A study of JNJ-88549968 for the treatment of calreticulin (CALR)-mutated myeloproliferative neoplasms
Myeloproliferative neoplasms (MPNs) are rare blood cancers that cause the bone marrow to make too many blood cells. This can lead to problems such as blood clots, fatigue, or bleeding. This clinical trial is testing a new drug called JNJ-88549968 in people with specific types of MPNs—called essential thrombocythemia (ET) or myelofibrosis (MF)—who carry a genetic change known as a CALR mutation. The study drug works by acting as a bridge. It connects the body's natural immune defence cells (T-cells) directly to the abnormal cancer cells to help destroy them. Because this medicine has not been given to humans before, the main goals are to find the safest dose and see how the body handles it. Participants will receive close monitoring and regular health checks at Guy's Hospital in London for up to two years.
A colpo d'occhio
Di cosa tratta questo studio?
Myeloproliferative neoplasms (MPNs) are a group of rare, long-term blood cancers. In healthy bone marrow, the body carefully controls how many blood cells are made. In people with MPNs, this control system fails, causing an overproduction of blood cells. This trial focuses on two types of MPNs: essential thrombocythemia (ET), where the body makes too many platelets, and myelofibrosis (MF), where scar tissue builds up in the bone marrow. Both conditions can cause serious complications, including blood clots, severe tiredness, an enlarged spleen, or progression to leukaemia.
Many people with ET or MF have an altered gene called the CALR mutation. This mutation causes cancer cells to carry an abnormal protein on their surface. The trial medicine, JNJ-88549968, is a specialised protein designed to spot this abnormal marker. At the same time, it latches onto T-cells, which are the infection-fighting soldiers of your immune system. By bringing these two cells together, the drug aims to help your own immune system recognise and destroy the cancer cells.
Because this is a 'Phase 1' trial, it is the first time JNJ-88549968 is being tested in humans. The primary purpose is not yet to prove how well it cures the disease, but to find the safest dose and understand any side effects. In the first part of the study, doctors test small, carefully chosen amounts to find the best dose. In the second part, more patients receive that chosen dose so doctors can study its safety and early effects in greater detail.
Taking part in this research helps doctors explore new, targeted ways to treat blood cancers. If successful, medicines like this could give patients more options when standard treatments are no longer working well.
Punti chiave
- Tests a new immune-based medicine (JNJ-88549968) for adults with CALR-mutated blood cancers (ET or MF).
- It is a Phase 1 study, meaning it is the first time the medicine is being tested in humans to find a safe dose.
- Designed for people whose previous standard treatments are no longer working well.
- The trial takes place at Guy's Hospital in London and lasts up to approximately two years.
- Participation is entirely voluntary, with thorough safety checks at every stage.
Chi può essere idoneo?
This trial is open to adults aged 18 and older who have been diagnosed with either essential thrombocythemia (ET) or myelofibrosis (MF). To take part, your blood cancer must test positive for a specific genetic change known as the CALR driver mutation.
You must have already tried standard treatments that have either stopped working or were not suitable. For ET, this means you have had at least two previous treatments to lower your blood counts, including a drug called hydroxyurea. For MF, you must have tried at least one medication known as a JAK inhibitor, unless your doctor determined that these medicines were not right for you. You also need to be reasonably active and able to look after your daily personal care.
You will not be able to join the trial if you have severe allergies to the study drug's ingredients, a history of an organ transplant, or another active cancer that has needed treatment within the past three years (apart from certain low-risk skin or cervical conditions). Your doctor will also check your blood counts before you join to ensure they meet safe minimum levels.
Questo studio potrebbe fare al caso tuo?
Rispondi a queste domande rapide per vedere se potresti essere idoneo. Questo è solo una guida — il team di ricerca prende la decisione finale.
- I am aged 18 or older.
- I have been diagnosed with either essential thrombocythemia (ET) or myelofibrosis (MF).
- My medical team has confirmed that my condition has the CALR gene mutation.
- I have previously tried standard treatments (such as hydroxyurea for ET or a JAK inhibitor for MF).
- I am well enough to carry out daily self-care and attend regular hospital visits in London.
Cosa comporta la partecipazione?
Taking part in this study lasts for up to two years and involves regular visits to Guy's Hospital in London. The study is split into two phases. In Part 1, doctors test different amounts of the drug to work out the safest dose. In Part 2, participants receive that chosen dose so researchers can look closer at how well it works and monitor safety.
During your visits, you will receive the study drug and undergo regular health assessments. These include physical examinations, blood and urine tests, heart traces (ECGs), and checks on your blood pressure and temperature. The medical team will keep a very close eye on how you are feeling and track any side effects. You will need to attend scheduled clinic visits for check-ups and follow-up care throughout the study period.
Rischi e benefici potenziali
Luoghi (1)
- -Approssimativo-, England
Domande comuni
What is a 'first-in-human' study?
It means this trial is the very first time this specific medicine is being given to people. Before this stage, the drug was only tested in laboratory settings to check its basic safety.
Is JNJ-88549968 a type of chemotherapy?
No. It is an immunotherapy treatment. Instead of attacking all fast-growing cells like traditional chemotherapy, it guides your own immune cells to seek out and destroy cancer cells carrying the CALR mutation.
Where does the study take place?
The study is running at Guy's Hospital in London, UK.
Do I have to pay to take part?
No, clinical trial medicines and study-related tests are provided free of charge. You can ask the hospital team if travel expenses can be reimbursed.
Can I leave the trial if I change my mind?
Yes. Taking part is completely your choice. You can withdraw from the trial at any point, for any reason, and your regular medical team will continue to look after you.
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