Long-Term Side Effects of Ruxolitinib in Treating Patients With Myelofibrosis

Open-Label Roll-Over Study to Assess the Long-Term Safety and Efficacy of Ruxolitinib in Subjects With Myelofibrosis

This phase II trial studies the long-term side effects of ruxolitinib in treating patients with myelofibrosis. Collecting data about the long-term safety and tolerability of ruxolitinib may better help future patients with myelofibrosis.

Study Overview

• Status: Completed
• Conditions: Myelofibrosis
• Intervention / Treatment: Other: Quality-of-Life Assessment; Drug: Ruxolitinib; Other: Long-term Follow-up

Detailed Description

PRIMARY OBJECTIVES:

I. To collect long term safety and tolerability data in patients with myelofibrosis previously treated with ruxolitinib on protocol 2007-0169.

OUTLINE:

Patients continue to receive ruxolitinib orally (PO) once daily (QD) or twice daily (BID). Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo follow-up assessment for safety over 10 minutes every 3 cycles.

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Texas
    • Houston, Texas, United States, 77030
      • M D Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Currently enrolled in study 2007-0169 and benefiting from therapy as determined by treating physician
2. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 at enrollment of this study
3. Ability and agreement to attend protocol-specified visits at the study site
4. Able to comprehend and willing to sign the informed consent form
5. Negative pregnancy test in females of childbearing potential; male patients with female partners of child-bearing potential and female patients of childbearing potential are required to use two forms of acceptable contraception, including one barrier method, during their participation in the study and for 30 days following last dose; acceptable forms of contraception include 1 highly effective method such as an intrauterine device (IUD), hormonal (birth control pills, injections, or implants), tubal ligation, or partner's vasectomy and at least 1 additional approved barrier method such as a latex condom, diaphragm, or cervical cap; female patients of childbearing potential must not be breast-feeding or planning to breast feed and must have a negative pregnancy test 7 days before first study treatment

Exclusion Criteria:

1. none

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment (ruxolitinib, follow-up); Patients continue to receive ruxolitinib PO QD or BID. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo follow-up assessment for safety over 10 minutes every 3 cycles.	Other: Quality-of-Life Assessment; Ancillary studies; Other Names: Quality of Life Assessment; Drug: Ruxolitinib; Given PO; Other Names: INCB-18424; INCB18424; Oral JAK Inhibitor INCB18424; Other: Long-term Follow-up; Undergo follow-up assessment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Most Common Treatment Related Adverse Events	A review of the most common adverse events related to study medication will be performed. Will follow standard reporting guidelines for adverse events. Safety data will be summarized by the number of participants affected.	to the date of failure or death from any cause, Up to 7 years, 3 months and 24 days

Collaborators and Investigators

• Sponsor: M.D. Anderson Cancer Center
• Collaborators: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Prithviraj Bose, M.D. Anderson Cancer Center

Publications and helpful links

Helpful Links

• MD Anderson Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): September 29, 2016
• Primary Completion (Actual): January 23, 2024
• Study Completion (Actual): January 23, 2024

Study Registration Dates

• First Submitted: May 20, 2016
• First Submitted That Met QC Criteria: May 26, 2016
• First Posted (Estimated): May 27, 2016

Study Record Updates

• Last Update Posted (Estimated): August 29, 2025
• Last Update Submitted That Met QC Criteria: August 12, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders; Hemic and Lymphatic Diseases; Primary Myelofibrosis; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; ruxolitinib
• Other Study ID Numbers: 2015-0872 (Other Identifier: M D Anderson Cancer Center); NCI-2016-01181 (Registry Identifier: CTRP (Clinical Trial Reporting Program))

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No