Elritercept for Myelodysplastic Syndrome

Recruiting at 188 trial locations

Overseen By Keros Therapeutics, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Keros Therapeutics, Inc.

Must not be taking: Antidepressants, Antipsychotics, others

Disqualifiers: Cardiovascular disease, Stroke, HIV, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 10

225 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how well elritercept, a new treatment, can reduce the need for red blood cell transfusions in people with myelodysplastic syndrome (MDS), a condition where the bone marrow doesn't produce enough healthy blood cells. The researchers aim to determine if elritercept can assist those who often require transfusions and ensure the treatment is safe and tolerated over time. Participants will receive either elritercept or a placebo (inactive treatment) every four weeks for up to 48 weeks. People with MDS who depend on frequent transfusions and have not responded well to other treatments might be a good fit for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, certain treatments like iron chelation, vitamin B12 or folate therapy, androgen use, and high-dose corticosteroids must be stable for a specified period before joining. It's best to discuss your specific medications with the study team.

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

Research has shown that elritercept is generally safe for individuals with low-risk myelodysplastic syndrome (MDS). Studies have found it to be well-tolerated over time. Some patients experienced improved blood cell counts and fewer symptoms of anemia, a condition characterized by insufficient healthy red blood cells. Importantly, these studies have identified no major safety issues, suggesting that elritercept could help reduce the need for red blood cell transfusions. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Elritercept is unique because it targets the TGF-beta superfamily, which plays a key role in blood cell production. Unlike current treatments for Myelodysplastic Syndrome (MDS), which often involve supportive care like blood transfusions or drugs that stimulate blood cell production, Elritercept directly addresses the underlying issues in blood cell development. Researchers are excited about Elritercept because it offers a novel approach that could improve blood counts and reduce the need for transfusions, potentially leading to better quality of life for patients with MDS.

What evidence suggests that elritercept might be an effective treatment for myelodysplastic syndrome?

Research shows that elritercept, which participants in this trial may receive, can greatly reduce the need for red blood cell transfusions in people with myelodysplastic syndromes. These syndromes are a group of cancers where blood cells in the bone marrow fail to develop into healthy blood cells. Previous studies have found that elritercept boosts hemoglobin levels, which is crucial because hemoglobin carries oxygen in the blood. Elritercept is also safe for patients with less severe conditions and may improve overall blood health. It helps increase blood cell counts and ease symptoms of anemia, a condition characterized by a lack of healthy red blood cells. Overall, elritercept shows promise in improving life for those who need frequent transfusions. ^{1 2 3 4 5}

Who Is on the Research Team?

Study Director

Principal Investigator

Takeda

Are You a Good Fit for This Trial?

Adults over 18 with very low to intermediate risk Myelodysplastic Syndromes (MDS) and anemia who need regular blood transfusions can join. They must understand the study, have had unsuccessful or intolerant responses to prior treatments like ESAs, and agree to use contraception. Those with more than 5% bone marrow blasts or poor physical condition cannot participate.

Inclusion Criteria

My latest bone marrow test shows less than 5% immature cells.

I am able to get out of my bed or chair and move around.

I agree to use effective birth control methods if I can have children or am a sexually active male.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-blind Treatment - Primary Phase

Participants receive either KER-050 or placebo subcutaneously every 4 weeks

24 weeks

Study visits every 2 weeks from Cycle 1 through Cycle 6 and every 4 weeks from Cycle 7

Double-blind Treatment - Secondary Phase

Continuation of double-blind treatment for participants who meet criteria after the Primary Phase

24 weeks

Study visits every 4 weeks

Double-blind Treatment - Extension Phase

Eligible participants continue receiving the same double-blind treatment until study unblinding or individual discontinuation

Variable, based on individual criteria

Safety Follow-up

Participants are monitored for safety after the last dose of study treatment

8 weeks

Study visits every 4 weeks

Long-term Follow-up

Quarterly follow-up for up to 5 years from the first dose or 3 years after the last dose

Up to 5 years

What Are the Treatments Tested in This Trial?

Interventions

• Elritercept

Trial Overview The trial is testing Elritercept (KER-050) against a placebo in adults with MDS-related anemia requiring transfusions. Participants are randomly assigned in a 2:1 ratio to receive either the drug or placebo during various phases including treatment and follow-up periods.

How Is the Trial Designed?

Group I: Elritercept Experimental Treatment 1 Intervention

Participants will be administered elritercept (TAK-226, KER-050), subcutaneously every 4 weeks, up to 48 weeks or until the end of treatment period.

Group II: Placebo Placebo Group 1 Intervention

Participants will be administered elritercept (TAK-226, KER-050) matching-placebo, subcutaneously every 4 weeks, up to 48 weeks or until the end of treatment period.

Who Is Running the Clinical Trial?

Keros Therapeutics, Inc.

Lead Sponsor

Trials

5

Recruited

600+

Takeda

Lead Sponsor

Trials

1,255

Recruited

4,219,000+

Dr. Naoyoshi Hirota

Takeda

Chief Medical Officer since 2020

MD from University of Tokyo

Christophe Weber

Takeda

Chief Executive Officer since 2015

PhD in Molecular Biology from Université de Montpellier

PSI CRO

Industry Sponsor

Trials

13

Recruited

2,800+

Published Research Related to This Trial

The TGF-B pathway is overactive in lower-risk myelodysplastic syndromes (MDS), leading to myelosuppression and contributing to anemia, which is a key treatment target.

Novel agents like luspatercept and sotatercept, which act as ligand traps for activin receptors, have shown early promise in treating anemia in MDS, particularly in patients with specific mutations, suggesting a new approach to therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Activin Receptor II Ligand Traps: New Treatment Paradigm for Low-Risk MDS. Komrokji, RS.[2020]

Recent research has identified new molecular therapeutic targets for myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), leading to the development of promising new drug classes such as farnesyltransferase inhibitors and receptor tyrosine kinase inhibitors.

Early clinical trials suggest these new drugs may change the standard treatment for MDS, focusing on resolving blood cell deficiencies and targeting the disease's specific mechanisms, although most studies are still in the early stages of development.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Inhibitors of signaling in myelodysplastic syndrome. Gore, SD.[2009]

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]

There have been no new drug approvals for myelodysplastic syndromes (MDS) in 13 years, highlighting a significant gap in treatment advancements since decitabine was approved in 2006.

Emerging therapies like luspatercept and imetelstat show promise for treating symptomatic anemia in lower-risk MDS patients, and future treatments are expected to be more personalized based on genetic and biomarker analyses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evolving therapies for lower-risk myelodysplastic syndromes. Bewersdorf, JP., Zeidan, AM.[2020]

Luspatercept has been approved for treating lower-risk myelodysplastic syndromes (LR-MDS) with specific mutations, showing promise in reducing anemia and potentially replacing erythropoiesis-stimulating agents as a first-line treatment.

Current therapies for LR-MDS, including ESAs and hypomethylating agents, are effective in less than 50% of patients, highlighting the need for new treatments targeting different biological pathways to improve patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Next-generation therapy for lower-risk MDS. Sébert, M.[2023]

Citations

1. ir.kerostx.com ir.kerostx.com/news-releases/news-release-details/keros-therapeutics-presents-clinical-data-its-elritercept

Keros Therapeutics Presents Clinical Data from its Elritercept ... Elritercept demonstrated a durable transfusion independence in lower-risk myelodysplastic syndromes, including in patients with high ...

2. ashpublications.org ashpublications.org/ashclinicalnews/news/8751/the-efficacy-and-safety-of-elritercept-in-adult

The Efficacy and Safety of Elritercept in Adult Participants With ... Elritercept, by not only improving hemoglobin levels and reducing transfusion dependence but also enhancing other cell lineages and improving ...

3. healthtree.org healthtree.org/mds/community/articles/how-effective-is-elritercept-low-risk-mds-patients

How Effective Is Elritercept for Low-risk MDS? Elritercept is generally safe for low-risk MDS patients. It also has the potential to improve patients' blood cell counts, alleviate certain symptoms of anemia ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06499285

NCT06499285 | A Study of Elritercept to Treat Anemia in ... The main aim of this study is to find out how well elritercept works in lowering the need for RBC transfusions. Other aims are to learn how well elritercept ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10428326/

S166: KER-050 TREATMENT IMPROVED MARKERS OF ... KER-050 was generally well-tolerated with longer-term treatment and has the potential to drive hematological improvement and reduce transfusion burden.