Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
This study, called RAINBOW, is looking into a new medicine called RAY121. It's designed for people with certain immune system diseases where the body's defence system mistakenly attacks itself. These conditions include Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), and Immune Thrombocytopenia (ITP). The main goals are to check if RAY121 is safe and well-tolerated, how the body handles the medicine, and if it might help symptoms. This is an early phase (Phase 1b) trial, so it's one of the first steps to understand if RAY121 has potential. Participants will receive multiple doses of the medicine.
At a glance
What is this study about?
When your body's immune system, which normally fights off infections, starts attacking healthy parts of your own body, it can lead to various 'immunological diseases'. This study is investigating a new medication called RAY121 for six different conditions where this happens: Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), and Immune Thrombocytopenia (ITP).
RAY121 works by targeting a specific part of the immune system called the 'classical complement pathway'. This pathway can sometimes become overactive in these conditions, causing problems. By blocking it, researchers hope to reduce the immune system's harmful activity and improve symptoms. This is a "basket trial," meaning it includes people with several different diseases, but they all share a similar problem with their immune system that RAY121 aims to address.
This is a 'Phase 1b' trial, which means it's an early stage of clinical research. The main focus at this stage is to check the safety of RAY121, how well people tolerate it, and how the medicine moves through and is used by the body. Researchers will also look for early signs that the medicine might be helpful for these conditions. This information is crucial for deciding if RAY121 should be studied further in larger trials.
Key takeaways
- This study tests a new medicine, RAY121, for several immune conditions.
- It's an early-stage trial focusing on safety and how the medicine works.
- Conditions studied include Antiphospholipid Syndrome, Bullous Pemphigoid, and Behçet's Syndrome.
- Participation involves regular clinic visits and health checks.
- You can stop participating at any time.
- Your involvement helps advance medical research.
Who may be eligible?
To join this study, you must be between 18 and 75 years old, though for Bullous Pemphigoid, you can be up to 85. You also need to be able to understand and follow the study's instructions. If you're a woman who could become pregnant, you'll need to agree to use effective contraception or avoid sexual intercourse. Men must also agree to use contraception and not donate sperm while in the study.
Beyond these general requirements, you must have one of the specific conditions being studied. For example, if you have Antiphospholipid Syndrome (APS), your doctor will need to confirm your diagnosis based on specific blood tests and symptoms like certain skin issues or kidney problems.
If you have Bullous Pemphigoid (BP), your diagnosis will need to be confirmed by specific skin tests, and your skin blisters and itching must be active enough to meet certain scores. If you have Behçet's Syndrome, you need to have been diagnosed, have frequent and active mouth sores, and have tried other treatments already.
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 between 18 and 75-85 years old?
- Do you have one of the specific immune conditions listed (APS, BP, BS, DM, IMNM, ITP)?
- Are you able to attend regular clinic visits for check-ups?
- If you could have children, are you willing to use effective contraception?
- Have you been diagnosed by a doctor with one of the study conditions and meet specific criteria from your medical history?
What does participation involve?
If you decide to take part in this study, you will first go through a screening process to make sure you're suitable. Once confirmed, you will receive doses of the study medicine, RAY121. The study will involve a number of visits to the clinic so the research team can carefully monitor your health. They will perform various assessments, which might include blood tests, physical examinations, and detailed checks of your symptoms and how you are feeling.
The research team will be looking at how the medicine affects your body, including any side effects. You will be asked to complete questionnaires or assessments about your condition. The total length of your participation in the study, including the number of doses, visits, and follow-up, will be explained in detail by the study team.
Potential risks and benefits
Locations (69)
- University of California-IrvineVerified postcodeOrange, United States· Recruiting
- Johns Hopkins UniversityVerified postcodeBaltimore, United States· Not yet recruiting
- Northwell Health, LLC PRIMEVerified postcodeLake Success, United States· Active not recruiting
- Hospital for Special SurgeryVerified postcodeNew York, United States· Active not recruiting
- Universtity of North Carolina at Chapel HillVerified postcodeChapel Hill, United States· Not yet recruiting
- Ohio State UniversityVerified postcodeColumbus, United States· Withdrawn
- Oregon Health & Science UniversityVerified postcodePortland, United States· Recruiting
- University of Pennsylvania, Perelman Center for Advanced MedicineVerified postcodePhiladelphia, United States· Recruiting
- Amarillo Center for Clinical ResearchVerified postcodeAmarillo, United States· Active not recruiting
- Austin Neuromuscular CenterVerified postcodeAustin, United States· Recruiting
- Nerve and Muscle Center of TexasVerified postcodeHouston, United States· Not yet recruiting
- Royal Prince Alfred HospitalVerified postcodeCamperdown, Australia· Recruiting
Common questions
What is RAY121?
RAY121 is a new medicine being tested in this study. It aims to block a part of the immune system that can be overactive in certain conditions.
What are 'immunological diseases'?
These are conditions where your immune system mistakenly attacks healthy parts of your own body, causing a range of symptoms.
Is this medicine approved for use?
No, RAY121 is an investigational medicine. It's in the early stages of testing to see if it's safe and effective.
How long will I be in the study if I join?
The exact length of time will depend on your specific condition and the study plan, but the team will explain this to you fully.
Will I receive actual medicine or a dummy pill?
This study involves receiving RAY121, but the team will explain if there are different treatment groups.
How to find out more
Clinical trials information
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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