Active not recruitingPHASE3INTERVENTIONAL

Phase 3 Study of Pelabresib (CPI-0610) in Myelofibrosis (MF) (MANIFEST-2)

This clinical trial, called MANIFEST-2, is a Phase 3 study investigating a new medication, pelabresib, for people with myelofibrosis. Myelofibrosis is a condition where scar tissue builds up in the bone marrow, affecting blood cell production and often causing an enlarged spleen and other symptoms. The study aims to find out if pelabresib, when given with ruxolitinib (a common myelofibrosis drug), works better than ruxolitinib alone. This is for adults who haven't yet used certain types of targeted drugs (called JAK inhibitors) for their myelofibrosis. Researchers want to see if the pelabresib combination can help improve symptoms, reduce spleen size, and generally make a meaningful difference to patients' lives.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

430

Start

09 Apr 2021

Estimated completion

30 Jun 2027

Myelofibrosis Primary Myelofibrosis Post-polycythemia Vera Myelofibrosis Post-essential Thrombocythemia Myelofibrosis

What is this study about?

This study is looking at a new medicine called pelabresib for people living with myelofibrosis. Myelofibrosis is a condition that affects your bone marrow, which is the spongy tissue inside your bones where blood cells are made. In myelofibrosis, scar tissue builds up in the bone marrow, making it harder for your body to produce enough healthy blood cells. This can lead to various symptoms like extreme tiredness, unexplained weight loss, night sweats, and often a swollen spleen.

Pelabresib is a new type of drug that works differently from some existing treatments. In this study, it's being tested alongside ruxolitinib, which is a medicine commonly used for myelofibrosis. The main goal is to see if taking pelabresib with ruxolitinib is more effective than just taking ruxolitinib on its own. Researchers are particularly interested in whether this combination can help reduce the size of the spleen and improve the challenging symptoms that people with myelofibrosis often experience.

This is a 'Phase 3' study, which means it's one of the final stages of testing before a new medicine might be considered for wider use. It involves a larger number of patients to gather strong evidence about how well the medicine works and if it's safe. Taking part in such a study contributes to medical knowledge and could potentially lead to new and better treatment options for myelofibrosis in the future.

Key takeaways

Tests a new drug (pelabresib) for myelofibrosis.
Compares pelabresib + ruxolitinib to ruxolitinib + placebo.
For adults with myelofibrosis who haven't used certain prior drugs.
Aims to improve symptoms and reduce spleen size.
Study contributes to finding better treatments for myelofibrosis.
Participation involves regular clinic visits for monitoring.

Who may be eligible?

To join this study, you would need to be at least 18 years old and have a confirmed diagnosis of myelofibrosis. This includes primary myelofibrosis or myelofibrosis that developed after having polycythemia vera or essential thrombocythemia. Importantly, you should not have received treatment with certain types of drugs called 'JAK inhibitors' before.

Doctors will also check your overall health, including how well your kidneys and liver are working. You would need to be experiencing certain symptoms related to your myelofibrosis, and your spleen would need to be enlarged. There are also specific health scores that your doctor would assess to determine if the study is right for you.

However, you wouldn't be able to participate if you've had surgery to remove your spleen or had radiation treatment to your spleen in the past six months. Also, if you have other serious health problems or are taking certain medications that might interfere with the study drugs, you might not be eligible.

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 at least 18 years old?
Do you have a confirmed diagnosis of myelofibrosis?
Have you NOT used drugs called 'JAK inhibitors' before for your myelofibrosis?
Are you currently experiencing myelofibrosis symptoms like tiredness or an enlarged spleen?
Have you NOT had spleen surgery or radiation in the last 6 months?

Answer every question to see your result.

What does participation involve?

If you join this study, you would be randomly assigned to one of two groups. One group would receive pelabresib along with ruxolitinib, and the other group would receive a 'placebo' (an inactive substance that looks like pelabresib) along with ruxolitinib. This is done so researchers can accurately compare the effects of pelabresib. You wouldn't know which group you are in, nor would your study doctor.

Participation would involve regular visits to the clinic for check-ups, blood tests, and assessments of your symptoms and spleen size. You would take the study medications as prescribed, usually as tablets. The total duration of your participation would depend on how your treatment progresses, but it would involve ongoing monitoring and follow-up to track your health and the effectiveness of the treatment.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as access to a new treatment (pelabresib) before it's widely available, and close monitoring by medical professionals. This could potentially improve your myelofibrosis symptoms and spleen size. However, there's no guarantee that the new treatment will be effective for everyone, and it might not work better than existing treatments. There could also be side effects from the study medications, some of which might be unknown. All potential side effects would be carefully explained to you by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (156)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Alabama at Birmingham
Verified postcode
Birmingham, United States
Ironwood Physicians P.C. dba Ironwood Cancer and Research Centers
Verified postcode
Chandler, United States
UC San Diego Moores Cancer Center
Verified postcode
La Jolla, United States
USC/Norris Comprehensive Cancer Center
Verified postcode
Los Angeles, United States
UCLA Medical Center
Verified postcode
Los Angeles, United States
Yale University School of Medicine
Verified postcode
New Haven, United States
Mayo Clinic
Verified postcode
Jacksonville, United States
Moffitt Cancer Center
Verified postcode
Tampa, United States
Decatur Memorial Hospital Cancer Care Center of Decatur/Cancer Care Specialists of IL
Verified postcode
Decatur, United States
Franciscan Health/Indiana blood and Marrow Transplantation
Verified postcode
Indianapolis, United States
Norton Cancer Institute, St. Matthews Campus
Verified postcode
Louisville, United States
University of Michigan Medical Center
Verified postcode
Ann Arbor, United States

Common questions

What is myelofibrosis?

Myelofibrosis is a bone marrow condition where scar tissue forms, making it hard for your body to produce normal blood cells. This can lead to problems like an enlarged spleen and tiredness.

What is pelabresib?

Pelabresib is a new experimental medicine being tested in this study. It's designed to work in a specific way to help manage myelofibrosis.

What is a 'Phase 3' study?

Phase 3 is an advanced stage of drug testing involving many people. It's done to confirm if a new medicine is effective and safe compared to existing treatments.

Will I know if I'm getting the new drug?

No, this is a 'blinded' study. Neither you nor your study doctor will know if you're receiving pelabresib or a placebo (inactive substance) alongside ruxolitinib.

Can I stop participating in the study?

Yes, you can choose to leave the study at any time, for any reason, without it affecting your current or future medical care.