vaccinations during the study, like those for rabies, tick-borne encephalitis, and a booster for common childhood diseases, if needed.
How long will I be monitored after the treatment?
You will be closely monitored regularly for two years, with specific check-ups at 6 months, 1 year, and 2 years after your treatment.
Who is eligible for this study?
This study is for men aged 18 and over who have a confirmed diagnosis of X-linked Hyper IgM Syndrome Type 1.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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