INCB000928 for Myelodysplastic Syndrome

(LIMBER Trial)

Recruiting at 18 trial locations

Overseen By Incyte Corporation Call Center (ex-US)

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Incyte Corporation

Must not be taking: ESAs, Immunomodulatory drugs

Disqualifiers: Stem cell transplant, Major surgery, Cardiac disease, Chronic liver disease, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Approximately up to 7 months

22 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests a new drug, INCB000928, for patients with MDS or MM who need regular blood transfusions or have severe anemia. The goal is to see if the drug can improve blood cell counts and reduce the need for transfusions by acting on specific pathways in the body.

Will I have to stop taking my current medications?

Yes, you may need to stop taking certain medications before joining the trial. Specifically, you cannot have had chemotherapy, immunomodulatory drugs, or other specified treatments within 28 days or 5 half-lives before starting the study drug. Also, you cannot be on certain other medications like strong CYP3A4/5 inhibitors or inducers during the study.

What makes the drug INCB000928 unique for treating myelodysplastic syndrome?

INCB000928, also known as Zilurgisertib, is a novel treatment option for myelodysplastic syndrome (MDS) that targets both the malignant blood cells and their surrounding environment, potentially overcoming resistance to current therapies. This approach is part of a new wave of treatments that aim to improve patient outcomes by addressing multiple aspects of the disease. ^{1 2 3 4 5}

Research Team

Ekatarine Asatiani, MD

Principal Investigator

Incyte Corporation

Eligibility Criteria

This trial is for adults with Myelodysplastic Syndromes (MDS) or Multiple Myeloma (MM) who need regular blood transfusions or have anemia symptoms. MDS patients must have tried other anemia treatments without success and can't be on certain therapies. MM patients should have already tried standard treatments without success. Participants must not be pregnant, planning to become pregnant, or father a child.

Inclusion Criteria

Agreement to avoid pregnancy or fathering children

I have MDS and only use hydroxyurea for treatment.

I need regular blood transfusions or have symptoms of anemia.

Exclusion Criteria

I haven't taken any blood cell-boosting drugs in the last 28 days.

I have a serious heart condition that is not under control.

You have had or currently have a significant heart rhythm problem as determined by the study doctor.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive INCB000928 as monotherapy to evaluate safety, tolerability, PK, PD, and efficacy

Approximately up to 7 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• INCB000928

Trial Overview The study tests INCB000928's safety and how well it works as a single therapy in people with MDS or MM who are dependent on transfusions or suffer from symptomatic anemia. It's a Phase 1/2 trial where researchers will also look at how the body processes the drug and its effects on the disease.

Participant Groups

Group I: INCB000928 Experimental Treatment 1 Intervention

INCB000928 will be administered in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.

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

Approved in United States as Zilurgisertib for:

• Fibrodysplasia ossificans progressiva (Orphan Drug Designation)

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

Findings from Research

Myelodysplastic syndrome (MDS) is a complex blood disorder with a significant risk of progressing to acute myeloid leukemia (AML) and currently lacks a proven curative treatment.

Emerging biological therapies that target both the cancerous blood cells and their surrounding environment show promise in overcoming resistance to existing treatments and may improve patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New options in the treatment of myelodysplastic syndrome. Grudeva-Popova, J.[2007]

Several new therapeutic agents are being developed for myelodysplastic syndromes (MDS), including clofarabine, ezatiostat, tipifarnib, laromustine, and histone deacetylase inhibitors, each with different mechanisms of action.

Despite the challenges of MDS, which often leads to complications within 3 to 5 years of diagnosis, the ongoing clinical trials suggest that new treatment options may become available in the next 5 to 10 years.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Novel therapies for myelodysplastic syndromes. Steensma, DP.[2018]

References

1. Cyprus pubmed.ncbi.nlm.nih.gov

New options in the treatment of myelodysplastic syndrome. [2007]

2. England pubmed.ncbi.nlm.nih.gov

Age and comorbidities deeply impact on clinical outcome of patients with myelodysplastic syndromes. [2015]

3. Germany pubmed.ncbi.nlm.nih.gov

The new diagnostic criteria for myelodysplasia-related acute myeloid leukemia is useful for predicting clinical outcome: comparison of the 4th and 5th World Health Organization classifications. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Health-related Quality of Life Profile of Newly Diagnosed Patients With Myelodysplastic Syndromes by Age, Sex, and Risk Group: A Real-world Study by the GIMEMA. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Novel therapies for myelodysplastic syndromes. [2018]