Tolerability of Ropeginterferon Alfa-2b Add-on to Ongoing Ruxolitinib Therapy in Myelofibrosis (RopeRux in Myelofibrosis)

NCT07521046 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: HCI171996
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this clinical trial is to learn if the study drug ropeginterferon alfa- 2b added to, standard of care, ruxolitinib is safe and effective in treating patients with Myelofibrosis.

Conditions

Myelofibrosis

Outcome Measures

Primary Outcomes (5)

• The frequency of adverse events (AEs) and serious adverse events (SAEs) characterized by type

  To assess the safety and tolerability of ropeginterferon alfa- 2b add-on to ruxolitinib in the study population.

  2 years

• The frequency of adverse events (AEs) and serious adverse events (SAEs) characterized by severity (as defined by the NIH CTCAE, version 6.0).

  To assess the safety and tolerability of ropeginterferon alfa- 2b add-on to ruxolitinib in the study population.

  2 years

• The frequency of adverse events (AEs) and serious adverse events (SAEs) characterized by seriousness.

  To assess the safety and tolerability of ropeginterferon alfa- 2b add-on to ruxolitinib in the study population.

  2 years

• The frequency of adverse events (AEs) and serious adverse events (SAEs) characterized by duration.

  To assess the safety and tolerability of ropeginterferon alfa- 2b add-on to ruxolitinib in the study population.

  2 years

• The frequency of adverse events (AEs) and serious adverse events (SAEs) characterized by the relationship to study treatment.

  To assess the safety and tolerability of ropeginterferon alfa- 2b add-on to ruxolitinib in the study population.

  2 years

Secondary Outcomes (6)

• The proportion of subjects who achieve >50% reduction in JAK2 V617F mutation burden.

  2 years

• Change in JAK2, CALR, MPL mutations allelic burden.

  2 years

• The proportion of subjects who achieve a 25% decrease in spleen volume by 24 weeks from initiation of combination treatment.

  24 weeks

• Change in quality of life Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) from baseline and throughout treatment.

  2 years

• The proportion of subjects who progress to blastic phase and secondary acute myeloid leukemia at 2 years post-treatment.

  2 years

Study Arms (1)

Treatment: All Patients

EXPERIMENTAL

This study will investigate the safety and tolerability of ropeginterferon alfa- 2b added on to standard of care ruxolitinib.

Drug: ropeginterferon alfa- 2b; Drug: Ruxolitinib

Interventions

ropeginterferon alfa- 2b DRUG

Ropeginterferon alfa- 2b will be administered as a subcutaneous injection every two weeks.

Treatment: All Patients

Ruxolitinib DRUG

Ruxolitinib will be administered per standard of care.

Treatment: All Patients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subject aged ≥ 18 years.
• Diagnosed with PMF, post-PV MF, or post-ET MF per WHO 2016 or 2022 criteria, bearing one of these MPN phenotype defining mutations (JAK2, CALR, and MPL), and with a DIPSS score of low, intermediate-1 or intermediate-2.
• Subjects must be already on standard of care ruxolitinib per the treating physician for at least 3 months or more, and on a stable dose for at least 6 weeks prior to screening.
• Subjects must have spleen volume of \> 450ml by either MRI or CT scan
• Subject must have a JAK2, CALR, or MPL allelic burden of ≥20% at screening
• Prior treatment for PV or ET with hydroxyurea or ruxolitinib is allowed. If the patient was on pegylated interferon in the past, the progression from PV/ ET to post-PV/ET MF must not have occurred while on pegylated interferon therapy.
• ECOG Performance Status ≤ 2.
• Adequate organ function as defined as:
• Hematologic:
• WBC count ≥ 4 x 109/L
• Absolute neutrophil count (ANC) ≥1500/mm3
• Platelet count ≥ 75,000/mm3
• Hemoglobin ≥ 8 g/dL
• Hepatic:
• Total Bilirubin ≤ 1.5x institutional upper limit of normal (ULN)

You may not qualify if:

• PV or ET patients who progressed while on pegylated interferon or ropeginterferon therapy.
• Receiving other investigational agents.
• Existence of, or history of severe psychiatric disorders, particularly severe depression, suicidal ideation, or suicide attempt (Patients with pre-existing depression who are well-controlled and on stable doses of antidepressants are eligible).
• Evidence of severe retinopathy or clinically significant eye disease.
• History or presence of active serious or untreated autoimmune disease.
• History of solid organ transplant.
• Liver cirrhosis Child-Pugh score B or C. -≥ 5% blasts in peripheral blood or bone marrow.
• Prior systemic anti-cancer therapy or any investigational therapy ≤ 14 days or within five half-lives prior to starting study treatment, whichever is shorter.
• Major surgery 4 weeks prior to starting study drug or who have not fully recovered from major surgery.
• The diagnosis of another malignancy which, in the investigator's opinion, is likely to significantly impact study participation.
• Current evidence of uncontrolled, significant intercurrent illness including, but not limited to, the following conditions:
• Cardiovascular disorders:
• Uncontrolled hypertension, in the opinion of the investigator
• Congestive heart failure New York Heart Association Class II or greater, unstable angina pectoris, serious cardiac arrhythmias.
• Stroke or myocardial infarction within the past 3 months

Sponsors & Collaborators

• PharmaEssentia: collaborator
• University of Utah: lead

Study Sites (1)

Huntsman Cancer Institute at University of Utah

Salt Lake City, Utah, 84112, United States

Location

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Nicole Fisher

CONTACT

801-587-7604; nicole.fisher@hci.utah.edu

Tsewang Tashi, MD

CONTACT

801-585-0255; u0820872@utah.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2026
• First Posted: April 9, 2026
• Study Start: May 1, 2026
• Primary Completion (Estimated): May 1, 2029
• Study Completion (Estimated): May 1, 2030
• Last Updated: April 17, 2026

Record last verified: 2026-04

Locations

US (1)