Continued Itacitinib Treatment for Myelofibrosis

Recruiting at 27 trial locations

Overseen By Incyte Corporation Call Center (ex-US)

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Incyte Corporation

Must be taking: Itacitinib

Disqualifiers: Uncontrolled illness, Pregnant, 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

Compensation: Varies

Number of Visits: Unknown

Duration: As long as tolerated and beneficial

18 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on providing continued treatment with itacitinib, a medication under study for myelofibrosis, a type of bone marrow cancer that disrupts blood cell production. The trial aims to maintain benefits for those already receiving itacitinib in a previous study and still finding it helpful. To join, participants must be currently enrolled in an Incyte-sponsored study of itacitinib and still benefiting from the treatment. As a Phase 2 trial, the research measures how well the treatment works in an initial, smaller group, offering valuable insights into its effectiveness.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, since this study is a continuation of previous itacitinib treatment, it is likely that you can continue your current regimen if it was part of the original study.

Is there any evidence suggesting that itacitinib is likely to be safe for humans?

Research shows that itacitinib is generally safe for patients. Previous studies found no new safety issues with itacitinib, whether used alone or with other treatments. Participants in these studies reported no unexpected side effects. This suggests that itacitinib might be safe for ongoing use. However, like any medication, monitoring for possible side effects is important. Always consult a doctor if there are concerns about joining a clinical trial. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for myelofibrosis?

Unlike the standard treatments for myelofibrosis, which often include medications like ruxolitinib that primarily target and inhibit JAK2 enzymes, itacitinib is unique because it specifically targets the JAK1 enzyme. This selective mechanism of action may reduce certain side effects associated with broader JAK inhibition, potentially offering a more tolerable treatment option. Researchers are excited about itacitinib because it might provide symptom relief and improve quality of life for patients who do not respond well to existing therapies.

What is the effectiveness track record for itacitinib in treating myelofibrosis?

Research has shown that itacitinib, the treatment under study in this trial, may help treat myelofibrosis, a type of bone marrow disorder. In one study, two out of ten patients had a noticeable decrease in spleen size after 24 weeks. Another study found that some patients experienced a 50% reduction in symptoms related to myelofibrosis after just 12 weeks. Itacitinib has also been used to prevent complications in other conditions, suggesting it might be safe and effective. While more research is needed, these findings highlight the potential benefits of itacitinib. ^{3 6 7 8 9}

Who Is on the Research Team?

Lance Leopold, MD

Principal Investigator

Incyte Corporation

Are You a Good Fit for This Trial?

This trial is for patients already taking itacitinib in other Incyte-sponsored studies, who are benefiting and tolerating the treatment well. They must understand and agree to study procedures, avoid pregnancy or fathering children, and not have any illness that could risk their safety or study compliance.

Inclusion Criteria

Has demonstrated compliance, as assessed by the investigator, with the parent protocol requirements

Willingness to avoid pregnancy or fathering children

Ability to comprehend and willingness to sign an ICF

Exclusion Criteria

Pregnant or breastfeeding women

Participants with an uncontrolled intercurrent illness or any concurrent condition that, in the investigator's opinion, would jeopardize the safety of the participant or compliance with the Protocol

I can get itacitinib through a pharmacy or my doctor.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive continued treatment with itacitinib as per the treatment dose and schedule from their original study

As long as tolerated and beneficial

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 years

What Are the Treatments Tested in This Trial?

Interventions

• itacitinib

Trial Overview The trial provides ongoing access to itacitinib for participants from previous related studies. It's an open-label study where all involved know they're receiving itacitinib at doses and schedules set by earlier trials as long as benefits continue and side effects remain manageable.

How Is the Trial Designed?

Group I: itacitinib Experimental Treatment 1 Intervention

Participants will receive treatment with itacitinib as per the treatment dose and schedule they received in the study in which they were originally enrolled. Participants who are receiving ruxolitinib under parent protocol INCB39110-209 may continue to receive it as described in that protocol.

Who Is Running the Clinical Trial?

Incyte Corporation

Lead 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

Published Research Related to This Trial

Myelofibrosis (MF) is a serious blood cancer with varying prognoses; patients with low-risk disease can survive 6-15 years, while those with high-risk disease or transformed acute myeloid leukemia (AML) have a much poorer prognosis, often less than 3 months.

The only curative treatment for MF is allogeneic stem cell transplantation, but since most patients are ineligible, they typically receive older drugs for symptom relief. However, new JAK1/JAK2 inhibitors like ruxolitinib show promise based on positive results from large phase III studies, indicating a potential shift in treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical and laboratory features of myelofibrosis and limitations of current therapies. Gregory, SA., Mesa, RA., Hoffman, R., et al.[2022]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ten years after ruxolitinib approval for myelofibrosis: a review of clinical efficacy. Pemmaraju, N., Bose, P., Rampal, R., et al.[2023]

Selective JAK2 inhibitors have shown significant efficacy in reducing symptoms and splenomegaly in myelofibrosis patients, providing benefits that previous treatments could not achieve, although they may cause anemia and gastrointestinal issues.

The development of enhanced prognostic models is improving the ability to assess individual patient outcomes in myelofibrosis, which is crucial for balancing the benefits of new therapies against the risks of more invasive treatments like stem cell transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessing new therapies and their overall impact in myelofibrosis. Mesa, RA.[2016]

In a study of 703 myelofibrosis patients, 31.2% discontinued ruxolitinib, and 27.4% of those were successfully rechallenged, showing significant improvements in spleen size and symptoms during the treatment.

Patients who were rechallenged with ruxolitinib had a median overall survival of 27.9 months, which was significantly longer compared to those who permanently discontinued the drug, indicating a potential survival benefit from rechallenging.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ruxolitinib rechallenge in resistant or intolerant patients with myelofibrosis: Frequency, therapeutic effects, and impact on outcome. Palandri, F., Tiribelli, M., Breccia, M., et al.[2022]

Citations

1. sciencedirect.com sciencedirect.com/science/article/abs/pii/S014521262500092X

A phase 2 study of itacitinib alone or in combination with ... Two of 10 patients on itacitinib alone had ≥ 20 % spleen volume reduction at week 24. •. No new safety signals observed with itacitinib alone or combined with ...

2. clinicaltrials.gov clinicaltrials.gov/show/NCT01633372

An Open Label Study of Itacitinib Administered Orally in ... This is a study of itacitinib (INCB039110) in patients with myelofibrosis. This study will evaluate safety and efficacy parameters of itacitinib (INCB039110).

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5286940/

Primary analysis of a phase II open-label trial of INCB039110 ... Although the 100 mg twice-daily cohort was not selected for expansion, two patients experienced a ≥50% reduction in myelofibrosis-related symptoms at week 12, ...

4. ashpublications.org ashpublications.org/blood/article/145/13/1382/526136/Itacitinib-for-prevention-of-graft-versus-host

Itacitinib for prevention of graft-versus-host disease and ... Itacitinib was extremely effective at preventing grade 2 to 4 CRS and grade 3/4 aGVHD, with no clear increase in relapse rate. Itacitinib was ...

5. rarediseaseadvisor.com rarediseaseadvisor.com/news/more-research-identify-best-itacitinib-ruxolitinib-dose-ratio-mf/

Research Needed to Identify Best Itacitinib-to-Ruxolitinib ... At week 12, 9 patients treated with itacitinib plus ruxolitinib and 7 patients treated with itacitinib alone achieved spleen volume reduction.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04629508

Study Details | NCT04629508 | To Assess the Safety, ... A 2-Part, Phase 2, Open-Label Study of the Safety, Tolerability, and Efficacy of Itacitinib Immediate Release in Participants With Primary Myelofibrosis or ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40516236/

A phase 2 study of itacitinib alone or in combination with ... Conclusions: Overall, 8 of 23 patients enrolled achieved SVR at week 24; larger average changes in SVR at week 12 were observed for itacitinib ...

8. hematologyadvisor.com hematologyadvisor.com/news/itacitinib-ruxolitinib-dose-ratio-myelofibrosis-more-research-treatment-risk/

Research Needed to Identify Best Itacitinib-to-Ruxolitinib ... A week 12, the mean percentage spleen volume reduction was 1.6% in patients treated with itacitinib plus ruxolitinib and 24.6% for those treated ...

9. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/279557

To Assess the Safety, Tolerability and Efficacy of Itacitinib ... To Assess the Safety, Tolerability and Efficacy of Itacitinib Immediate Release Tablets in Participants With Primary or Secondary Myelofibrosis Who Have ...