Phase 2
N=6
Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation
Accelerated/Blast-phase Myeloproliferative Neoplasm · Chronic-phase Myelofibrosis · IDH2 Mutation
Bottom Line
View on ClinicalTrials.gov: NCT04281498 ↗Enrolled (actual)
6
Serious AEs
16.7%
Results posted
Dec 2024
Primary outcome: Primary: Number of MPN Participants With Response — 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ruxolitinib (Drug); Enasidenib (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- John Mascarenhas
- Primary completion
- May 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of MPN Participants With Response |
2 | — |
| SECONDARY Number of MPN Participants With Blast Response |
3 | — |
| SECONDARY Number of MF-CP Participants With Any Response |
— | — |
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.
Eligibility Criteria
INCLUSION CRITERIA
- Subjects must be ≥ 18 years at the time of signing the Informed Consent Form (ICF).
- Understanding and voluntary signing an IRB-approved informed consent form.
- Diagnosis of:
- Accelerated-phase (≥ 10% blasts in PB or BM) or blast-phase (≥ 20% blasts in PB or BM) myeloproliferative neoplasm (with history of prior myelofibrosis, polycythemia vera, or essential thrombocythemia)
- Previously treated patients with myelofibrosis with persistent disease or progressive disease (persistent or progressive splenomegaly, leukocytosis, anemia, or thrombocytopenia) with intermediate-1 or greater risk disease according to 2013 International Working Group (IWG) criteria, and 4-9% circulating blasts.
- Demonstration of an IDH2 mutation.
- Platelet count > 75, 000 X 109/L for chronic phase myelofibrosis patients.
- Prior therapy with either ruxolitinib or enasidenib is permitted, but not a combination of ruxolitinib and enasidenib.
- Patients with chronic phase myelofibrosis on ruxolitinib must be on the drug for at least 3 months and on a stable dose for at least one month.
- Subjects must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2. ECOG 3 status will be allowed if attributable to MPN.
- Patients must have adequate organ function as demonstrated by the following: a. Direct bilirubin 450 ms
All inclusion and exclusion criteria will be reviewed by the Investigator or qualified designee to ensure that the patient qualifies for the trial.
Data sourced from ClinicalTrials.gov (NCT04281498). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.