Ruxolitinib for Myelofibrosis

Recruiting at 4 trial locations

Overseen By Gabriela Hobbs, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Massachusetts General Hospital

Must be taking: Ruxolitinib

Must not be taking: Strong CYP3A4 inhibitors

Disqualifiers: Prior transplant, Heart failure, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 56

44 Participants Needed

Trial Summary

What is the purpose of this trial?

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

Research Team

Gabriela Hobbs, MD

Principal Investigator

Massachusetts General Hospital

Eligibility Criteria

This trial is for adults aged 18-75 with primary or secondary myelofibrosis, who are either already taking Ruxolitinib or will start it. They must have symptoms like an enlarged spleen and meet certain blood count criteria. Participants should be fit enough for a stem cell transplant from a matched donor and not have severe organ damage, uncontrolled infections, heart failure, other recent cancers (except some skin cancers), or be pregnant.

Inclusion Criteria

I am eligible for or already on ruxolitinib for my enlarged spleen or symptoms, and my spleen has not grown >5 cm since starting treatment.

You are expected to live for at least 3 more months.

I am not on any treatment for myelofibrosis except ruxolitinib.

Exclusion Criteria

You have an allergy or extreme sensitivity to any medication that belongs to the JAK inhibitor family.

My blood or bone marrow had a rapid increase in immature cells before my cell transplant.

I have an infection that is not under control.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-Transplant Treatment

Participants receive Ruxolitinib therapy before hematopoietic stem cell transplantation (HCT) based on their eligibility

2-6 months

Continuous dosing every 28 days

Post-Transplant Treatment

Participants receive Ruxolitinib therapy after HCT to assess efficacy and tolerability, and to examine GVHD rates

Up to 2 years

Continuous dosing every 28 days

Follow-up

Participants are monitored for GVHD free and relapse free survival, overall survival, and other outcomes

1-2 years

Treatment Details

Interventions

• Ruxolitinib

Trial Overview The study tests the drug Ruxolitinib in patients with myelofibrosis before, during, and after they receive a hematopoietic stem cell transplant (HSCT). The goal is to see how well Ruxolitinib works as part of the treatment process for this condition.

Participant Groups

Group I: Ruxolitinib Not Eligible pre-HSCT Experimental Treatment 1 Intervention

* Ruxolitinib will be taken orally at a fixed dose twice every day after transplant * Dosing will be continuous, with a new cycle scheduled to start every 28 days. * There will be no break in dosing between cycles * Ruxolitinib can be administered with or without food.

Group II: Ruxolitinib Eligible pre-HSCT Experimental Treatment 1 Intervention

* Ruxolitinib will be taken orally at a fixed dose twice every day * Dosing will be continuous, with a new cycle scheduled to start every 28 days. * There will be no break in dosing between cycles * Ruxolitinib can be administered with or without food.

Ruxolitinib is already approved in United States, European Union for the following indications:

Approved in United States as Jakafi for:

• Intermediate or high-risk myelofibrosis
• Polycythemia vera
• Steroid-refractory acute graft-versus-host disease
• Chronic graft-versus-host disease
• Vitiligo

Approved in European Union as Jakavi for:

• Intermediate or high-risk myelofibrosis
• Polycythemia vera
• Steroid-refractory acute graft-versus-host disease
• Chronic graft-versus-host disease
• Non-segmental vitiligo

Who Is Running the Clinical Trial?

Massachusetts General Hospital

Lead Sponsor

Trials

3,066

Recruited

13,430,000+

Incyte Corporation

Industry Sponsor

Trials

408

Recruited

66,800+

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School