Recruiting

Phase 2

DigoxinDigoxin Medulloblastoma Study

Sponsor:

H. Lee Moffitt Cancer Center and Research Institute

Code:

NCT06701812

Conditions

Medulloblastoma

Medulloblastoma, Non-WNT/Non-SHH

Eligibility Criteria

Sex: All

Age: 1 - 30

Healthy Volunteers: Not accepted

Interventions

Digoxin

Study Details

Brief summary:

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.

Conditions

Medulloblastoma

Medulloblastoma, Non-WNT/Non-SHH

Study ID

NCT06701812

Start date

Jan 15, 2026

Status verified date

Aug, 2026

Completion date

Feb, 2027

Anticipated

Primary completion date

Feb, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 1 - 30

Healthy Volunteers: Not accepted

Inclusion Criteria:

Patients must be age >12 months and <30 years at the time of enrollment.
Patients must have relapsed non-WNT, non-SHH medulloblastoma confirmed by a CAP/CLIA certified assay (such as nanostring or methylation) performed on tissue from diagnosis or relapse.
Patients must have received at least one prior course of chemotherapy for their medulloblastoma. They must also have received irradiation.
Prior therapy: Therapy may not have been received more recently than the timeframes defined below: Craniospinal radiotherapy: At least 3 months have elapsed since prior craniospinal radiotherapy (at doses ≥ 18 Gy). Local radiotherapy: At least 3 months since prior local radiotherapy to primary tumor. Focal radiotherapy: At least 2 weeks since prior focal radiotherapy to symptomatic metastatic sites. Myelosuppressive chemotherapy and/or immunotherapy and/or biologics: More than 3 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosoureas), immunotherapy, or biologics. Hematopoietic growth factor: Seven days must have elapsed since the completion of therapy with colony-stimulating factors (e.g., filgrastim \[G-CSF\], sargramostim \[GM-CSF\], or erythropoietin), or platelet-stimulating agents.
Patients must have recovered from any surgical procedures such as biopsy, with neurological stability for > 7 days.
Patients must have clear residual disease, defined as tumor that is measurable in two perpendicular diameters on MRI (ie, largest tumor diameter and its largest perpendicular). The size of a measurable lesion at baseline should be at least 2 times the thickness of the slices showing the tumor (adding the interslice gap).
Patients must have a Lansky or Karnofsky performance status score of ≥ 50%. Use Karnofsky for patients > 16 years of age and Lansky for patients < 16 years of age. Patients who are unable to ambulate but who are functional in a wheelchair will be considered ambulatory for the purpose of assessing the performance score.
Patients must have normal organ and marrow function.
Patient has no evidence of Wolff-Parkinson-White syndrome or high-grade AV block (form of second-degree heart block) on screening ECG.
Patient has no evidence of hypertrophic obstructive cardiomyopathy on screening echo.
Any patient that reports recent palpitations (within the last month), or concerning findings on echo or ECG must be evaluated and cleared for treatment with digoxin by a cardiologist prior to enrollment. Study PI should be contacted for additional questions/concerns regarding these patients.
Patients receiving concurrent dexamethasone are eligible, provided dosage is stable or decreasing for ≥7 days prior to study enrollment.
Patients must have a stable neurologic status for ≥7 days prior to study enrollment. If a patient experiences neurologic decline following enrollment but prior to day 1 of cycle 1, they should be reassessed for eligibility.
Pregnancy: Females of childbearing potential must have a negative urine or serum pregnancy test prior to enrollment. Female patients who are lactating must agree to stop breastfeeding.
Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
All patients and/or their parents or legal guardians must have the ability to understand and the willingness to sign a written informed consent or assent document.

Exclusion Criteria:

Participants who are receiving concurrent anticancer or any other investigational agents are ineligible.
Participants taking digoxin for any reason during treatment for initial diagnosis of medulloblastoma or relapse are ineligible. Exposure to digoxin therapy prior to initial diagnosis of medulloblastoma is allowed.
Patients with a history of allergic reactions attributed to compounds of similar chemical or biologic composition to digoxin are ineligible.
Patients with serious or inadequately controlled cardiac arrhythmias, including baseline ectopy, ventricular tachycardia, frequent premature ventricular contractions (PVCs), or symptomatic sinus bradycardia are excluded from the study.
Patients taking medications that are known to interfere with digoxin metabolism are ineligible.
Participants with uncontrolled intercurrent illness, concurrent clinically significant unrelated systemic illness (e.g. serious infection) or significant cardiac, pulmonary, hepatic, or other organ dysfunction that would compromise the patient's ability to tolerate study treatment or would likely interfere with study procedures or results are ineligible.
Participants with psychiatric illness/social situations that would limit compliance with study requirements are ineligible.
Pregnant women or women unwilling to stop breastfeeding are excluded from this study because it is unknown how pregnant women with recurrent medulloblastoma will metabolize and tolerate digoxin. There is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with digoxin in this setting.
Participants who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study.

Study Design

Enrollment

23 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Digoxin Treatment

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

Interventions

Digoxin

2.5-10 mcg/kg/day orally divided twice daily or once daily based on age on a continuous dosing schedule.

Primary outcome measure

Progression Free Survival at 4 months (PFS4) [ Time Frame: 4 months ]

Central Contacts and Locations

Central contacts

Jessica Crimella

813-745-6250 Jessica.Crimella@moffitt.org

Locations

University of Alabama at Birmingham Children's of Alabama

Recruiting

Birmingham, Alabama, United States, 35233

Principal Investigator:

Laura Metrock, MD

Phoenix Childrens Hospital

Recruiting

Phoenix, Arizona, United States, 85016

Principal Investigator:

Ross Mangum, MD

Arkansas Childrens Hospital

Recruiting

Little Rock, Arkansas, United States, 72202

Principal Investigator:

David Douglass, MD

Connecticut Children's Medical Center

Recruiting

Hartford, Connecticut, United States, 06106

Principal Investigator:

Michael Isakoff, MD

Children's National Medical Center

Recruiting

Washington D.C., District of Columbia, United States, 20010

Principal Investigator:

Eugene Hwang, MD

University of Miami

Recruiting

Miami, Florida, United States, 33136

Principal Investigator:

Bradley Gampel, MD

Johns Hopkins All Children's

Recruiting

St. Petersburg, Florida, United States, 33701

Principal Investigator:

Stacie Stapleton, MD

St. Joseph's Children's Hospital

Recruiting

Tampa, Florida, United States, 33607

Principal Investigator:

Trisha Larkin, MD

University of Kentucky

Recruiting

Lexington, Kentucky, United States, 40536

Principal Investigator:

Tom Badgett, MD, PhD

Johns Hopkins Sidney Kimmel Comprehensive Cancer Center

Recruiting

Baltimore, Maryland, United States, 21218

Principal Investigator:

Kenneth Cohen, MD

Washington University St. Louis

Recruiting

St Louis, Missouri, United States, 63130

Principal Investigator:

Mohamed Abdelbaki, MD

Montefiore Medical Center

Recruiting

The Bronx, New York, United States, 10467

Principal Investigator:

Alice Lee, MD

UNC Lineberger Comprehensive Cancer Center

Recruiting

Chapel Hill, North Carolina, United States, 27514

Principal Investigator:

David Kram, MD

Levine Cancer Institute

Recruiting

Charlotte, North Carolina, United States, 28204

Principal Investigator:

Erin Trovillion, MD

Vanderbilt University Medical Center

Recruiting

Nashville, Tennessee, United States, 37232

Principal Investigator:

Scott Borinstein, MD

UT Southwestern

Recruiting

Dallas, Texas, United States, 75390

Principal Investigator:

Matthew Campbell, MD

More Information

Sponsor

H. Lee Moffitt Cancer Center and Research Institute

Last update posted

Aug 26, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by H. Lee Moffitt Cancer Center and Research Institute on 2026-08-26.