A Phase 3, Randomized, Double-blind, Add-on Study Evaluating the Safety and Efficacy of Navtemadlin Plus Ruxolitinib vs Placebo Plus Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Kartos Therapeutics, Inc.
- Enrollment
- 600
- Locations
- 214
- Primary Endpoint
- To compare total symptom score reduction (TSS50) between Arm 1 and Arm 2
Study Overview
Brief Summary
This clinical trial is evaluating whether addition of navtemadlin to ruxolitinib treatment will provide more clinical benefit than ruxolitinib alone for patients with Myelofibrosis who have a suboptimal response to ruxolitinib treatment alone.
Subjects will start by receiving ruxolitinib alone in the run-in period. Those who demostrate a suboptimal response from ruxolitinib alone will then be randomized 2:1 to receive navtemadlin or navtemadlin placebo as add-on treatment to their ongoing ruxolitinib. Randomized means that subjects will be assigned to a group by chance, like a flip of a coin. The study is blinded, meaning the subjects, doctors, central endpoint assessors and sponsor will not know which add on treatment (navtemadlin or navtemadlin placebo) the subject is receiving.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Placebo-controlled
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Arm 1
Navtemadlin administered orally once daily on Days 1-7, off treatment on Days 8-28, in 28-day treatment cycles. Ruxolitinib administered orally twice a day.
Intervention: Navtemadlin (Drug)
Arm 1
Navtemadlin administered orally once daily on Days 1-7, off treatment on Days 8-28, in 28-day treatment cycles. Ruxolitinib administered orally twice a day.
Intervention: Ruxolitinib (Drug)
Arm 2
Navtemadlin placebo administered orally once daily on Days 1-7, off treatment on Days 8-28, in 28-day treatment cycles. Ruxolitinib administered orally twice a day.
Intervention: Navtemadlin placebo (Drug)
Arm 2
Navtemadlin placebo administered orally once daily on Days 1-7, off treatment on Days 8-28, in 28-day treatment cycles. Ruxolitinib administered orally twice a day.
Intervention: Ruxolitinib (Drug)
Outcomes
Primary Outcomes
To compare total symptom score reduction (TSS50) between Arm 1 and Arm 2
Time Frame: 24 weeks
The proportion of subjects in each arm with a TSS50 by the Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 24 weeks after start of the randomized period
To compare spleen volume reduction (SVR35) between Arm 1 and Arm 2
Time Frame: 24 weeks
The proportion of subjects in each arm with SVR35 by MRI/CT scan (central review) 24 weeks after start of the randomized period
Secondary Outcomes
- To compare time to progression between Arm 1 and Arm 2(Up to 8 years)
- To compare overall survival (OS) between Arm 1 and Arm 2(Up to 8 years)