CompletedPhase 2ACTRN12611000159954

Safety and Efficacy Study of CYT387 in Primary Myelofibrosis (PMF) or Post-polycythemia Vera (PV) or Post-essential Thrombocythemia (ET)

A Phase I/II, Open-Label, Dose-Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Orally-Administered CYT387 in Primary Myelofibrosis or Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis.

Sponsor

Gilead Sciences Inc.

Enrollment

140 participants

Start Date

Aug 31, 2011

Study Type

Interventional

Conditions

Primary Myelofibrosis

Summary

This is an open-label, non-randomized, dose-escalation study, to be conducted in two phases: a single-centre dose-escalation phase with supernumerary patient addition (Part 1), to determine the safety and tolerability of CYT387, and to identify a therapeutic dose for the confirmation portion of the study; and a multiple-centre dose-confirmation phase (Part 2), which will be a cohort expansion at or below the maximum tolerated dose (MTD) of CYT387.

Eligibility

Sex: Both males and femalesMin Age: 18 Yearss

Plain Language Summary

Simplified for easier understanding

This study tests a drug called CYT387 for people with a rare blood cancer called myelofibrosis. It is for adults aged 18 and older with intermediate or high-risk disease. People with active infections, significant heart problems, HIV, hepatitis, or who are pregnant or breastfeeding cannot participate.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

In the Phase I dose-escalation phase of the study, patients were assigned to dose levels in successive cohorts starting with a dose in the first cohort of 100 mg/day. Dose-escalation proceeded initia

In the Phase I dose-escalation phase of the study, patients were assigned to dose levels in successive cohorts starting with a dose in the first cohort of 100 mg/day. Dose-escalation proceeded initially with a 1.5-fold increment, however, based on toxicity and efficacy information at a specific dose level, the dose escalation increment may have been reduced to a 1.25-fold escalation at the discretion of the investigator. At any dose level, if one patient experienced a Grade 2 toxicity or higher, the dose-escalation could only proceed with 1.25-fold increments. The dose escalation proceeded as follows: Cohort 1 = 100 mg Cohort 2 = 150 mg Cohort 3 = 200 mg Cohort 4 = 300 mg Cohort 5 = 400 mg CYT387 will be orally self-administered as a single daily dose beginning on Day 1 of the study, and thereafter at approximately the same time each day of the 28-day cycle. It is recommended that all doses be preceded by a 2-hour fast from food and beverages, and be followed by a 1-hour post-dose fast from food and beverages. Patients will receive up to 9 cycles of therapy unless one of the following occurs: - disease progression (Progressive disease; PD) by IWG-MRI consensus criteria - Intercurrent illness that prevents further administration of treatment - Unacceptable adverse event(s) - Patient decides to withdraw from the study - General or specific changes in the patient's condition render the patient unacceptable for further treatment in the judgment of the investigator Twenty additional patients will be assigned to a 150 mg BID (twice daily) dosing schedule. CYT387 will be orally self-administered twice-daily with doses approximately 10-12 hours apart beginning on Day 1 of the study, and thereafter at approximately the same times each day of the 28-day cycle. Patients will continue for up to 9 cycles as above. For the Part 2 dose confirmation portion of the study, 60 additional patients will be assigned to either 150 mg or 300 mg QD (once daily) dose groups. As with Part 1 of the study, patients will receive up to 9 cycles of CYT387, at 28 days per cycle as above.