84 Participants Needed

INCB000928 + Ruxolitinib for Myelofibrosis

(LIMBER Trial)

Recruiting at 42 trial locations
IC
IC
Overseen ByIncyte Corporation Call Center (ex-US)
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Incyte Corporation
Must be taking: Ruxolitinib
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but if you are in the TGB group, you must have been on a stable regimen of ruxolitinib for at least 12 weeks before starting the study treatment. Other medications, especially those used to treat your disease, may need to be stopped if they were taken within a certain time frame before the study.

What data supports the effectiveness of the drug INCB000928 + Ruxolitinib for Myelofibrosis?

Ruxolitinib, a key component of the treatment, has been shown to be effective for myelofibrosis, as it was the first approved drug for this condition and remains a standard treatment. It works by targeting specific pathways involved in the disease, and its effectiveness has been demonstrated in various studies, including the phase 3 COMFORT-I/II trials.12345

What is known about the safety of ruxolitinib for myelofibrosis?

Ruxolitinib has been used for over a decade to treat myelofibrosis, and studies show it is generally safe, with common side effects being anemia (low red blood cell count) and thrombocytopenia (low platelet count). These side effects rarely lead to stopping the treatment, and no new safety concerns have been identified in recent large studies.13467

What makes the drug INCB000928 + Ruxolitinib unique for treating myelofibrosis?

INCB000928 combined with Ruxolitinib is unique because it represents a new combination approach to treating myelofibrosis, potentially offering benefits beyond Ruxolitinib alone, which has been the standard treatment for over a decade. This combination may target additional pathways involved in the disease, providing an option for patients who do not respond optimally to Ruxolitinib alone.13489

What is the purpose of this trial?

This trial is testing a new drug called INCB000928, either alone or with ruxolitinib, to see if it is safe and effective. It targets people with Myelofibrosis who need regular blood transfusions or have severe anemia. The goal is to see if the drug can reduce the need for transfusions and improve anemia symptoms.

Research Team

AM

Amanda McBride, MD

Principal Investigator

Incyte Corporation

Eligibility Criteria

This trial is for people with myeloproliferative disorders causing anemia who've had prior treatment with JAK inhibitors, are dependent on blood transfusions, and have a life expectancy over 6 months. They must not be pregnant or fathering children and should not have received certain other treatments recently.

Inclusion Criteria

Life expectancy greater than 6 months
I have been treated with JAK inhibitors for over 12 weeks and my condition is rated intermediate-2 or high.
I am fully active or restricted in physically strenuous activity but can do light work.
See 4 more

Exclusion Criteria

I haven't taken any cancer treatments except ruxolitinib for TGB within the last 28 days.
Laboratory Values outside of protocol defined range at screening
I have had or am a candidate for a stem cell transplant.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Participants receive escalating doses of INCB000928 to evaluate safety and tolerability

Approximately up to 13 months

Expansion

Participants receive INCB000928 as monotherapy or in combination with ruxolitinib to further assess efficacy

Approximately up to 13 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • INCB000928
  • ruxolitinib
Trial Overview The study tests INCB000928 alone or combined with ruxolitinib to assess safety, how the body processes it (PK/PD), and effectiveness in treating symptomatic anemia in myelofibrosis patients. It includes dose-finding phases to determine the best amount of drug to give.
Participant Groups
3Treatment groups
Experimental Treatment
Group I: Treatment Group C (TGC)Experimental Treatment2 Interventions
INCB000928 will be administered in combination with ruxolitinib.
Group II: Treatment Group B (TGB)Experimental Treatment2 Interventions
INCB000928 will be administered in combination with ruxolitinib.
Group III: Treatment Group A (TGA)Experimental Treatment1 Intervention
INCB000928 will be administered once daily (QD).

INCB000928 is already approved in United States for the following indications:

🇺🇸
Approved in United States as Zilurgisertib for:
  • Fibrodysplasia ossificans progressiva (Orphan Drug Designation)

Find a Clinic Near You

Who Is Running the Clinical Trial?

Incyte Corporation

Lead Sponsor

Trials
408
Recruited
66,800+
Steven Stein profile image

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot profile image

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

Findings from Research

Ruxolitinib, an oral JAK1 and JAK2 inhibitor, has transformed the treatment of myelofibrosis (MF) since its approval, demonstrating significant clinical benefits in patients as shown in the phase 3 COMFORT-I/II trials.
Over the past 10 years, extensive research on ruxolitinib has enhanced the understanding of MF and its management, paving the way for future combination therapies to improve treatment outcomes.
Ten years after ruxolitinib approval for myelofibrosis: a review of clinical efficacy.Pemmaraju, N., Bose, P., Rampal, R., et al.[2023]
Ruxolitinib has significantly changed the treatment approach for myelofibrosis (MF), but many hematologists in Italy do not use advanced genetic scoring systems for prognosis due to limited access to next-generation sequencing.
Key management practices for ruxolitinib include assessing infection risk before treatment, establishing clinical triggers for therapy regardless of risk category, and implementing a monitoring schedule during the first 12 weeks, highlighting the need for standardized international guidelines.
Tracing the decision-making process for myelofibrosis: diagnosis, stratification, and management of ruxolitinib therapy in real-word practice.Breccia, M., Baratè, C., Benevolo, G., et al.[2023]
Ruxolitinib has been a key treatment for myelofibrosis for over a decade, but the emergence of new JAK inhibitors like fedratinib and pacritinib necessitates a clear understanding of what constitutes 'failure' of ruxolitinib therapy.
A consensus among experts suggests that in cases of ruxolitinib failure, either continuing ruxolitinib with an additional agent or switching to a different JAK inhibitor may be beneficial, highlighting the need for personalized treatment strategies.
Moving beyond ruxolitinib failure in myelofibrosis: evolving strategies for second line therapy.Bose, P., Kuykendall, AT., Miller, C., et al.[2023]

References

Ten years after ruxolitinib approval for myelofibrosis: a review of clinical efficacy. [2023]
Tracing the decision-making process for myelofibrosis: diagnosis, stratification, and management of ruxolitinib therapy in real-word practice. [2023]
Moving beyond ruxolitinib failure in myelofibrosis: evolving strategies for second line therapy. [2023]
A subgroup analysis of JUMP, a phase IIIb, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis in a Brazilian cohort. [2021]
A phase I/II study of the Janus kinase (JAK)1 and 2 inhibitor ruxolitinib in patients with relapsed or refractory acute myeloid leukemia. [2021]
Ten years of treatment with ruxolitinib for myelofibrosis: a review of safety. [2023]
Primary analysis of JUMP, a phase 3b, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis, including those with low platelet counts. [2021]
Analysis of Predictive Factors for Early Response to Ruxolitinib in 320 Patients with Myelofibrosis From the Polish Adult Leukemia Group (PALG) Registry. [2023]
Which patients with myelofibrosis should receive ruxolitinib therapy? ELN-SIE evidence-based recommendations. [2021]
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