What is the mechanism of action of this treatment?

Common treatments for Myelodysplastic Syndrome (MDS) include DNA methyltransferase inhibitors like azacitidine and decitabine, which work by reactivating tumor suppressor genes through hypomethylation of DNA. Lenalidomide, particularly effective in patients with del(5q) MDS, modulates the immune system and promotes the repopulation of normal bone marrow cells. Targeted therapies and immunotherapies, such as those being studied with CC-95251, aim to enhance the body's immune response against malignant cells or directly target specific pathways involved in the disease. Understanding these mechanisms is crucial for MDS patients as it helps in selecting the most effective treatment, predicting responses, and managing potential side effects.

What side effects are associated with this type of intervention?

Common treatments for Myelodysplastic Syndrome (MDS), such as azacitidine and decitabine, are associated with side effects including cytopenias (low blood cell counts), gastrointestinal symptoms (nausea, vomiting, diarrhea), and injection site reactions. Targeted therapies and immunotherapies, similar to those studied in the trial CC-95251, may also cause myelosuppression, fatigue, infections, and potential liver enzyme elevations. Patients should discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

For the treatment of Myelodysplastic Syndrome (MDS), the FDA has approved hypomethylating agents such as azacitidine and decitabine. These agents are commonly used to manage MDS and have shown efficacy in improving overall survival and quality of life. Additionally, the FDA has approved the use of thrombopoietin mimetics like eltrombopag for certain cases of MDS with severe thrombocytopenia. While not specifically approved for MDS, IDH inhibitors like ivosidenib (for IDH1 mutations) are used in combination with HMAs for targeted therapy in related conditions such as acute myeloid leukemia (AML).

What other types of research exist?

Promising novel alternatives to CC-95251 for Myelodysplastic Syndrome patients include hypomethylating agents (HMAs) like azacitidine and decitabine, which have shown comparable efficacy and tolerability. Additionally, venetoclax combined with low-dose cytarabine (LoDAC) is a potential option, especially for patients who are not candidates for HMA-based treatment. Other emerging therapies include anti-CD47 agents, which are currently in clinical trials and showing promising early results.

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CC-95251 for Leukemia

Phase 1

Waitlist Available

Research Sponsored by Bristol-Myers Squibb

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

TN AML as defined by the 2016 WHO Classification, including secondary AML and therapy-related AML in participants who are ineligible (IE) for intensive chemotherapy (IC) and allogeneic hematopoietic stem cell transplant (HSCT)

R/R myelodysplastic syndromes (MDS) as defined by the 2016 WHO Classification with intermediate, high or very high risk by Revised International Prognostic Scoring System (IPSS-R)

Must not have

Prior systemic cancer-directed treatments or investigational modalities ≤ 5 half-lives or 4 weeks prior to starting study treatment, whichever is shorter (relapsed or refractory participants only)

Acute promyelocytic leukemia

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug called CC-95251 to see if it can help treat patients with difficult-to-treat blood cancers. The study will check if the drug is safe and effective when used alone or with other cancer treatments.

Who is the study for?

This trial is for adults with relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), as well as treatment-naive MDS at higher risk. Participants should be able to perform daily activities with minimal assistance. They can't join if they have life-threatening complications, prior treatments with similar drugs, uncontrolled infections, recent cancer treatments, are on immunosuppressants, or are pregnant/nursing.

What is being tested?

The study tests the safety and effectiveness of CC-95251 alone and combined with other cancer drugs in treating AML and MDS. It's looking at how well patients respond to the treatment and what side effects occur when using CC-95251 with Azacitidine and Venetoclax.

What are the potential side effects?

Possible side effects include reactions related to the immune system such as fever or chills, fatigue from anemia or low blood counts due to bone marrow suppression by Azacitidine/Venetoclax, bleeding issues from low platelets count, infection risks due to weakened immunity.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a type of leukemia that can't be treated with intense chemo or stem cell transplant.

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My condition is a type of blood disorder classified as intermediate, high, or very high risk.

Select...

I can take care of myself and am up and about more than half of my waking hours.

Select...

My condition is relapsed or refractory acute myeloid leukemia.

Select...

My MDS is untreated and is intermediate, high, or very high risk.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I haven't had cancer treatments or experimental drugs within the last 4 weeks or 5 half-lives, whichever is shorter.

Select...

I have been diagnosed with acute promyelocytic leukemia.

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I have been treated with a CD47 or SIRPα targeting agent before.

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I do not have severe, life-threatening complications from leukemia.

Select...

I am on long-term medication to suppress my immune system.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: CC-95251 monotherapyExperimental Treatment1 Intervention

Group II: CC-95251 + azacitidine + venetoclaxExperimental Treatment1 Intervention

Group III: CC-95251 + azacitidineExperimental Treatment2 Interventions

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CC-95251

2022

Completed Phase 1

~60

Azacitidine

2012

Completed Phase 3

~1440

Venetoclax

2019

Completed Phase 3

~2240

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Myelodysplastic Syndrome (MDS) include DNA methyltransferase inhibitors like azacitidine and decitabine, which work by reactivating tumor suppressor genes through hypomethylation of DNA. Lenalidomide, particularly effective in patients with del(5q) MDS, modulates the immune system and promotes the repopulation of normal bone marrow cells. Targeted therapies and immunotherapies, such as those being studied with CC-95251, aim to enhance the body's immune response against malignant cells or directly target specific pathways involved in the disease. Understanding these mechanisms is crucial for MDS patients as it helps in selecting the most effective treatment, predicting responses, and managing potential side effects.

SOHO State of the Art and Next Questions: Management of Myelodysplastic Syndromes With Deletion 5q.Lenalidomide as a disease-modifying agent in patients with del(5q) myelodysplastic syndromes: linking mechanism of action to clinical outcomes.Combination therapy with DNA methyltransferase inhibitors in hematologic malignancies.