NCT03427866

Ruxolitinib Pre-, During- and Post-HSCT for Patients With Primary or Secondary Myelofibrosis.

A Phase II Study of Ruxolitinib Pre-, During- and Post-Hematopoietic Stem Cell Transplantation for Patients With Primary or Secondary Myelofibrosis.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 44 (actual)
• Sponsor: Massachusetts General Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This research study is studying a drug called Ruxolitinib as a possible treatment for Myelofibrosis.

Detailed description

This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means that the drug is being studied. The FDA (the U.S. Food and Drug Administration) has approved Ruxolitinib as a treatment option for this disease. This study examines two different cohorts of participants: * Cohort 1: Participants who are eligible for Ruxolitinib therapy before transplant, based on their platelet counts. These participants will receive their first dose of the study drug between 2 and 6 months before HCT. * Cohort 2: Participants who are not eligible for Ruxolitinib therapy pre-treatment based on their platelet counts. These participants will receive their first dose of Ruxolitinib 1 week before the conditioning period. Ruxolitinib is a medication that blocks certain proteins called tyrosine kinases. Specifically, it blocks tyrosine kinases called JAK2. Many cancers have over active "cell signaling." What this means is that certain functions in the cancer cells never turn off and this makes them grow in an uncontrolled way. Ruxolitinib, shuts down the pathway that depends on the JAK2 tyrosine kinases. The JAK2 pathway is over active in the participant's disease, acute myeloid leukemia. The exact way ruxolitinib does this is not yet clear but it may have to do with its ability to block the JAK2 pathway since this pathway can also lead to inflammation in the body. Ruxolitinib has also been shown to lower the rates of Graft-Versus-Host-Disease (GVHD), a complication of transplant. GVHD is a disease that occurs when the immune cells in transplanted donor tissue from your HCT attack the participant's own tissues and organs. There are two types of GVHD: acute and chronic. Acute GVHD generally occurs within 1 week to 3 months after your HCT and may affect your skin, intestines, and liver. Chronic GVHD begins later on and may affect the organs prone to acute GVHD complications, as well as the lungs, mucous membranes, or other organs. There is also evidence that ruxolitinib is associated with reduced instances of enlarged spleen size after HCT. Enlarged spleens play a role in the engraftment rate after HCT, which is the rate at which donated tissue and your own tissue begin reproducing and growing together. In this research study, the investigators are: * assessing the efficacy (how well the study drug works) and tolerability of Ruxolitinib before, during, and after HCT. * examining the rates of GVHD after HCT when ruxolitinib is administered. * determining whether engraftment rates improve when ruxolitinib is given

Conditions

• Myelofibrosis

Interventions

Type	Name	Description
DRUG	Ruxolitinib	Ruxolitinib is a medication that blocks certain proteins called tyrosine kinases. Specifically, it blocks tyrosine kinases called JAK2. The JAK2 pathway is over active in the disease, acute myeloid leukemia.

Timeline

• Start date: 2018-08-28
• Primary completion: 2024-05-01
• Completion: 2025-05-19
• First posted: 2018-02-09
• Last updated: 2025-07-29

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study