Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Idelalisib in Adults Receiving Ruxolitinib as Therapy for Primary, Post-Polycythemia Vera, or Post-Essential Thrombocythemia Myelofibrosis With Progressive or Relapsed Disease

• Status: Terminated
• Trial end date: 2017-11-20
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of this study is to evaluate the safety, tolerability, and pharmacokinetics of idelalisib in adults receiving ruxolitinib as therapy for intermediate to high-risk primary myelofibrosis (PMF), post-polycythemia vera, or post-essential thrombocythemia myelofibrosis (post-PV MF or post-ET MF) with progressive or relapsed disease.

This is a dose-escalation study. There will be 4 cohorts (A, B, C, D). Participants will receive an escalating dose or dose frequency of idelalisib based on the safety data of available cohort(s).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gilead Sciences
• Treatments: Idelalisib

Criteria

Key Inclusion Criteria:

• Individuals must have been on a stable dose of ruxolitinib for at least 4 weeks prior to study entry

• Individuals with PMF, post-PV MF, or post-ET MF classified as high risk or intermediate risk as defined by the Dynamic International Prognostic Scoring System (DIPSS) for PMF or DIPSS Plus, if cytogenetics are available

• Individuals with PMF, post-PV MF, or post-ET MF who are receiving ruxolitinib and meet 2013 Revised International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) and European Leukemia Net (ELN) response criteria with progressive and relapsed disease, with modifications for progressive disease complete remission (CR), partial remission (PR), or clinical improvement (CI)

• European Cooperative Oncology Group (ECOG) performance status of ≤ 2

• Required screening laboratory values as described in the protocol

• Willing and able to comply with scheduled visits, drug administration plan, imaging studies, laboratory tests, other study procedures, and study restrictions including mandatory prophylaxis for pneumocystis jiroveci pneumonia (PJP)

• Able to understand and willing to sign the informed consent form

Key Exclusion Criteria:

• Individuals on a stable ruxolitinib dose of 5 mg once daily

• History of prior allogeneic bone marrow progenitor cell or solid organ transplantation

• Ongoing drug-induced liver injury, alcoholic liver disease, non-alcoholic steatohepatitis, primary biliary cirrhosis, extrahepatic obstruction caused by cholelithiasis, cirrhosis of the liver

• Ongoing drug-induced pneumonitis

• Ongoing inflammatory bowel disease

• Ongoing alcohol or drug addiction

• Symptomatic congestive heart failure (New York Heart Association Classification > Class II), unstable angina, or unstable cardiac arrhythmia requiring medication

• Known hypersensitivity to the study investigational medicinal product (IMP), the metabolites, or formulation excipients

• Unwilling or unable to take oral medication

• Unresolved non-hematologic toxicities from prior therapies that are > Common terminology Criteria for Adverse Events (CTCAE) Grade 1 (with the exception of alopecia [Grade 1 or 2 permitted])

• Pregnant or lactating females

• Cytomegalovirus (CMV): Ongoing infection, treatment, or prophylaxis within the past 28 days

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.