NCT04281498

Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation

A Phase II Open-label Study of Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: John Mascarenhas · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Accepted

Summary

The presence of IDH mutation is associated with worse survival in patients with myelofibrosis. Moreover IDH mutations are among the most frequently encountered events in MPNs that have progressed to acute myeloid leukemia. Ruxolitinib, a JAK1/2 inhibitor, and enasidenib an IDH2 inhibitor are effective and tolerable treatments for patients with myelofibrosis (MF) and acute myeloid leukemia (AML), respectively. The study team hypothesize that the combination of these agents in patients with MPN with an IDH2 mutation will improve the overall clinical response to therapy.

Detailed description

At this time, there is no standard medical treatment for MPN-AP/BP and most patients with accelerated and blast phase MPN do not respond well to treatment This is a phase II open-label study to evaluate the safety and efficacy of combined ruxolitinib and enasidenib in patients with accelerated/blast-phase myeloproliferative neoplasm or chronic phase myelofibrosis with high risk features and IDH2 mutation. Ruxolitinib (Jakafi/Jakavi) is FDA approved for myelofibrosis and was shown to reduce splenomegaly and improve symptoms. Enasidenib is a potent inhibitor of the IDH2 mutant enzyme and is FDA approved for relapsed refractory AML where it showed effectivity. Pre-clinical studies indicate increased disease mitigating effects with the combination of enasidenib and ruxolitinib. This study will enroll up to 32 patients. Ruxolitinib and enasidenib will be given orally in 28-day cycles.

Conditions

• Accelerated/Blast-phase Myeloproliferative Neoplasm
• Chronic-phase Myelofibrosis
• IDH2 Mutation

Interventions

Type	Name	Description
DRUG	Ruxolitinib	Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
DRUG	Enasidenib	50mg -100mg daily

Timeline

• Start date: 2021-01-14
• Primary completion: 2023-05-30
• Completion: 2023-05-30
• First posted: 2020-02-24
• Last updated: 2025-02-04
• Results posted: 2024-12-18

Locations

11 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study