RecruitingPhase IIIInterventional

A Phase III placebo-controlled efficacy and safety study of mocravimod as an adjunctive and maintenance treatment in acute myeloid leukemia patients undergoing allogeneic hematopoietic cell transplantation

Acute myeloid leukaemia (AML) is an aggressive blood cancer. A stem cell transplant from a healthy donor is often used to treat it. The donated cells help destroy cancer, but they can also attack healthy organs. This serious side effect is called graft-versus-host disease (GvHD). This Phase III clinical trial tests whether an experimental medicine called mocravimod can improve transplant outcomes. Researchers believe mocravimod may help keep donated immune cells inside the bone marrow. This allows them to destroy leftover leukaemia cells while preventing them from traveling around the body to harm healthy tissue. Adults aged 18 to 75 with AML who are scheduled for a donor transplant can take part. Two out of three participants will receive mocravimod, and one out of three will receive a dummy pill (placebo). Treatment lasts for up to one year alongside standard transplant medicines.

At a glance

Status

Recruiting

Phase

Phase III

Sponsor

Priothera S.A.S.

Enrolment target

249

Start

30 Jun 2022

Estimated completion

31 May 2027

Acute myeloid leukemia

What is this study about?

Acute myeloid leukaemia (AML) affects blood-forming cells in the bone marrow. A standard treatment for many patients is an allogeneic stem cell transplant, where you receive healthy blood-forming cells from a donor. These donated immune cells are vital because they can hunt down and kill any remaining leukaemia cells. However, transplants carry significant risks. The disease can still return, and donated cells often attack the patient's own healthy tissues, causing a serious condition known as graft-versus-host disease (GvHD).

This trial investigates whether adding a new drug, mocravimod, to standard transplant care can solve both problems at once. Mocravimod is designed to stop donated white blood cells from wandering into other organs like the skin, liver, or gut. By keeping these cells focused mainly in the bone marrow, doctors hope the donated cells will wipe out hidden leukaemia cells without causing severe GvHD elsewhere in the body.

Finding better ways to manage transplants is crucial. Current drugs can help prevent GvHD, but it remains a dangerous complication, and preventing leukaemia relapse is still a major hurdle. If mocravimod proves safe and effective, it could help future transplant patients live longer, healthier lives without their cancer coming back.

Key takeaways

Tests a new drug called mocravimod added to donor stem cell transplants for adults with AML.
Aims to prevent leukaemia returning while reducing graft-versus-host disease (GvHD).
Two in three participants receive the trial drug, and one in three receives a placebo.
Treatment lasts for up to one year, with regular clinic visits and bone marrow checks.
Participation is voluntary, and you may leave the study at any point without losing standard care.

Who may be eligible?

This trial is open to adults aged between 18 and 75 who have been diagnosed with acute myeloid leukaemia (AML) and are already scheduled to receive a donor stem cell transplant. Your cancer must currently be in complete remission, meaning standard tests show no active signs of leukaemia in your blood or marrow, though some people with slow blood count recovery may still take part.

To be eligible, you must weigh at least 50 kg (around 7 stone 12 lbs) and be in good enough general health to carry out daily activities, such as light housework or desk work. Your care team must also expect your general life expectancy to be at least six months at the start of screening.

You cannot join this study if you have a specific subtype of blood cancer called acute promyelocytic leukaemia (APL), or if you are not deemed suitable for a donor stem cell transplant by your specialist team.

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.

I am between 18 and 75 years old.
I have been diagnosed with acute myeloid leukaemia (AML).
My leukaemia is currently in complete remission.
I am already scheduled to have a stem cell transplant from a donor.
I weigh at least 50 kg (approx. 7 stone 12 lbs).
I am well enough to walk and perform light daily tasks.

Answer every question to see your result.

What does participation involve?

Taking part begins shortly before your donor stem cell transplant and continues for up to one year of treatment, followed by long-term health monitoring. You will be randomly assigned to take either mocravimod or an inactive dummy pill (placebo) once a day. Neither you nor your doctor will know which one you are taking. You have a two-in-three chance of receiving the trial drug.

Much of the early trial will take place while you are already staying in hospital for your transplant. Once you go home, you will attend regular hospital appointments for check-ups. These visits will include blood tests, heart scans (MUGA scans), CT scans, eye examinations, and routine bone marrow samples to check if the leukaemia has returned.

Throughout the study, an independent safety committee will monitor all participants to ensure the treatment is safe. After your one-year treatment finishes, your doctors will continue to check your health and quality of life as part of your standard long-term follow-up care.

Potential risks and benefits

Taking part may or may not help your condition directly. If mocravimod works as hoped, it could lower the chance of your leukaemia returning and reduce the risk of graft-versus-host disease, but this cannot be guaranteed. Your involvement will also help doctors discover better treatments for future patients. However, there are risks. Mocravimod may cause mild to serious side effects, though you will receive medications to help manage them. Routine study tests may also cause temporary discomfort, including bruising from blood tests, soreness from bone marrow biopsies, blurry vision from eye drops, or minor radiation exposure from scans. You can leave the study at any time without giving a reason, and your regular NHS medical care will not be affected.

Locations (1)

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Approximate
-, England

Common questions

What is the difference between mocravimod and standard care?

Standard care uses medicines to calm the new immune system and prevent donor cells attacking your body. Mocravimod is an experimental add-on designed to keep those cells inside the bone marrow so they attack leftover cancer rather than healthy organs.

Will I definitely receive the trial drug?

No. Two out of three participants will receive mocravimod, while one out of three will receive a placebo (dummy medicine). A computer randomly decides which you receive.

Will taking part delay my stem cell transplant?

No. The trial medication starts just before your planned transplant and is designed to fit alongside your scheduled hospital treatment.

Can I keep taking mocravimod after the study ends?

No. The trial medicine is provided for up to one year. Once this period finishes, your doctor will guide you on the best standard maintenance or follow-up plan for your recovery.

What happens if I change my mind?

Joining the study is completely voluntary. You are free to withdraw at any stage, and doing so will never affect your standard NHS treatment or relationship with your healthcare team.

How to find out more

Eduardo Olavarria

e.olavarria@nhs.net +44 (0)203 313 1000 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.