To Evaluate the Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis (LIMBER-313)

ID#: NCT04551066

Age: 18 years - 66+

Gender: All

Healthy Subjects: No

Study Phase: Phase 3

Recruitment Status: Recruiting

Start Date: April 30, 2021

End Date: April 14, 2026

Contact Information:
Incyte Corporation Call Center (US)
Incyte Corporation Call Center (ex-US)
Summary: The purpose of the study is to compare the efficacy of parsaclisib when combined with ruxolitinb versus placebo combined with ruxolitinib in participants with myelofibrosis.

Inclusion Criteria:

- Diagnosis of PMF, PPV-MF, or PET-MF.

- DIPSS risk category of intermediate-1, intermediate-2, or high.

- Palpable spleen of ≥ 5 cm below the left costal margin on physical examination at the screening visit.

- Active symptoms of MF at the screening visit, as demonstrated by the presence of a TSS of ≥ 10 using the Screening Symptom Form.

- Participants with an ECOG performance status score of 0, 1, or 2.

- Screening bone marrow biopsy specimen and pathology report(s) available that was obtained within the prior 2 months or willingness to undergo a bone marrow biopsy at screening/baseline; willingness to undergo bone marrow biopsy at Week 24 and every 24 weeks there after. Screening/baseline biopsy specimen must show diagnosis of MF.

- Life expectancy of at least 24 weeks.

- Willingness to avoid pregnancy or fathering children.

Exclusion Criteria:

- Prior use of any JAK inhibitor.

- Prior therapy with any drug that inhibits PI3K (examples of drugs targeting this pathway include but are not limited to INCB040093, idelalisib, duvelisib, buparlisib, copanlisib, and umbralisib).

- Use of experimental drug therapy for MF or any other standard drug (eg, danazol, hydroxyurea) used for MF within 3 months of starting study drug and/or lack of recovery from all toxicities from previous therapy to ≤ Grade 1.

- Inability to swallow food or any condition of the upper gastrointestinal tract that precludes administration of oral medications.

- Recent history of inadequate bone marrow reserve.

- Inadequate liver and renal function at screening.

- Active bacterial, fungal, parasitic, or viral infection that requires therapy.

- Active HBV or HCV infection that requires treatment or at risk for HBV reactivation.

- Known HIV infection.

- Uncontrolled, severe, or unstable cardiac disease that in the investigator's opinion may jeopardize the safety of the participant or compliance with the Protocol.

- Active invasive malignancy over the previous 2 years.

- Splenic irradiation within 6 months before receiving the first dose of study drug.

- Concurrent use of any prohibited medications.

- Active alcohol or drug addiction that would interfere with the ability to comply with the study requirements.

- Use of any potent CYP3A4 inhibitors or inducers within 14 days or 5 half lives(whichever is longer) before the first dose of study drug or anticipated during the study.

- Inadequate recovery from toxicity and/or complications from a major surgery before starting therapy.

- Currently breastfeeding or pregnant.

- Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.

- History of Grade 3 or 4 irAEs from prior immunotherapy.

- Receipt of any live vaccine within 30 days of the first dose of study drug