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.
Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms |
Yes |
Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies. |
Interventional |
Eligible Ages | 18 Years and Over |
Gender | All |
Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries. |
NCT04281498 |
Phase
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use. |
Phase 2 |
Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data. |
John Mascarenhas |
Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study. |
John Mascarenhas, MDRuben Mesa, MDRonald Hoffman, MDMichal Bar-Natan, MD |
Principal Investigator Affiliation | Icahn School of Medicine at Mount SinaiMays Cancer Center at UT HealthIcahn School of Medicine at Mount SinaiIcahn School of Medicine at Mount Sinai |
Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial. |
Other, Industry, NIH |
Overall Status | Recruiting |
Countries | Canada, United States |
Conditions
The disease, disorder, syndrome, illness, or injury that is being studied. |
Accelerated/Blast-phase Myeloproliferative Neoplasm, Chronic-phase Myelofibrosis, IDH2 Mutation |
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.
Experimental: Patients with MPN
Ruxolitinib and Enasidenib combination therapy
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
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
Status
Not yet recruiting
Address
Mayo Clinic - Arizona
Scottsdale, Arizona, 85259
Status
Not yet recruiting
Address
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Status
Not yet recruiting
Address
Moffitt Cancer Center
Tampa, Florida, 33612
Status
Not yet recruiting
Address
University of Kansas Cancer Center
Westwood, Kansas, 66205
Status
Not yet recruiting
Address
University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, 48109-5936
Status
Recruiting
Address
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Status
Not yet recruiting
Address
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
Status
Not yet recruiting
Address
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27157
Status
Recruiting
Address
Taussig Cancer Center Institute
Cleveland, Ohio, 44195
Status
Recruiting
Address
Mays Cancer Center at UT Health San Antonio
San Antonio, Texas, 78229
Status
Not yet recruiting
Address
Princess Margaret Cancer Centre
Toronto, , M5G 2M9