For COVID-19 vaccine updates, please review our information guide. For patient eligibility and scheduling availability, please visit VaccineTogetherNY.org.

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Navitoclax in Combination with Ruxolitinib Versus Ruxolitinib in Subjects with Myelofibrosis (TRANSFORM-1)

Clinical Trial Details

Myelofibrosis is a blood cancer that causes scarring in the bone marrow leading to a change in the production of blood cells in the body. A majority of people with myelofibrosis have a mutation of the Janus-associated kinase-2 (JAK-2) gene. This mutation causes the body to produce the wrong number and type of blood cells. 

This study is looking at the potential of comparing the drug Ruxolitinib in combination with navitoclax versus Ruxolitinib alone for the treatment of myelofibrosis. Ruxolitinib is an oral medication that inhibits the JAK-2 signaling pathway and works by blocking certain enzymes in the body that affect blood cell production. Navitoclax is an oral protein inhibitor which may prevent fibrosis in the bone marrow.

Ruxolitinib is approved by the U.S. Federal Drug Administration (FDA) for the treatment of myelofibrosis, however, ruxolitinib is not currently approved to treat myelofibrosis in combination with navitoclax. Navitoclax is considered experimental, meaning it is not yet approved by the FDA for the treatment of myelofibrosis.

Participants will be randomly assigned by chance to receive one of the following treatments:
   • Arm A: Approximately 115 patients will receive navitoclax once daily and ruxolitinib twice daily.
   • Arm B: Approximately 115 patients will receive ruxolitinib twice daily and a placebo once daily.

A placebo is not a drug, it is not expected to have any chemical effects on your body, and it is not designed to treat any disease or illness. It looks like the study drug to ensure patients and the study staff cannot guess what you are taking. This allows the study scientists to make the best judgment on whether the study drug is having effects that are greater than are expected by chance alone. The combination of navitoclax with ruxolitinib may or may not work better than ruxolitinib with a placebo.

This is a double-blinded study, which means neither you nor your study doctor will know if you were assigned navitoclax or placebo. In case of emergency, your study doctor can find out this information. Neither you nor your study doctor will be able to pick which study drug you receive.

The starting dose for navitoclax and ruxolitinib will be chosen by the study doctor depending on a patients platelet count, the cells in the blood that stop bleeding. The study doctor may decrease the dose if a participant has side effects or is unable to tolerate the prescribed dose or may increase to a higher dose if the response is not as expected.

Key Eligibility: 
  • A documented diagnosis of primary myelofibrosis or secondary myelofibrosis (post polycythemia vera [PPV]-MF or post essential thrombocythemia [PET] – MF) as defined by the World Health Organization classification
  • At least 2 symptoms as measured by the Myelofibrosis Symptom Assessment Form, such as fatigue, early satiety, abdominal discomfort, night sweats, and bone pain.
  • Subject must not have received prior treatment with a JAK2 inhibitor

Detailed eligibility criteria will be reviewed with the study team.

Study contact by location

Upper East Side - Manhattan

Contact(s)

Tania J. Curcio, NP, FNP-BC
(212) 746-2571
tjc9003@med.cornell.edu

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:

2010022760

ClinicalTrials.gov:

NCT04472598

Sponsor:

Abbvie M16-191

Status

Open to Enrollment

Age Group

Adult

Sponsor