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JAK2 Inhibitor

Pacritinib for Myelofibrosis (PACIFICA Trial)

Phase 3

Recruiting

Research Sponsored by Swedish Orphan Biovitrum

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

TSS of ≥10 on the MPN-SAF TSS 2.0 or a single symptom score of ≥5 or two symptoms of ≥3, including only the symptoms of left upper quadrant pain, bone pain, itching, or night sweats

Age ≥18 years

Must not have

QT corrected by the Fridericia method (QTcF) prolongation >450 ms or other factors that increase the risk for QT interval prolongation (e.g., hypokalemia [defined as serum potassium <3.0 mEq/L that is persistent and refractory to correction], or history of long QT interval syndrome

Active or uncontrolled inflammatory or chronic functional bowel disorder such as Crohn's Disease, inflammatory bowel disease, chronic diarrhea, or chronic constipation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up end of week 12 to 2 years following week 24 visit

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a new drug, pacritinib, to see if it is better than the current standard of care for treating patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis who have severe thrombocytopenia (low platelet count). 399 patients will be enrolled and randomly assigned to either pacritinib or the current standard of care. The primary outcome measure is spleen response rate.

Who is the study for?

This trial is for patients with Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis who have severe thrombocytopenia (platelet count <50,000/μL). Participants must be adults with certain risk levels of the disease and able to tolerate MRI or CT scans. They should not have been treated with pacritinib before or other MF therapies recently and must not have significant recent bleeding history or certain heart conditions.

What is being tested?

The study tests Pacritinib (200 mg twice daily) against Physician's Choice medications in patients with specific types of myelofibrosis and severe low platelet counts. It randomly assigns participants in a 2:1 ratio to either Pacritinib or Physician's Choice therapy, aiming to enroll about 399 patients.

What are the potential side effects?

While the side effects for this trial are not explicitly listed here, common side effects from drugs like Pacritinib may include diarrhea, nausea, vomiting, fatigue, muscle pain. There can also be risks of more serious issues such as liver problems or an increased risk of infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I experience significant pain, itching, or night sweats due to my condition.

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I am 18 years old or older.

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I can take care of myself and am up and about more than half of my waking hours.

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My liver and kidney functions are within the required limits.

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I have taken ruxolitinib, but never more than 10 mg per day, and for no longer than 270 days.

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I have taken a JAK2 inhibitor for 90 days or less.

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My condition is classified as Intermediate-1, Intermediate-2, or High risk according to DIPSS.

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My spleen is enlarged and can be felt at least 5 cm below my rib cage.

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I have been diagnosed with a type of myelofibrosis.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My heart's electrical cycle is longer than normal or I have low potassium/have a history of long QT syndrome.

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I have an active or uncontrolled bowel disorder like Crohn's.

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I have been treated with more than one JAK2 inhibitor.

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I am not allergic to pacritinib, its ingredients, or certain diarrhea medicines.

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I do not have stomach or metabolic issues affecting medication absorption.

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I have been treated with pacritinib before.

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I have had my spleen removed or will have it removed.

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I am not pregnant or breastfeeding.

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I haven't had serious bleeding in the last 3 months not caused by surgery or injury.

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I have moderate to severe heart failure.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ end of week 12 to 2 years following week 24 visit

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~end of week 12 to 2 years following week 24 visit

This trial's timeline: 3 weeks for screening, Varies for treatment, and end of week 12 to 2 years following week 24 visit for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Spleen volume

Total Symptom Score (TSS) (excluding tiredness)

Secondary study objectives

Overall Survival (OS)

Patient Global Impression of Change (PGIC) assessed at Week 24

To compare the safety of pacritinib versus P/C therapy

Other study objectives

>25% SVR

Best response in SVR

Hemoglobin A1c

+12 more

Side effects data

From 2022 Phase 1 & 2 trial • 40 Patients • NCT02891603

70%

Diarrhea

50%

Febrile neutropenia

50%

Dehydration

50%

Anorexia

40%

Mucositis oral

30%

Rash maculo-papular

30%

Nausea

30%

Fatigue

30%

Hypertension

20%

Colitis

20%

Neutrophil count decreased

20%

Alanine aminotransferase increased

20%

Platelet count decreased

20%

Abdominal pain

20%

Skin infection

20%

C Diff

20%

Pain in extremity

10%

Sinusitis

10%

Soft tissue infection

10%

Oral pain

10%

Creatinine increased

10%

Oral fungus

10%

Anemia

10%

Pain of skin

10%

Edema trunk

10%

Rectal pain

10%

Ileus

10%

Vomiting

10%

Aspartate aminotransferase increased

10%

Hyponatremia

10%

CMV+

10%

Electrocardiogram QT corrected interval prolonged

10%

Periorbital edema

10%

Anaphylaxis

10%

Headache -migraine

10%

Abdominal Pain

10%

Metabolism and nutrition disorders - Other

10%

Acute GVHD of skin

10%

Urticaria

10%

Metabolism and Nutrition disorders - Other

10%

Edema limbs

10%

Hypotension

10%

Thromboembolic event

10%

Hypoxia

10%

Insomnia

10%

Anxiety

10%

Hallucinations

10%

Headache

10%

Cardiac disorders - Other

10%

Allergic reaction

100%

80%

60%

40%

20%

Study treatment Arm

Phase 1, Level 1: Pacritinib With Sirolimus and Tacrolimus

Phase 1, Level 2: Pacritinib With Sirolimus and Tacrolimus

Phase 2: Pacritinib With Sirolimus and Tacrolimus

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Pacritinib 200 mg BIDExperimental Treatment1 Intervention

To receive pacritinib 200 mg twice daily (BID) orally, at the same time of day, with or without food

Group II: Physician's Choice (P/C) therapyActive Control1 Intervention

The Physician's Choice (P/C) therapy (limited to single drugs from the following list: corticosteroids, hydroxyurea, danazol, or low-dose ruxolitinib). The proposed P/C regimen for a patient must be selected prior to randomization.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pacritinib

2017

Completed Phase 2

~330

0 / 19Eligibility criteria met