Selumetinib for Cancer

No longer recruiting at 120 trial locations
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness of selumetinib sulfate (also known as AZD6244, MEK162, or Koselugo) in treating certain cancers that have spread, recurred, or resisted treatment. The focus is on cancers involving solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with a specific gene mutation known as MAPK pathway activation. The treatment may halt cancer growth by blocking enzymes necessary for cell growth. Suitable candidates for this trial include patients with these cancers who have measurable disease and have not responded to other treatments. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

Yes, you may need to stop taking certain medications. The trial does not allow participants to take other anti-cancer agents, investigational drugs, or certain medications that affect specific liver enzymes (CYP3A4 and CYP2C19). If you are on corticosteroids, you must be on a stable or decreasing dose for at least 7 days before joining the trial.

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

Research has shown that selumetinib sulfate has been safe in past studies. In one study with 74 children and teens, participants took selumetinib for up to 7.7 years, and no new safety issues emerged. This suggests the treatment is well-tolerated.

Another study with adults found that selumetinib reduced tumor size and pain. While this primarily demonstrates the drug's effectiveness, it also suggests safety for ongoing use.

Overall, these studies indicate that selumetinib is generally safe for both children and adults. However, like any treatment, some side effects may occur. Always discuss potential risks and benefits with a healthcare provider.12345

Why do researchers think this study treatment might be promising?

Selumetinib is unique because it targets a specific pathway in cancer cells known as the MEK pathway. While many standard cancer treatments, like chemotherapy, attack rapidly dividing cells in general, selumetinib specifically inhibits this pathway, which is often overactive in certain cancers. This targeted approach means selumetinib might offer a more personalized treatment option with potentially fewer side effects compared to traditional treatments. Researchers are excited about selumetinib because it represents a shift towards precision medicine, focusing on the molecular characteristics of cancer rather than a one-size-fits-all approach.

What evidence suggests that selumetinib sulfate might be an effective treatment for cancer?

Research has shown that selumetinib, the treatment under study in this trial, can help shrink tumors in some cancer patients. One study found that 66% of patients experienced a reduction in tumor size by at least 20%. Evidence also indicates that selumetinib can slow tumor growth and extend the period during which the cancer does not worsen. This treatment blocks certain enzymes that cancer cells need to grow. These promising results suggest that selumetinib could effectively treat cancers with specific genetic mutations.12467

Who Is on the Research Team?

CE

Carl E Allen

Principal Investigator

Children's Oncology Group

Are You a Good Fit for This Trial?

This trial is for patients with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have MAPK pathway mutations and are not responding to treatment. Participants must be able to swallow capsules, have a body surface area >= 0.5 m^2, and meet specific health criteria including organ function and blood counts. Pregnant women or those on certain medications cannot join.

Inclusion Criteria

I have recovered from side effects of my previous cancer treatments.
My stem cell treatment followed the required time guidelines.
My cancer has a specific BRAF V600 mutation.
See 9 more

Exclusion Criteria

Pregnant or breast-feeding women
I am currently taking other medications.
I do not have any infections that are currently uncontrolled.
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive selumetinib sulfate 25 mg/m2/dose orally twice daily on days 1-28. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.

Up to 2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 5 years

What Are the Treatments Tested in This Trial?

Interventions

  • Selumetinib Sulfate
Trial Overview The study tests Selumetinib Sulfate's effectiveness in treating relapsed or refractory cancers with MAPK mutations. It's a phase II trial aiming to see if this drug can halt cancer cell growth by inhibiting necessary enzymes.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Treatment (selumetinib)Experimental Treatment3 Interventions

Selumetinib Sulfate is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Koselugo for:
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Approved in European Union as Koselugo for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials
14,080
Recruited
41,180,000+

Citations

Efficacy and safety of selumetinib in adults with ...The observations of reduction in tumour volume by cycle 16, reduction in chronic and spike pain, reduction in analgesia, and decrease in pain ...
2.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40473450/
Efficacy and safety of selumetinib in adults with ... - PubMedThe observations of reduction in tumour volume by cycle 16, reduction in chronic and spike pain, reduction in analgesia, and decrease in pain ...
3.koselugohcp.comkoselugohcp.com/efficacy
Koselugo® (selumetinib) | EfficacyKoselugo delivered a strong response with evidence from long-term data ... View the long-term safety profile here. ... At DCO June 2018, 66% (33/50) achieved ≥20% ...
KOSELUGO® (selumetinib) Showed Significant and ...These results showed reduction in tumor volume, building on the established safety and efficacy profile of KOSELUGO in certain children.
Clinical Review - Selumetinib (Koselugo) - NCBI - NIHThe results suggest selumetinib confers a benefit in terms of reduction in the rate of tumour growth and improvement in PFS. However, due to important between- ...
Why KoselugoThese data include 74 children and teens who received a Koselugo dose of 20 mg/m2 to 30 mg/m2 twice daily for up to 7.7 years with no new safety signals ...
Observational Study to evaluate the effect and safety of ...Selumetinib is an oral selective inhibitor of MEK 1 and 2 and has been confirmed effective for NF1-associated PN in SPRINT Phase II Stratum 1.
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