To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib (LIMBER-304)

Trial ID: NCT04551053

This is a Phase 3 clinical study investigating the safety and efficacy of a potential treatment for participants with Myelofibrosis (MF) who have suboptimal response while receiving ruxolitinib monotherapy.

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Trial Details

The purpose of this Study is to compare the safety and therapeutic effects of adding parsaclisib (the Study Drug) or placebo to ruxolitinib therapy in patients with MF who continue to have significant enlargement of the spleen and symptoms of MF while on treatment with ruxolitinib only. The first part of the Study is double-blind, which means that you and your Study Doctor will not know if you are receiving Study Drug + ruxolitinib or placebo + ruxolitinib. This Study is also randomized, which means you will be randomly assigned (like flipping a coin) to receive the Study Drug + ruxolitinib or placebo + ruxolitinib. Placebo looks like the Study Drug but contains no medication. After you complete your 24-Week assessments, and if you were assigned to placebo + ruxolitinib, you will have an opportunity to “Crossover” to receive Study Drug + ruxolitinib. You will be in this Study for about 2 years, during which time you cannot participate in other research studies.

If you have questions about this study or would like to prescreen over the phone, please call: +1 (833) 278-6055

Estimated Enrollment

212 Participants

Phase

3

Eligibility Criteria

Participants must:

Must be 18 Years and older
Diagnosis of PMF, PPV-MF, or PET-MF
DIPSS risk category of intermediate-1, intermediate-2, or high
Ruxolitinib treatment ≥ 3 months, with a stable dose ≥ 8 weeks (patients can continue ruxolitinib treatment)
Suboptimal response to ruxolitinib as defined by palpable spleen ≥5 cm Below Left Costal Margin (BLCM) and Total Symptom Score (TSS) ≥10
Platelets ≥50 *109/L

Participants must not:

Prior therapy with PI3K inhibitor
Prior use of experimental drug therapy for MF, or any other standard drug, except ruxolitinib, within 3 months of starting study and/or lack of recovery from all toxicities from therapy to grade 1 or better
Recent history of inadequate bone marrow reserve
Inadequate liver or renal function at screening

Site Locations

Sites are located in the cities/states identified with the red pin:

What Will Happen in This Study

What is involved?

You will need to visit a study clinic. A study doctor will provide you with an informed consent and explain the trial. You will then be fully evaluated for participation and eligibility. If eligible, you will be enrolled in the study.

Your participation in the study will be approximately 3 years, and include regular, scheduled visits to the clinical study center. During these visits, you will undergo a physical exam and have blood work performed. A scan (MRI or CT) will be performed at protocol defined frequency during your study participation. Once the study has completed, if you are benefitting from treatment, you will have the option to continue the treatment.

Full details about study treatment, clinic visit schedule and exams will be discussed with you prior to any enrollment.

If you have questions about this study or would like to prescreen over the phone, please call: +1 (833) 278-6055

Frequently Asked Questions (FAQs)

Will I get paid to be a part of this clinical trial?

Some trials pay for the participants’ travel costs, time and commitment. Not all do. The amount paid varies. You should read the informed consent and ask the study doctor for this information for a specific clinical trial.

What is a placebo?

A placebo looks like a treatment but does not have any real treatment. In clinical trials, investigational treatments are often compared with placebos. Some participants are given a placebo instead of a treatment. This helps make sure that any effects are actually caused by the treatment being studied.

How can I check to see if I am eligible for this trial?

You can check if you are potentially eligible for this trial by completing a set of screening questions. You can find these on this web page by clicking the ‘Get Started’ button.

What does a typical research study look like?

Clinical trials help determine the safety and effectiveness of new vaccines or treatments. A research plan or protocol is created that outlines how trial participants may receive interventions which can include investigational medications, devices, or procedures. The trial may compare a new medical approach to a standard treatment option already available, to a placebo with no active investigational medication, or to no intervention at all. The trial team will share more details before you participate.

Can I quit the trial at any time?

Study volunteers have the right to drop out of the study at any time for any reason, or no reason at all. The study doctor or sponsor also has the right to discontinue a study volunteer at any time for the participant’s safety, or if the participant no longer meets the study requirements.

Who has access to your personal information should you inquire about participation in the study through this site?

If you share your personal data to contact a site, that data will be used by TrialScope to contact you about clinical trials and related information.

This trial is not actively recruiting.