Digoxin for Medulloblastoma

Recruiting at 18 trial locations

Overseen By Jessica Crimella

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Must not be taking: Digoxin, Metabolism interferers

Disqualifiers: Cardiac arrhythmias, Uncontrolled illness, Psychiatric, 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

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 4 months

23 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness of digoxin (also known as Lanoxin or Digitalis) in treating relapsed medulloblastoma, a type of brain cancer, in children and young adults. Researchers are focusing on participants with non-SHH and non-WNT forms of this cancer, which have returned after treatment. Eligible participants should have previously undergone chemotherapy and radiation and have a visible tumor on an MRI. This trial is for those who have recovered from any recent surgeries and do not have serious heart issues. As a Phase 2 trial, the research measures how well the treatment works in an initial, smaller group, offering participants a chance to contribute to important advancements in cancer treatment.

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

The trial does not specify if you need to stop taking your current medications, but you cannot take medications that interfere with digoxin metabolism or any other investigational agents. It's best to discuss your current medications with the study team.

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

Research shows that digoxin, a drug typically used for heart problems, might also help fight certain cancers. Lab studies have demonstrated its potential against medulloblastoma, a type of brain tumor. Some research suggests that digoxin could be safe for cancer patients if doctors carefully adjust the doses.

This study is in an early stage, so researchers are still testing its safety. However, since digoxin has been used for many years to treat heart issues, doctors have some knowledge about its safety. This experience is useful, but using it for cancer is different and requires careful monitoring.

Before deciding to participate, it's important to talk with a healthcare provider about any concerns. They can provide more detailed information about how well digoxin has been tolerated in other situations. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for medulloblastoma?

Unlike the standard treatments for medulloblastoma, which typically involve surgery, radiation, and chemotherapy, Digoxin offers a unique approach by utilizing a well-known cardiac drug for cancer treatment. Researchers are excited about Digoxin because it targets cancer cells by disrupting their sodium and potassium ion balance, a mechanism different from traditional chemotherapies. This repurposing of a cardiac drug could potentially provide a less toxic and more targeted option for patients, sparking hope for improved outcomes with fewer side effects.

What evidence suggests that digoxin might be an effective treatment for medulloblastoma?

Research suggests that digoxin, which participants in this trial will receive, may help treat certain types of brain cancer called medulloblastoma. In studies with mice, digoxin helped them live longer by shrinking their tumors. Specifically, one study showed that digoxin increased survival in a type of medulloblastoma known as group 4. Although these results come from animal studies, they indicate that digoxin might benefit the treatment of certain brain cancers in humans. ^{1 2 4 6 7}

Who Is on the Research Team?

Jonathan Metts, MD

Principal Investigator

Moffitt Cancer Center

Laura Metrock, MD

Principal Investigator

University of Alabama at Birmingham Children's of Alabama

Are You a Good Fit for This Trial?

This trial is for pediatric and young adult patients aged >12 months and <30 years with relapsed non-SHH, non-WNT medulloblastoma. They must have measurable disease on MRI, stable neurological status, specific cardiac criteria met, normal organ/marrow function, and a performance score of ≥50%. Prior chemotherapy and irradiation are required. Contraception use is necessary for those who can bear children.

Inclusion Criteria

My heart health meets the required standards, and I've seen a cardiologist if needed.

My neurological condition has been stable for at least 7 days.

Patients must have specific timeframes since prior therapy: craniospinal radiotherapy (≥18 Gy): at least 3 months, local radiotherapy to primary tumor: at least 3 months, focal radiotherapy to symptomatic metastatic sites: at least 2 weeks, myelosuppressive chemotherapy and/or immunotherapy and/or biologics: more than 3 weeks (6 weeks for nitrosoureas), hematopoietic growth factor: 7 days

Exclusion Criteria

Participants unable to comply with safety monitoring requirements are ineligible

I am not currently taking any cancer treatments or experimental drugs.

I do not have serious or uncontrolled heart rhythm problems.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Digoxin will be administered orally at a standard maintenance dosing. Each cycle will be 28 days.

4 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 months

What Are the Treatments Tested in This Trial?

Interventions

• Digoxin

Trial Overview The study tests the effectiveness of digoxin in treating specific types of medulloblastoma that have returned after previous treatments. It focuses on patients who do not respond to standard therapies for this brain tumor affecting children and young adults.

How Is the Trial Designed?

Group I: Digoxin Treatment Experimental Treatment 1 Intervention

Digoxin will be administered orally at a standard maintenance dosing. Each cycle will be 28 days.

Digoxin is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Lanoxin for:

• Atrial fibrillation
• Atrial flutter
• Supraventricular tachycardia
• Congestive heart failure

Approved in United States as Lanoxin for:

• Atrial fibrillation
• Atrial flutter
• Supraventricular tachycardia
• Congestive heart failure

Approved in Canada as Lanoxin for:

• Atrial fibrillation
• Atrial flutter
• Supraventricular tachycardia
• Congestive heart failure

Approved in Japan as Lanoxin for:

• Atrial fibrillation
• Atrial flutter
• Supraventricular tachycardia
• Congestive heart failure

Who Is Running the Clinical Trial?

H. Lee Moffitt Cancer Center and Research Institute

Lead Sponsor

Trials

576

Recruited

145,000+

National Pediatric Cancer Foundation

Collaborator

Trials

8

Recruited

300+

Published Research Related to This Trial

In a study of nearly 3 million hospital admissions in The Netherlands from 2001-2004, digoxin intoxication was found in only 0.04% of cases, indicating that such incidents are infrequent among patients receiving digoxin therapy.

Women were found to be 1.4 times more likely to experience digoxin intoxication compared to men, highlighting a gender-specific risk factor in digoxin therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Age- and gender-specific incidence of hospitalisation for digoxin intoxication. Aarnoudse, AL., Dieleman, JP., Stricker, BH.[2018]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06701812

NCT06701812 | Digoxin Medulloblastoma Study The purpose of this study is to evaluate the efficacy of digoxin in treating relapsed non-SHH, non-WNT medulloblastoma in pediatric and young adult patients.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6644046/

Systems biology-based drug repositioning identifies digoxin ... Together, our data suggest that digoxin has an antitumor effect in vivo against preformed orthotopic PDX tumors from both groups 3 and 4 MB.

3. withpower.com withpower.com/trial/phase-2-medulloblastoma-10-2024-452a0

Digoxin for Medulloblastoma Digoxin. Trial Overview The study tests the effectiveness of digoxin in treating specific types of medulloblastoma that have returned after previous treatments.

4. today.uconn.edu today.uconn.edu/2018/10/old-drug-new-hope-pediatric-brain-cancer/

Old Drug, New Hope for Pediatric Brain Cancer - UConn Today The researchers also showed that one of the heart drugs, digoxin, helped mice with medulloblastomas live longer. The mice survived even longer ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5464078/

P11.08 Systematic drug repurposing for faster cures of ... P11.08 Systematic drug repurposing for faster cures of pediatric cancer identifies that Digoxin prolongs survival in a PDOX model of group 4 medulloblastoma.

6. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2025-01884

Digoxin for the Treatment of Pediatric and Young Adult ... Giving digoxin may kill more tumor cells in pediatric and young adult patients with relapsed non-SHH, non-WNT medulloblastoma. Eligibility Criteria ...

7. aacrjournals.org aacrjournals.org/cancerres/article/77/13_Supplement/3214/618955/Abstract-3214-Systematic-drug-repurposing-for

Abstract 3214: Systematic drug repurposing for faster cures of ... Histological evaluation of recurrent tumors following digoxin treatment demonstrated changes in the pattern of tumor spread, vascularity and ...