A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 in Participants With Myelofibrosis

1. Current treatment with a JAK inhibitor (e.g. ruxolitinib or fedratinib) or a history of treatment with a JAK inhibitor within two weeks of enrolment.

2. Positive hepatitis panel and/or positive HIV test.

3. Any concurrent severe and/or uncontrolled medical conditions that could increase the participant's risk for toxicity while in the study or that could confound discrimination between disease- and study treatment-related toxicities. Any planned major surgery during the study period

4. Impaired cardiac function or clinically significant cardiac diseases, including any of the following:

1. History or presence of ventricular tachyarrhythmia.

3. Clinically significant resting bradycardia (\< 50 bpm) and use of a cardiac pacemaker or implantable cardioverter defibrillator.

4. Angina pectoris or acute myocardial infarction ≤ 90 days prior to starting study drug.

5. Other clinically significant heart disease (e.g., symptomatic congestive heart failure; uncontrolled arrhythmia or hypertension; history of labile hypertension or poor compliance with an antihypertensive regimen).

5. Participants who are currently receiving chronic (\> 14 days) treatment with corticosteroids at a dose \> 10 mg of prednisone (or its glucocorticoid equivalent) per day, or any other chronic immunosuppressive treatment that cannot be discontinued prior to starting study drug.

6. Participants with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of GB2064 as per physician's opinion.

7. Participants who received radiotherapy within the last month prior to screening procedures, or patients who received splenectomy in the previous three months or are scheduled for the procedure in the next three months.

8. Participants who had a history of malignancy in the past 3 years, except for treated early stage squamous, basal cell carcinoma or treated, localised prostate cancer.

9. Presence of clinically meaningful active bacterial, fungal, parasitic or viral infection which requires therapy.

10. Previous history of Progressive Multifocal Leuko-encephalopathy (PML).

11. Pregnant or breast feeding (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive β- HCG laboratory test.

12. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 90 days after study treatment. Highly effective contraception methods must be used.

13. Sexually active males must use a condom during intercourse while taking the drug and for 90 days after stopping study drug and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.

14. Hypersensitivity to GB2064 and/or its excipients.

15. Participants unable or unwilling to comply with protocol requirements.

16. Participants related to PI/site staff.

17. Participants who have had a hematopoietic stem cell transplantation.

18. Participants who are eligible, have a donor and are willing to undergo a hematopoietic stem cell transplantation.

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 in Participants With Myelofibrosis

What this study is about

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 in Participants With Myelofibrosis

What this study is about