A Phase 1/2 Study of Oral SB1518 in Subjects With Chronic Idiopathic Myelofibrosis

Study Overview
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Brief Summary
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The study consists of two phases: The first portion of the study is a Phase 1 dose escalation study to determine the maximum tolerated dose and the dose limiting toxicities of SB1518 when given as a single agent orally once daily in subjects with Chronic Idiopathic Myelofibrosis (CIMF) regardless of their JAK2 mutational status. The second portion of the study is a Phase 2 study to define the efficacy and safety profile of single agent SB1518 at the recommended dose in subjects with CIMF.

Official Title
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A Phase 1/2 Study of Oral SB1518 in Subjects With Chronic Idiopathic Myelofibrosis

Conditions
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Myelofibrosis, Myeloproliferative Disorders, Polycythemia Vera, Essential Thrombocythemia

Intervention / Treatment
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* Drug: SB1518


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria Subjects with CIMF (including post ET/PV MF) requiring therapy, including: Eastern Cooperative Oncology Group (ECOG) performance status 0-2 Men of reproductive potential who can agree to practice effective contraception during the entire study period and for one month after the last study treatment Women of child-bearing potential who have a negative pregnancy test within 14 days prior to the first dose of study drug and can agree to practice effective contraception during the entire study period and for one month after the last study treatment, unless documentation of infertility exists Subjects who are able to understand and willing to sign the informed consent form Exclusion Criteria Subjects with uncontrolled inter-current illness including, but not limited to, ongoing active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements as judged by treating physician. Subjects receiving antibiotics for infections that are under control may be included in the study unless the antibiotic is a CYP3A4 inducer/inhibitor Subjects known to be HIV-positive Subjects with known active hepatitis A, B, or C, or latent hepatitis B Women who are pregnant or lactating Subjects with prior radiation therapy to more than 20% of the hematopoietic marrow (prior radiation to spleen is allowed)

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: SB1518|SB1518 taken orally daily for 28 consecutive days in a 28-day cycle|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Phase 1: to establish the maximum tolerated dose of SB1518 as a single agent when administered orally daily |  | Throughout the study | 
| Phase 2: to assess the clinical benefit rate in subjects with CIMF who are treated with SB1518 at the recommended dose |  | Throughout the study | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assess the safety and tolerability of SB1518, administered orally once daily in subjects with CIMF |  | Throughout the study | 
| Assess the pharmacokinetic profile of SB1518 |  | Throughout the study | 
| Assess the pharmacodynamic profile of SB1518 |  | Throughout the study | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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SB1518, Chronic Idiopathic Myelofibrosis, Myeloproliferative disorders, Polycythemia Vera, Essential Thrombocythemia, JAK2 inhibitor